Archive for the ‘Gene Therapy Research’ Category

That next day, Drake preferred sleeping over eating. But then, thats common with newborns. Tarah and Eric would wake him for feeding, careful to make sure he got plenty of nourishment.

By Saturday, these experienced parents became uneasy. Drake was just too lethargic. It was harder to wake him for feedings. The OSullivans called Drakes doctor and were assured there was nothing to be concerned about; Drake had been healthy when he left the hospital two days ago. And, the doctors office assured them, they would be checking him again on Monday at a scheduled office visit.

But the OSullivans disquiet grew by the hour. By Sunday evening, Drake would not open his eyes or respond to them. He was growing limp and struggling to breathe. The OSullivans rushed Drake to the hospital where the staff flew into emergency mode. Too sick for care at the local hospital, Drake was stabilized for transport to the pediatric intensive care unit (PICU) at Greenville Memorial Hospital. Just 72 hours after birth, Drake lapsed into a coma. And no one knew why.

That unforgettable night was the beginning of a long journey of test after test and a diagnosis by elimination.

Drake continued to decline as each negative test pushed aside another horrible possibility. You would think that eliminating terrible diseases would be a good thing, says Eric. But that just meant we were looking at something very rare.

Finally, blood tests revealed an ever-elevating level of glycine in Drakes blood, a symptom of an extremely rare, genetic metabolic disease called nonketotic hyperglycinemia or NKH.

The words nonketotic hyperglycinemia meant nothing to Tarah and Eric. But the next words were clear: Drake had a less than 10 percent chance of survival.

The diagnosis was like a starters pistol for the OSullivans. From that moment, everything would be a race against time to save Drake.

After 28 days of tests, monitors, tubes and wires, Drake was released to go home. There, as Tarah explains, Our house became a sort of lab. There were blood tests, feedings, medications and monitoring day and night, 24/7. Glycine became the OSullivans obsession as they tried desperately through medication and diet to moderate Drakes levels. They began to search for information, research, treatment, medical advice anything to save his life.

The OSullivans contacted anyone who might know about NKH, have a related research project or could tell them more. They learned that NKH affects fewer than 500 people worldwide and has no cure. There was no research underway, and no funding for research. And because there is no medically recognized cure for NKH, all treatments are considered experimental and not covered by medical insurance. Period.

So Tarah became a lay scientist. She read everything, called and emailed medical researchers and established the Drake Rayden Foundation to raise awareness for NKH, fight for better treatment and support research. She entered a world of genetics and vectors, glycine and metabolic pathways. Tarah had quit college just shy of completing her business degree. Now she desperately needed the scientific expertise that would help her understand the disease and find the cure.

Tarah decided to return to college.

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The Face of Science - Clemson World magazine

The good news is that total U.S. medical and health R&D grew 6.4 percent from 2017 to 2018, topping a total of $194 billion and outpacing overall healthcare spending growth for the third year in a row.

The bad news is that research investment is just 5 cents of every health dollar spent, according to a new report from Research!America.

This growth in R&D investment is positive and welcome, certainly, said Research!Americas chair, the honorable Michael N. Castle. However, our nations total investment is not tracking with disease burden. Increased investment in medical and health R&D is essential to ending diseases that are taking time and quality of life from Americans and people across the globe.

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Academic and research institutions, including colleges and universities, independent research institutes, and independent hospital medical research centers invested $15.7 billion, or 8.1 percent, while foundations put in $2.3 billion or 1.2 percent.

State and local government put in $2.1 billion, or 1.1 percent, and voluntary health and professional groups invested $1.5 billion or 0.8 percent.

Increased investment across all sectors contributing to R&D is the right path for our nation, but relative to unmet medical needs, we are walking, rather than running down that path, Mary Woolley, president and CEO, Research!America, said in a statement. Federal funding and policies need to be aligned behind the objective of empowering both public sector and private sector-driven research, because research saves lives.

Illness that more research investment could combat kills almost 130,000 under age 45 each year, and chronic disease cost the U.S. $1.1 trillion in 2018 almost six times the amount all sectors spend on R&D, according to the report.

In other December research news, Harvard, MIT, GE and Fujifilm formed a Massachusetts-based, $50 million biomed consortium that hopes to smooth the path for cutting-edge gene therapy and cancer immunotherapy from research lab to hospital clinic.

The center will include a board of directors from Harvard University, Massachusetts Institute of Technology, Fujifilm Diosynth Biotechnologies, GE Healthcare Life Sciences, Alexandria Real Estate Equities Inc., and have contributing members from Beth Israel Deaconess Medical Center, Boston Childrens Hospital, Brigham and Womens Hospital, Dana-Farber Cancer Institute and Massachusetts General Hospital, MilliporeSigma, and the Commonwealth of Massachusetts.

"This is a momentous opportunity, noted Martin Meeson, president and COO of Fujifilm Diosynth Biotechnologies, U.S., said at the time. "Our participation as one of the founding members is to enable these very important therapies to be accessible to patients. We seek to bring very much needed expertise and capacity to the one of the leading biotechnology ecosystem in the world."

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U.S. R&D investment 'not tracking with disease burden': report - DOTmed HealthCare Business News

Diabetic retinopathy (DR) is a sight-threatening complication of diabetes and the main cause of blindness in the United States among working adults. Initially, DR progresses as a non-proliferative DR (NPDR) which leads to blinding proliferative DR (PDR). Nearly all people with Type 1 Diabetes (T1D) undergo gradual vision loss over a 20-year period of diabetes, and about 2030% of them progress to the advanced blinding stage of the disease, PDR.

Tight glycaemic management lowers the risk of complications, yet, many diabetic patients develop DR, despite having good glycaemic control. Furthermore, there is no cure or a preventive measure to block PDR. Only recently, over the past decade, with the advent of medicines that block the actions of the vascular endothelial growth factor (VEGF), has considerable progress been made in therapeutic options for PDR.

Nevertheless, not all patients achieve a satisfactory response and many of responders experience frequent invasive intravitreal injections and off-target effects, including increased risk of neuronal toxicity and geographic atrophy. While additional molecular targets, including the plasma kallikrein pathway, lipoprotein-associated phospholipase A2 (Lp-PLA2) and Tie-2, have been identified, with some being clinically evaluated, a critical gap still remains ineffective treatments.

Evidence suggests that targeting DR in the earlier stages, such as mild to moderate NPDR, before permanent damage occurs, would provide long-term benefit. In recent years, our research has focused on the molecular mechanisms of early DR including retinal oxidative stress, mitochondrial dysfunction, mitophagy, inflammation, and premature cell death in diabetes using both in vitro retinal cell cultures and in vivo diabetic rodent models.

Intriguingly, we have discovered a protein called thioredoxin-interacting protein (TXNIP) is strongly induced by diabetes in early DR where it is responsible for mediating cellular oxidative stress, mitophagy, inflammation and premature cell death. Knockdown of TXNIP by intravitreal injection of TXNIP shRNA prevents early molecular defects seen in DR. TXNIP binds to and inhibits the anti-oxidant and thiol-reducing capacity of thioredoxins (Trx) causing cellular redox imbalance and oxidative stress. Trx1 is present in the cytosol and nucleus while Trx2 is located in the mitochondrion. TXNIP is observed in all cellular compartments. Therefore, targeting TXNIP itself or downstream pathways could prevent or slow down the progression of early DR (NPDR) and hence PDR.

Currently, no clinically used, professional TXNIP inhibitors exist; however, several FDA-approved medicines have been reported to interfere with TXNIP pathway, but none of them has been used to treat DR. These drugs include amlexanox, tranilast, and romidepsin and they have been extensively studied for their efficacy, toxicity and safety. This consequently leads to saving time and money and may accelerate their entry to experimental clinical trials centred on targeting an over-activated TXNIP system to arrest DR away from their initial use.

Amlexanox is an inhibitor of TANK-Binding Kinase 1 (TBK1), which phosphorylates mitophagy adaptor optineurin and regulates mitophagic flux to lysosomes. In addition, TBK1 also phosphorylates interferon responsive factor 3 (IRF3) and mediates Type 1 interferon expression and inflammation. Tranilast has been shown to inhibit TXNIP and Nod-like NLRP3 inflammasome; therefore, it may help in preventing cellular oxidative stress and innate immune responses.

Therefore, a combination therapy using amlexanox and tranilast may prove to be effective in preventing or slowing down the progression of DR. Recently, we also demonstrated that a combination therapy of SS-31 (a mitochondria-targeted anti-oxidant), amlexanox and tranilast prevents auranofin-induced redox stress, mitochondrial-lysosomal axis dysregulation and proinflammatory pyroptotic cell death in retinal pigment epithelial cells, suggesting that combination therapies may be more effective than a single drug therapy.

In addition to drug treatment, gene therapy using a TXNIP promoter linked with a neuroprotective factor or an anti-oxidant gene may also be a potential approach for DR treatment. This is because the TXNIP promoter is strongly induced by hyperglycaemia in retinal cells in culture and in diabetic rodent retinas, but not under physiological glucose levels. In addition, the TXNIP promoter is also activated significantly by histone deacetylase inhibitors (HDACi), including suberoylanilide hydroxamic acid (SAHA) or romidepsin.

Therefore, HDACi and TXNIP-promoter gene therapy may also be incorporated in DR therapy for retinal neuroprotective gene expression, which could include pigment epithelium-derived factor (PEDF), glia-derived neurotrophic factor (GDNF), thioredoxin encoded Rod-Derived Cone Viability Factor (RdCVF) and others. These factors are known to be downregulated in DR.

It is currently accepted that neurodegeneration (particularly photoreceptor dysfunction) occurs early in DR before microvascular pathology develops. Therefore, early neuroprotective efforts may constitute a meaningful therapeutical approach to prevent or slow down the progression of late microvascular complications and PDR. Diabetes is a chronic and complex metabolic disease in which PDR develops only after prolonged hyperglycaemic exposure.

Therefore, a window of diabetic duration may exist early to prevent retinal neurovascular dysfunction and progression of PDR; within this time frame, we may devise preventive interventions activating endogenous genes or factors with neuroprotective drugs (preferably orally active drugs) and gene therapy including those mentioned above.

Such a hypothesis is supported by a recent observation, which showed that the expression of retinol binding protein 3 (RBP3), a protein secreted by photoreceptors in the retina, may play a protective role against PDR. RBP3 interacts with glucose transporter Glut1 and reduces excess cellular glucose uptake under hyperglycaemia in diabetics, thus, preventing glucotoxicity in retinal cells including Muller glia and capillary endothelial cells. Those individuals expressing RBP3 do not develop PDR although they have had 50 years of T1D.

In conclusion, new preventive therapies for PDR may be successfully developed by activating endogenous cellular survival mechanisms using drug and gene therapies once a clinical sign of NPDR is observed but before PDR. Such treatments may prevent blindness in diabetic patients.

References

1 Perrone L, Devi TS, Hosoya KI, Terasaki T, Singh LP. Inhibition of TXNIP expression in vivo blocks early pathologies of diabetic retinopathy. Cell Death Dis. 2010 Aug 19;1:e65. PMID: 21364670.

2 Devi TS, Somayajulu M, Kowluru RA, Singh LP. TXNIP regulates mitophagy in retinal Mller cells under high-glucose conditions: implications for diabetic retinopathy. Cell Death Dis. 2017 May 11;8(5):e2777. PMID: 28492550.

3 Lalit PS, Thangal Y, Fayi Y, Takhellambam SD. Potentials of Gene Therapy for Diabetic Retinopathy: The Use of Nucleic Acid Constructs Containing a TXNIP Promoter. Open Access J Ophthalmol. 2018;3(2). PMID: 31106306.

4 Devi TS, Yumnamcha T, Yao F, Somayajulu M, Kowluru RA, Singh LP. TXNIP mediates high glucose-induced mitophagic flux and lysosome enlargement in human retinal pigment epithelial cells. Biol Open. 2019 Apr 25;8(4). PMID: 31023645.

5 Yumnamcha T, Devi TS, Singh LP. Auranofin Mediates Mitochondrial Dysregulation and Inflammatory Cell Death in Human Retinal Pigment Epithelial Cells: Implications of Retinal Neurodegenerative Diseases. Front Neurosci. 2019 Oct 10;13:1065. PMID: 31649499.

6 Yokomizo H, Maeda Y, Park K, Clermont AC, Hernandez SL, et. al., Retinol binding protein 3 is increased in the retina of patients with diabetes resistant to diabetic retinopathy. Sci Transl Med. 2019 Jul 3;11(499). PMID: 31270273.

7 Ibrahim AS, Saleh H, El-Shafey M, Hussein KA, et. al., Targeting of 12/15-Lipoxygenase in retinal endothelial cells, but not in monocytes/macrophages, attenuates high glucose-induced retinal leukostasis. BBA: Mol Cell Biol Lipids. 2017 Jun;1862(6):636-645. PMID: 28351645.

8 Ibrahim AS, Elshafey S, Sellak H, Hussein KA, El-Sherbiny M, et. al., A lipidomic screen of hyperglycemia-treated HRECs links 12/15-Lipoxygenase to microvascular dysfunction during diabetic retinopathy via NADPH oxidase. J Lipid Res. 2015 Mar;56(3):599-611. PMID: 25598081.

Funding

NIH/NEI R01 EY023992 (LSP, OVAS).

NIH/NEI core grant P30EY004068 (LDH, OVAS).

Research to Prevent Blindness (MSJ, OVAS).

American Heart Association Grant 18CDA34080403 to ASI.

Please note: This is a commercial profile

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Repurposing FDA-approved medicines and gene therapy to combat diabetic retinopathy - Open Access Government

Cancer Gene Therapy Market is expected to grow at a Compound Annual Growth Rate (CAGR) of xx%. The base year considered for the study is 2018 and the forecast period considered is 2020 To 2026

Cancer Gene Therapy Market, projects a standardized and in-depth study on the ongoing state of Market, providing basic industry insights such as definitions, classifications, supply chain, applications and industry cost structure. The report precisely delivers productive information about development policies and plans as well as manufacturing processes and techniques.

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Dominant Companies Profiled in this report includes:

Novartis AG, Gilead Sciences,,UniQure N.V. ,Spark Therapeutics LLC, Bluebird Bio, Juno Therapeutics, GlaxoSmithKline PLC, Celgene Corporation,Shire PLC, Sangamo Biosciences,Dimension Therapeutic,Voyager Therapeutics

Key questions answered in the report include:

What will the market size and the growth rate be in 2026?

What are the key factors driving the Cancer Gene Therapy Market?

What are the key market trends impacting the growth of the Cancer Gene Therapy Market?

What are the challenges to market growth?

Who are the key vendors in the Cancer Gene Therapy Market?

What are the market opportunities and threats faced by the vendors in the Cancer Gene Therapy Market?

What are the key outcomes of the five forces analysis of the Cancer Gene Therapy Market?

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For the better regional outlook, analysts examine different global regions such as North America, Latin America, Europe, Asia-Pacific, and India on the basis of different economic attributes like profit margin, shares and pricing structures. Leading key players in the global Cancer Gene Therapy market have been examined by considering different parameters such as productivity, manufacturing base, applications, and raw material. It explores different approaches to augment client base in the domestic and international market.

Major Points Covered in Table of Contents:

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Cancer Gene Therapy Market to grow massively by 2020 with profiling players Novartis AG, Gilead Sciences, UniQure NV, Spark Therapeutics LLC, Bluebird...

For Immediate Release

Chicago, IL January 2, 2019 Zacks Equity Research Keysight Technologies KEYS as the Bull of the Day, Analog Devices ADI as the Bear of the Day. In addition, Zacks Equity Research provides analysis on Alexion Pharmaceuticals, Inc. ALXN, Amarin Corporation plc. AMRN and Cellectis CLLS.

Here is a synopsis of all five stocks:

Bull of the Day:

Keysight Technologiesis a $19 billion provider of electronic design, measurement and test instrumentation systems. Keysight emerged as a public company from the 2014 move by Agilent Technologies to split apart divisions.

KEYS provides specialized electronics solutions to dozens of industries including aerospace and defense, automotive and energy, telecommunications, government and education, and, of course, semiconductors.

Keysight calls the top 25 technology enterprises in the world customers, and 78 of the Fortune 100, including Alphabet, Amazon, AT&T, Broadcom, Boeing, Cisco, Microsoft, NVIDIA, Samsung, Tesla and Toyota.

In fiscal 2019 (ended October), the company generated revenues of $4.312 billion, up 10% over the 2018 tally. In its Q4 reported on November 26, KEYS delivered sales of $1.122 billion, up 7% year over year.

Keysight generated 40.5% of non-GAAP revenues from Americas in fourth-quarter fiscal 2019. Meanwhile, revenues from Europe and Asia Pacific came in at 15.4% and 44.1%, respectively.

Evolving for the Hyper-Speed IoT/5G/AI World

As Keysight has prepared itself for advanced capabilities in wireless and mobile data environments, management sees a transformation from a hardware-centric product company to a software-centric solutions one.

In the company's most recent investor presentation in May, they outlined the evolution of their new "go-to-market" strategy thus...

Old model of scattered selling across separate channels to multiple divisions:

Individual Products with hardware bias

Slower, complex decision-making due to multiple owners interfacing with customers

Incentivized and compensated on parts of a solution

New model of total system designs:

Complete Solutions: Hardware + Software + Services

Faster customer commitments and solution development; one decision owner

Incentivized and compensated on total customer solutions by industry organization

Keysight management also announced their goal to be a top 5G solutions provider and have several first-to-market 5G design wins.

And this strategic focus on "complete solutions" also keeps them at the heart of other bleeding edge innovations in machine learning and artificial intelligence where the requirements for hardware to be embedded with specialized software stacks is only increasing.

Segment Breakdown

Keysight reports under three operating segments namely Communications Solutions Group or CSG, Electronic Industrial Solutions Group or EISG and Ixia Solutions Group (a 2017 acquisition) or ISG. In a bid to remove ambiguity in the reporting processes, Keysight removed Services Solutions Group or SSG as a discrete reportable segment, from first-quarter fiscal 2019.

Under CSG segment (62.9% of the non-GAAP revenues in fourth-quarter fiscal 2019), the company offers radio frequency (RF) and microwave test instruments and allied software, and electronic design automation (EDA) software instruments, laser source products, optical amplifiers, and other software solutions.

EIS Group (25.3%) accounts for design verification devices; general purpose test and measurement equipments; end-to-end manufacturing systems, and material analysis devices.

ISG (11.8%) was formed after conclusion of Ixia buyout in Apr 18, 2017. Under the segment, Keysight offers test and visibility solutions, and software maintenance services.

Quiet, Steady Growth that Investors Favor

Keysight's fortunes and stock price certainly benefited from the tailwinds for the semiconductor industries that were able to shake off supply chain disruptions from the tariff battles.

Even before the hype over 5G, smart technology investors were tuning out the trade war and focusing on the major trends as I described in my 2018 report and video The Technology Super Cycle.

Story continues

But you may be surprised to learn that the 70% advance of KEYS shares in 2019 -- to trade at 4.5X sales and 20X EPS -- is about to be challenged by revenue growth rates for the current fiscal year and next that are expected to slow to around 6.5%, with EPS growth down to just under 10%.

Yet this growth has been enough for Wall Street because its exceeding the plan that KEYS management laid out in 2015 where they expected 4-5% CAGR.

And management is also delivering on its promise to turn 17-18% operating margins into 20%-plus. They certainly keep giving positive surprises with the past 4 quarters averaging a +19% EPS beat.

With global leadership positions in their key markets, the company currently maintains 24% market share in a $16-17 billion addressable market.

That market will keep expanding as new technologies in 5G, datacenters, aerospace and autos evolve. Keysight's strategic "complete solutions" approach to serving the world's biggest tech, hardware, communications and transportation companies will keep them growing at a steady 4-5% right along with those markets -- especially as their customers seek more specialized and custom electronics solutions that KEYS has the platform and vision for.

Bear of the Day:

Analog Devicesis the $44 billion manufacturer of analog, mixed signal and digital signal processing (DSP) integrated circuits, and other semiconductor devices found in cars, planes, factories and home appliances around the globe.

ADI moved to the cellar of the Zacks Rank after reporting a disappointing Q4 fiscal 2019 (ended October) and outlook that caused analysts to take down estimates for their FY20. Adjusted earnings of $1.19 per share missed the Zacks Consensus Estimate of $1.21, as the bottom line decreased 19.6% year over year and 12.5% sequentially.

Revenues of $1.44 billion in the quarter also missed the Zacks Consensus Estimate by 0.6%. And the top line declined 6% year over year and 5.5% from the fiscal third quarter.

This downside can be attributed to weak performance of the company in all end-markets served. Moreover, macroeconomic headwinds negatively impacted the topline.

Analysts responded to the company outlook by dropping full-year 2020 revenue projections to $5.66 billion, representing a 5.46% decline from FY19.

And the 2020 EPS consensus fell 10.3% to $4.78 from $5.33, for a projected 7.2% drop on the bottom line.

Revenues by End Markets

Industrial generated revenue of $744.1 million (accounting for 52% of total revenues), which was flat year over year.

Communications revenue came in at $260.1 million (18% of revenues), decreasing 19% year over year.

Automotive revenue fell to $226.1 million (16% of revenues), down 8% from the year-ago quarter.

Consumer generated revenue of $212.8 million (15% of revenues), reflecting a 7% decline on a year-over-year basis.

Guidance Lowered

For the first quarter of fiscal 2020, Analog Devices expects revenues to be $1.30 billion (+/- $50 million), representing a 15.6% decline from the year ago quarter. Prior to this disappointing guidance, the Zacks Consensus Estimate for Q1 was pegged at $1.41 billion.

Non-GAAP earnings are expected to be $1 (+/- $0.07) per share, reflecting a 24% y-o-y drop. The consensus mark for the same was $1.16 per share.

The company anticipates non-GAAP operating margins to be approximately 36.7% (+/- 100 bps).

Clearly ADI is not going out of business despite being a leader of analog solutions in a digital world.

But until the estimates stop going down and start heading back up, it may be best to stand aside. The Zacks Rank will let you know.

3 Biotechs Likely to Maintain Solid Momentum in 2020

It has been a roller-coaster ride for the volatile biotech sector in 2019 after a disappointing run in 2018. The year started with a bang for this sector, with the announcement of the mega-merger of majors Bristol-Myers and Celgene. This significantly perked up the prices of quite a few stocks. However, these were partially offset by the overall weakness in the global market. Nevertheless, the sector has again picked up in the past couple of months, primarily owing to the recent spree of mergers and acquisitions, and positive pipeline readouts.

Overall, the Nasdaq Biotechnology Index has seen 23.2% growth in the past year.

Relatively, the biotech sector continued being riskier than the more stable large cap pharmaceuticals industry or the overall medical sector, as investors are mostly banking on companies with very few approved drugs in the portfolios.

A slowdown in mature products due to increasing competition and the rise of biosimilars forced most pharma/biotech behemoths to target lucrative buyouts in the biotech space to bolster their portfolios. In particular, biotechs (both small and big), which have a dominant position in the lesser competitive arena of rare diseases, gene therapy and NASH, and are well-equipped with path-breaking technologies, are significant acquisition targets.

A slew of licensing and buyout deals was struck by most companies eyeing smaller entities with impressive pipelines. Novartis recently announced that it will acquire The Medicines Company and add a potentially transformational investigational cholesterol-lowering therapy to its portfolio.

Roche is finally set to acquire Spark Therapeutics after a prolonged delay, while Japanese company Astellas Pharma is taking over gene therapy company Audentes Therapeutics, Inc and has already acquired privately-held, development-stage biotechnology company Xyphos Biosciences, Inc to boost its immuno-oncology pipeline.

Meanwhile, new drug approvals and label expansions of blockbuster drugs boosted investor sentiment. Key approvals include Vyondys 53, Oxbryta, Givlaari, Reblozyl, Trikafta, Inrebic, Vyleesi and Evenity, among others.

Choosing a biotech stock for investment can be tricky as smaller biotechs carry a risk with their product pipelines being several years away from commercialization. Nevertheless, here we zero in a few biotech companies, which have a market capitalization of more than $500 million.

With a favorable Zacks Rank #2 (Buy), these companies are likely to perform well in 2020. You can see the complete list of todays Zacks #1 Rank (Strong Buy) stocks here.

Moreover, the Zacks Biomedical and Genetics industry is placed within the top 22% of the 252 Zacks-ranked industries.

Alexion Pharmaceuticals, Inc. is a biopharmaceutical company, focused on developing and commercializing life-transforming drugs for the treatment of patients with ultra-rare disorders. Its blockbuster drug Soliris approved for paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) continues to perform well.

The drugs label expansion for the generalized myasthenia gravis indication has boosted sales further. The company received a significant boost with the FDA approval of its long-acting C5 complement inhibitor Ultomiris for the treatment of adult patients with PNH.

The approval has strengthened Alexion's PNH franchise and reduced its dependence on Soliris for growth. The company is working on the label expansion of Ultomiris and taking steps to further diversify its pipeline, which should reap returns in the long run.

Shares of Alexion have gained 10.2% in the past year.

Amarin Corporation plc. focuses on developing and commercializing therapeutics to cost-effectively improve cardiovascular health. The company recently obtained FDA approval for the label expansion of its key drug Vascepa. The drug is now approved as an adjunct to maximally tolerated statin therapy to reduce the risk of myocardial infarction, stroke, coronary revascularization and unstable angina requiring hospitalization in adult patients with elevated triglyceride (TG) levels (150 mg/dL) and established cardiovascular disease or diabetes mellitus and two or more additional risk factors for cardiovascular disease.

The label expansion should significantly boost Vascepa sales as, per estimates, millions of high-risk patients in the United States could benefit from this one-of-a-kind prescription therapy. We expect the initial uptake of the drug to be strong and boost the top line, given the market potential.

Amarins shares have surged 54.5% in the past year.

Cellectisis a clinical-stage biopharmaceutical company, focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART). The company is developing life-changing product candidates utilizing TALEN, its proprietary gene-editing technology, and PulseAgile, its pioneering electroporation system, to harness the power of the immune system to target and eradicate cancer cells. The company is striving hard to develop life-saving UCART product candidates to address unmet needs for multiple cancers, including acute myeloid leukemia (AML), B-cell acute lymphoblastic leukemia (B-ALL), multiple myeloma (MM), Hodgkin lymphoma (HL) and non-Hodgkin lymphoma (NHL).

Gene therapy is set to become one of the most vital spaces with high prospects within the volatile biotech sector.

Cellectis shares have gained 3.7% in a year.

Zacks Top 10 Stocks for 2020

In addition to the stocks discussed above, would you like to know about our 10 top tickers for the entirety of 2020?

These 10 are painstakingly hand-picked from over 4,000 companies covered by the Zacks Rank. They are our primary picks to buy and hold. Start Your Access to the New Zacks Top 10 Stocks >>

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Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportKeysight Technologies Inc. (KEYS) : Free Stock Analysis ReportAlexion Pharmaceuticals, Inc. (ALXN) : Free Stock Analysis ReportAmarin Corporation PLC (AMRN) : Free Stock Analysis ReportCellectis S.A. (CLLS) : Free Stock Analysis ReportAnalog Devices, Inc. (ADI) : Free Stock Analysis ReportTo read this article on Zacks.com click here.

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Keysight, Analog Devices, Alexion, Amarin and Cellectis highlighted as Zacks Bull and Bear of the Day - Yahoo Finance

The Global Gene Therapy Market provides a complete market outlook and growth rate during the past present and forecast period. With concise study, Gene Therapy market effectively explains the market value, volume, price trend, and growth opportunities. The market size section gives the Gene Therapy market revenues, covering both the historic growth of the market and forecasting the future. The report covers market characteristics, size and growth, segmentation, regional breakdowns, competitive landscape, market shares, trends and strategies for this market.

Request for sample copy of the Gene Therapy Industry report @: https://www.globalmarketers.biz/report/automotive-and-transportation/global-automotive-gasoline-engine-turbocharger-market-2019-by-manufacturers,-regions,-type-and-application,-forecast-to-2024/130925 #request_sample

As per the world economic growth rate of the past four years, market size is estimated from Gene Therapy million $ in 2014 to Gene Therapy million $ in 2019. The Gene Therapy Market is expected to exceed more than US$ Gene Therapy million by 2024 at a CAGR of xx% in the given forecast period.

This research report categorizes the Gene Therapy industry analysis data by top players, product type, and application.

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The Gene Therapy market report covers the analysis about business overview, market size, share, trends, gross margin, opportunities, challenges and risks factors concerning the market.

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Global Gene Therapy Market- Technology, New Innovations, Opportunities, Future Guidelines, Key Players, Trends and Forecast 2019-2024 -...

San Francisco:In a ray of hope for those who have to go for breast cancer screening and even for healthy women who get false alarms during digital mammography, an Artificial Intelligence (AI)-based Google model has left radiologists behind in spotting breast cancer by just scanning the X-ray results.

Reading mammograms is a difficult task, even for experts, and can often result in both false positives and false negatives.

In turn, these inaccuracies can lead to delays in detection and treatment, unnecessary stress for patients and a higher workload for radiologists who are already in short supply, Google said in a blog post on Wednesday.

Googles AI model spotted breast cancer in de-identified screening mammograms (where identifiable information has been removed) with greater accuracy, fewer false positives and fewer false negatives than experts.

This sets the stage for future applications where the model could potentially support radiologists performing breast cancer screenings, said Shravya Shetty, Technical Lead, Google Health.

Digital mammography or X-ray imaging of the breast, is the most common method to screen for breast cancer, with over 42 million exams performed each year in the US and the UK combined.

But despite the wide usage of digital mammography, spotting and diagnosing breast cancer early remains a challenge, said Daniel Tse, Product Manager, Google Health.

Together with colleagues at DeepMind, Cancer Research UK Imperial Centre, Northwestern University and Royal Surrey County Hospital, Google set out to see if AI could support radiologists to spot the signs of breast cancer more accurately.

The findings, published in the journal Nature, showed that AI could improve the detection of breast cancer.

Google AI model was trained and tuned on a representative data set comprised of de-identified mammograms from more than 76,000 women in the UK and more than 15,000 women in the US, to see if it could learn to spot signs of breast cancer in the scans.

The model was then evaluated on a separate de-identified data set of more than 25,000 women in the UK and over 3,000 women in the US.

In this evaluation, our system produced a 5.7 per cent reduction of false positives in the US, and a 1.2 per cent reduction in the UK. It produced a 9.4 per cent reduction in false negatives in the US, and a 2.7 per cent reduction in the UK, informed Google.

The researchers then trained the AI model only on the data from the women in the UK and then evaluated it on the data set from women in the US.

In this separate experiment, there was a 3.5 per cent reduction in false positives and an 8.1 per cent reduction in false negatives, showing the models potential to generalize to new clinical settings while still performing at a higher level than experts.

Notably, when making its decisions, the model received less information than human experts did.

The human experts (in line with routine practice) had access to patient histories and prior mammograms, while the model only processed the most recent anonymized mammogram with no extra information.

Despite working from these X-ray images alone, the model surpassed individual experts in accurately identifying breast cancer.

This work, said Google, is the latest strand of its research looking into detection and diagnosis of breast cancer, not just within the scope of radiology, but also pathology.

Were looking forward to working with our partners in the coming years to translate our machine learning research into tools that benefit clinicians and patients, said the tech giant.

Read the rest here:
Novel discovery in gene therapy to treat kidney diseases - WeForNews

After years of ethical debates and breakthroughs in the lab, CRISPR has finally made its way to clinical trials. Researchers are now looking at whether the DNA-editing tool, as well as more conventional gene therapies, can effectively treat a wide array of heritable disorders and even cancers.

Theres been a convergence of the science getting better, the manufacturing getting much better, and money being available for these kinds of studies, says Cynthia Dunbar, a senior investigator at the National Heart, Lung, and Blood Institute. Its truly come of age.

CRISPR formally known as CRISPR-Cas9 has been touted as an improvement over conventional gene therapy because of its potential precision. CRISPR (clustered regularly interspaced short palindromic repeats) is a genetic code that, contained in a strand of RNA and paired with the enzyme Cas9, acts like molecular scissors that can target and snip out specific genes. Add a template for a healthy gene, and CRISPRs cut can allow the cell to replace a defective gene with a healthy one.

In April, scientists at the University of Pennsylvania announced they had begun using CRISPR for cancer treatments. The first two patients one with multiple myeloma, the other with sarcoma had cells from their immune systems removed. Researchers used CRISPR to genetically edit the cells in the lab, and then returned them back into their bodies.

On the other side of the country, Mark Walters, a blood and bone marrow transplant specialist at the University of California, San Francisco, Benioff Childrens Hospital in Oakland, is gearing up for trials that will use CRISPR to repair the defective gene that causes sickle cell disease. With CRISPR, once youve made that type of correction, [that cell] is 100 percent healthy, says Walters.

Another team is tackling the same disease using a type of hemoglobin, a protein in red blood cells, thats normally made only in fetuses and newborn babies. Researchers found that some adults continue to produce these proteins throughout their lives, and when those adults also have sickle cell disease, their symptoms are mild. So the international team used CRISPR to disable the gene that interferes with production of this hemoglobin, resuming its production and protecting the adult patients against sickle cell disease.

Several other CRISPR studies are in the works to treat a range of inherited disorders, including hemophilia and SCID-X1 (also known as X-linked severe combined immunodeficiency, the so-called bubble boy disease in which babies are born without a functioning immune system).

At St. Jude Childrens Research Hospital, a gene therapy trial cured Gael Jesus Pino Alva (pictured with his mother, Giannina) of SCID-X1, the bubble boy disease. (Credit: St. Jude Children's Research Hospital/Peter Barta)

The past year also saw success in a handful of experiments on conventional gene therapy. Instead of using CRISPR to repair disease-causing genes, these treatments use hollowed-out viruses to ferry healthy versions of genes into cells. Millions of these altered cells are released into the bloodstream or bone marrow in hopes that enough will land in the right places. But because scientists cant predict where the circulating genes may end up, this shotgun approach has had unintended, sometimes fatal, consequences including, in an earlier study, inadvertently activating leukemia-causing genes in patients treated for SCID-X1.

But in 2019, researchers learned that using a different type of virus one related to HIV to transport the genes may prevent these side effects. In an April study, researchers at St. Jude Childrens Research Hospital in Memphis, Tennessee, and UCSF Benioff Childrens Hospital in Oakland collected bone marrow from eight newborns with SCID-X1. They loaded corrective genes into the disabled HIV-related virus, which carried them into the patients bone marrow stem cells. The infants also received low doses of busulfan, a chemotherapy that gave the doctored stem cells room to grow. So far, we havent seen anything worrisome, says Ewelina Mamcarz, a pediatric oncologist at St. Jude who led the research team. The study recently added its 12th patient.

Gene therapy does have its momentum [back], says Mamcarz, reflecting on the fields setback after the earlier studys leukemia side effects. Theres so much that still needs to be done, and so many questions, she says. [But] this is how medicine evolves. We always want to be better than we were a week ago.

In the future, the hope is that gene therapy technologies will move beyond mending simple genetic mistakes and be used to combat big killers like diabetes or heart disease. [Those diseases are] more challenging, but a lot of them would benefit from knocking out a bad gene, says Dunbar.

For now, though, researchers are optimistic about the progress thats already been made. All of this has been very encouraging, says Dunbar. [And] for sickle cell in the U.S. and hemophilia in the developed world, these diseases may soon be solved.

[This story originally appeared in print as "Gene Therapy Gets Clinical."]

Originally posted here:
Gene Therapies Make it to Clinical Trials - Discover Magazine

For decades, the DNA of living organisms such as corn and salmon has been modified, but Crispr, invented in 2012, made gene editing more widely accessible. Image: YinYang/IStock.com via AFP Relaxnews

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering by editing her genome.

Victoria Grays recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research gene therapy.

I have hoped for a cure since I was about 11, the 34-year-old told AFP in an email.

Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency.

Over several weeks, Grays blood was drawn so doctors could get to the cause of her illness stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 pronounced Crisper a new tool informally known as molecular scissors.

The genetically edited cells were transfused back into Grays veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

This is one patient. This is early results. We need to see how it works out in other patients, said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

But these results are really exciting.

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

Its all developing very quickly, said French geneticist Emmanuelle Charpentier, one of Crisprs inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Cures

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practicing the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not such as making normal red blood cells, in Victorias case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the United States and a blood disease in the European Union.

They join several other gene therapies bringing the total to eight approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

Twenty-five, 30 years, thats the time it had to take, he told AFP from Paris.

It took a generation for gene therapy to become a reality. Now, its only going to go faster.

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a breakthrough period.

We have hit an inflection point, said Carrie Wolinetz, NIHs associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion and fighting a general infection.

You cannot do this in a community hospital close to home, said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Bioterrorism

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who dont necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal and his excommunication from the scientific community when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

That technology is not safe, said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr scissors often cut next to the targeted gene, causing unexpected mutations.

Its very easy to do if you dont care about the consequences, Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesnt believe in the more dystopian scenarios predicted for gene therapy, including American biohackers injecting themselves with Crispr technology bought online.

Not everyone is a biologist or scientist, she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies crops?

Charpentier thinks that technology generally tends to be used for the better.

Im a bacteriologist weve been talking about bioterrorism for years, she said. Nothing has ever happened.IB/JB

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2019: The year gene therapy came of age - INQUIRER.net

"If it disagrees with experiment, it's wrong. That's all there is to it." So said famed physicist Richard Feynman at a lecture about the scientific method at Cornell University in 1964.

Feynman appears to be only half correct, though. Yes, one's proposed theory is wrong if it doesn't agree with experiment. But that's not all there is to it. With carelessness or outright fraud, you can make it seem that your theory is correct and get it published in a top scientific journal.

Usually, such deception is eventually discovered. This past year was rich in scientific retractions of papers filled with poor processes and, in many cases, blatant fabrications. Here are five from 2019 that made the news in part because they mislead and provide false hope.

God created the Earth 6,000 years ago, according to many Christian creationists. And on the sixth day of creation, God made three species of timber vole with ribonucleotides that would come to demonstrate the shortcomings of the theory of evolution, according to a 1989 paper in the International Journal of Neuroscience.

Russian scientist Dmitrii Kuznetsov, the author of this paper, claimed that each of these three very closely related voles have ribonucleotides enzymes that are the building blocks of DNA and thus DNA repair that are utterly incompatible across the three species. This finding supports "the general creationist concept on the problems of the origin of boundless multitudes of different and harmonically functioning forms of life," Kuznetsov wrote in the paper.

But did Kuznetsov break the commandment about bearing false witness? Swedish biologist Dan Larhammar, who in 2018 became president of the Royal Swedish Academy of Sciences, questioned Kuznetsov's findings in a letter to the journal published way back in 1994. As reported in The Scientist in November 2019, Larhammar claimed that the results were superficially demonstrated and that many of the references couldn't be verified, even after he contacted scientists cited in the paper.

The International Journal of Neuroscience agreed with Larhammar and retracted the paper, albeit 30 years later. Kuznetsov has been accused multiple times of scientific misconduct, including for his analysis of the Shroud of Turin, which scholars claim originated in the Middle Ages but which Kuznetsov suggested could be the 2,000-year-old death shroud of Jesus.

Why the 30-year delay for a retraction? Thirty years in a 6,000-year-old Earth would be equivalent to 20 million years in a 4-billion-year-old Earth. Maybe the journal was hesitant to retype the original title, "In Vitro Studies of Interactions Between Frequent and Unique Mrnas and Cytoplasmic Factors from Brain Tissue of Several Species of Wild Timber Voles of Northern Eurasia, Clethrionomys Glareolus, Clethrionomys Frater and Clethrionomys Gapperi: A New Criticism to a Modern Molecular-Genetic Concept of Biological Evolution."

The vaccine against the human papillomavirus (HPV) has the potential to eliminate most cases of cervical cancer worldwide and save millions of lives. The HPV vaccine can also prevent the majority of vaginal, anal and penile cancers. But that's only if parents vaccinate their children against HPV.

A growing number are opting out over fears that the HPV vaccine is harmful. In Japan, for example, HPV vaccination rates fell from about 70 percent to 1 percent, its current level, in just a few years after unfounded reports of vaccine side effects, according to research published this year in the journal Expert Review of Vaccines.

As such, vaccine proponents are skeptical of any new study purporting problems with the HPV vaccine. Gayle DeLong, an associate professor of economics and finance at Baruch College in New York, learned that quickly. In 2018, she published a paper in the Journal of Toxicology and Environmental Health, Part A, in which she reported a link between the HPV vaccine and infertility. Married women between ages 25 and 29 who had received the HPV vaccine were less likely to have conceived compared with married women who didn't receive the vaccine, DeLong found.

The finding was promoted within anti-vaccination circles, but the study had multiple statistical shortcomings, such as not controlling for birth-control use. Moreover, those women who received the vaccine had a higher educational level. So, it could be that college-educated women who had received the vaccine were delaying childbirth until after age 30, as is the U.S. trend.

The journal retracted the paper in December 2019, noting "serious flaws in the statistical analysis and interpretation of the data in this paper." The World Health Organization has placed the HPV vaccine on its list of essential medicines, right up there with penicillin and acetaminophen, as a sign of its safety and efficacy.

On Nov. 13, 2019, Cao Xuetao, one of China's most prominent scientists, spoke to his fellow countrymen from the Great Hall of People in Beijing about research integrity. Some 6,000 people were in attendance, and the speech was live-streamed to 800,000 college students across the vast nation, mandatory viewing for most.

The topic was a contentious one. Just a year prior, the Chinese Ministry of Science and Technology (MOST) and several other agencies had promulgated a series of punitive measures to be used in cases of scientific misconduct, a sign that the Chinese government was considering the matter seriously. This had come in the wake of numerous scientific scandals in China, such as the retraction of more than 100 papers in 2017 over faked peer review and data manipulation.

Cao is a former president of the Chinese Academy of Medical Sciences, current president of the prestigious Nankai University, leader of several labs and chief research integrity officer for all Chinese research. His accolades are many. But now, Cao's actions are drawing close scrutiny, as he has been accused of scientific misconduct.

As reported on Nov. 22 in the journal Science, a multitude of Cao's papers appear to have doctored images. Science sleuth Elisabeth Bik, based in San Francisco, noticed that several images from a 2009 paper, in particular, looked like repeats. Bik has outed many scientists for data manipulation. Cao's body of work was soon scrutinized; they found examples of charts and images appearing to be repeated and manipulated in dozens of papers, which soon may be retracted.

Cao pledged to look into the matter. As noted, he's the leader of several labs and has a full-time gig as a university president, and he likely relies on postdoctoral fellows and graduate students to conduct actual research. And they likely want to please the boss with superficially good results. The same would apply to other elite scientists in China, which means the problem of scientific misconduct might be difficult to root out.

The cancer research community was ecstatic over a study published in the journal Nature in September 2018 that described a homing system to deliver the powerful anti-cancer chimeric antigen receptor (CAR) T cell therapy to brain cancer cells, which have long been out of reach to drug therapies.

But the researchers who conducted the study, from Baylor College of Medicine in Texas, may not have crossed the bloodbrain barrier, after all, but rather the factfiction barrier.

Within a few weeks of publication, other scientists began homing in on what may be widespread image manipulation. Nearly every image appeared to be fudged and not supportive of the underlying data, according to comments posted on an anonymous post-publication peer-review website called PubPeer.

The journal Nature investigated and retracted the paper in February 2019. The validity of this homing system remains in doubt. Some commenters on PubPeer noted that Nature should have spotted the image manipulation during the peer-review process. Software exists to detect it. It's either that or expect scientists to be honest.

He Jiankui has not been seen publicly since January 2019, just a few months after he infamously announced the birth of twin girls whose DNA was edited using CRISPR. His plan was to make the girls immune to HIV infection by modifying a gene known to offer some protection against the virus.

Seemingly proud of his achievement, He encountered swift worldwide condemnation not merely over the secrecy of the experiment but also for the possible harm that could have been done to the babies, whose genes were manipulated while in an embryonic state. CRISPR is an imperfect technique that can alter DNA in unknown and sometimes harmful ways, as animal studies have demonstrated.

The Chinese government, which may have supported He's efforts, has since suspended all of his research activities and, according to the New York Times, has kept him under guard.

Not much is known about He's procedure. Here's what is known: Scientists have stated that the basic premise of the work altering a gene called CCR5 to prevent HIV infection is shortsighted because this altered gene, found in nature, does not offer uniform HIV protection to those people who carry it. Moreover, the twins were given imperfect versions of this altered gene, and the health consequences are unknown, according to investigative work done by MIT Technology Review.

So, this was an experimental study otherwise suitable only for lab animals, medically unnecessary and poorly executed at that. There was a third gene-edited baby, too, perhaps born in the summer of 2019. Nothing is known of the baby's fate.

At issue is germline gene-editing on embryos. Gene alteration at this early stage ensures that all genetic modifications are copied into every cell in the body, including egg and sperm cells, making the changes inheritable. Otherwise, CRISPR and similar technologies continue to show great promise in curing genetic diseases in children and adults through more isolated and limited gene modification.

Originally published on Live Science.

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Take That Back: The Top Scientific Retractions of 2019 - Livescience.com

Gene Therapy for Ovarian Cancer Market research report 2019 gives detailed information of major players like manufacturers, suppliers, distributors, traders, customers, investors and etc. Gene Therapy for Ovarian Cancer Market Report presents a professional and deep analysis on the present state of Gene Therapy for Ovarian Cancer Market that Includes major types, major applications, Data type include capacity, production, market share, price, revenue, cost, gross, gross margin, growth rate, consumption, import, export and etc. Industry chain, manufacturing process, cost structure, marketing channel are also analysed in this report.

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Market Segment by Product Types IntravenousIntratumoralIntraperitoneal

Market Segment by Applications/End Users Ovarian Cancer (unspecified)Recurrent Ovarian Epithelial CancerPlatinum-Resistant Ovarian Cancer

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Gene Therapy for Ovarian Cancer Market Research Report Analysis And Forecasts To 2025 - Market Research Sheets

Global Gene Therapy for Age-related Macular Degeneration Market 2019 by key players, regions, type, and application, forecast to 2025. Gene Therapy for Age-related Macular Degeneration Market Report contains a forecast of 2019 and ending 2025 with a host of metrics like supply-demand ratio, Gene Therapy for Age-related Macular Degeneration Market frequency, dominant players of Gene Therapy for Age-related Macular Degeneration Market, driving factors, restraints, and challenges. The report also contains market revenue, sales, Gene Therapy for Age-related Macular Degeneration production and manufacturing cost that could help you get a better view of the market. The report focuses on the key global Gene Therapy for Age-related Macular Degeneration manufacturers, to define, describe and analyze the sales volume, value, market competition landscape, market share, SWOT analysis and development plans in future years.

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Major Players included in this report are as follows RetroSense TherapeuticsREGENXBIOAGTC

Gene Therapy for Age-related Macular Degeneration Market can be segmented into Product Types as SubretinalIntravitrealUnspecified

Gene Therapy for Age-related Macular Degeneration Market can be segmented into Applications as MonotherapyCombination Therapy

Gene Therapy for Age-related Macular Degeneration Market: Regional analysis includes:Asia-Pacific (Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)Europe (Turkey, Germany, Russia UK, Italy, France, etc.)North America (United States, Mexico, and Canada.)South America (Brazil etc.)The Middle East and Africa (GCC Countries and Egypt.)

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Gene Therapy for Age-related Macular Degeneration Market Research Report Analysis And Forecasts To 2025 - Market Research Sheets

Shares of IVERIC bio (ISEE) have risen by 450% since its name change in April, where the company shed the name of its failed predecessor Ophthotech and refocused efforts on the ocular gene therapy space.

The turnaround has been a long time coming, starting in August 2017 after prior lead asset Fovista failed its phase 3 trial in combination with approved VEGF inhibitors in patients with wet AMD. The focus then shifted to the company's C5 inhibitor Zimura, which was in trials for multiple ocular disease indications. The company entered into various gene therapy pacts, including a global license deal with the University of Florida Research Foundation and the University of Pennsylvania to develop a novel adeno-associated virus gene therapy for the treatment of rhodopsin-mediated autosomal dominant retinitis pigmentosa (NASDAQ:RP). In October 2018, the company acquired privately-held Inception 4, a developer of retinal diseases therapies based on inhibiting a protein called high temperature requirement A serine peptidase 1 protein (HtrA1). In exchange, Ophthotech issued 5.2 million shares of common stock in addition to being on the hook for additional milestone payments. Separately, the company inked an exclusive option agreement with the University of Pennsylvania and University of Florida Research Foundation for novel AAV gene therapy products for the treatment of Best vitelliform macular dystrophy (Best disease), a rare inherited disorder characterized by the progressive degeneration of the retina.

Flash forward to the present, and a strong showing since December's secondary offering provides us the green flag we need to dig deeper. The company priced 6.25 million common shares at $4 per share along with pre-funded warrants to purchase 3.75 million shares at $3.999 per pre-funded warrant for gross proceeds of around $40 million (to my eyes looks like a sweetheart deal to key institutional holders). As the stock currently trades at $7.75, these investors in the secondary offering have done quite well for themselves so far.

Let's take a closer look to determine if this gene therapy pioneer in the ocular disease space is de-risked enough to make it as a "ROTY idea."

Chart

Figure 1: ISEE daily advanced chart (Source: Finviz)

When looking at charts, clarity often comes from taking a look at distinct time frames in order to determine important technical levels to get a feel for what's going on. In the above chart (daily advanced), we can see the stock jump in late October after a positive data readout for the phase 2b study of Zimura in patients with dry age-related macular degeneration (AMD) with geographic atrophy (GA). From there the stock hardly dipped after news of a secondary offering in early December and has experienced much appreciation in the weeks that followed (definitely a green flag). Despite the run-up, at first glance consolidation below the $8 level appears to be offering readers an opportunity to at least initiate a pilot position.

For readers interested in a brief but very insightful overview of the company's operations, management's presentation at Evercore ISI 2nd Annual HealthCONx Conference was a worthwhile listen for me.

CEO and President Glenn Sblendorio starts by stating that during the last three years they've been building a portfolio in inherited retinal diseases with therapeutics (Zimura) as well as a deep gene therapy portfolio. He reminds us that recently they reported positive topline data for Zimura in the first of two pivotal studies (hit its primary endpoint, good safety). Aside from GA secondary to dry AMD, they also are going after Stargardt Disease with Zimura (similar to wet AMD but in younger patients, finished enrollment and expecting data later in 2020). Interestingly enough, this is the largest study in Stargardt patients that has ever been done.

Figure 2: Pipeline (Source: corporate presentation)

As for the preclinical portfolio, the company's HtrA1 inhibitor will be developed for GA secondary to Dry AMD (Genentech recently started a phase 2 study on that same target).

As for the gene therapy portfolio, there are six programs along with some research work. Lead program is in RHO-adRP and should make its way into the clinic next year (11k patients in US and EU5). Best1 related retinal diseases is behind that asset, while the next three programs are in conjunction with the University of Massachusetts medical school using minigene vector (takes large functional protein that is too large to fit into existing AAV vectors, and making it into a smaller protein but keeping the functionality).

Going back to GA secondary to dry AMD, Sblendorio acknowledges the competitor running its own trial (Apellis Pharmaceuticals' APL-2 which binds to a pocket of C3, see Apellis' corporate presentation) and notes that the condition itself is still not well understood. As for pathogenesis, it's pointed out that the complement system is quite complex (over 40 different variables, three different pathways) and management believes both classic and alternative pathways are activated early in the disease of GA.

Figure 3: GA impact on functional vision in daily life (Source: corporate presentation)

As for a development plan, they are taking all comers as opposed to segmenting (could come later on). With Zimura the company is seeking to preserve upstream complement activation while hitting the downstream, as contrasted to Apellis' C3 inhibitor (local suppression). Management states hitting closer to C5 with Zimura could have safety advantages. As for other C5 inhibitors that have been tested in GA and issues experienced in the past, Alexion's C5 inhibitor was administered systemically and had difficulty getting through blood brain barrier (Zimura on the other hand is administered via intravitreal injection). As for Novartis' (NYSE:NVS) monoclonal antibody trial in 99 patients, low dose was inconclusive and higher dose was in seven patients but study was terminated,

As for recent data for Zimura in the phase 2b study, 286 patients were enrolled to three drug arms (1mg, 2mg, 4mg) and the two higher doses hit statistical significance with the primary endpoint (mean rate of change in GA over 12 months measured by fundus autofluorescence at baseline, month 6, and month 12). Dose response (not statistically significant) was observed and safety profile was "very good." Meetings with FDA after the data have resulted in management's plan to run one more trial (to get underway Q1 2020).

Figure 4: Primary efficacy endpoint achieved with 2 mg dose of Zimura versus sham (Source: corporate presentation)

As for Stargardt data next year, here the objective also is to show slowing of disease progression but using a longer time frame (18 month) as Stargardt progresses more slowly than GA.

As for the early stage gene therapy platform, management used a logical selection criteria by choosing programs that are validated and have compelling science. Additionally, these programs are addressing indications with high unmet medical need and are commercially viable. Stargardt and Usher have high prevalence, with the former having 62,000+ patients in the US+EU5 and for the latter 50,000+ patients in US + EU5.

On June 17 IVERIC bio announced that it entered an agreement with Catalent (CTLT) for production and manufacturing of GMP-grade adeno-associated virus (AAV) vector for its gene therapy product candidates, IC-100 for the treatment of rhodopsin-mediated autosomal dominant retinitis pigmentosa and IC-200 for the treatment of BEST1 related retinal diseases. The first of these is expected to enter the clinic in 2020 with IC-200 to follow 1H 2021.

Figure 5: IC-100 proof of concept in canine model shows preservation of retinal function (Source: corporate presentation)

On July 23 the company announced it had exercised an option to enter into an exclusive global license agreement with the University of Massachusetts Medical School for rights to develop and commercialize mutation independent novel AAV gene therapy product candidates for the treatment of Leber Congenital Amaurosis type 10 (LCA10) due to mutations to the CEP290 gene, the most common type of LCA. Apparently, the collaboration resulted in additional research data that supports the plans of moving forward. Separately, IVERIC bio also announced expansion of its gene therapy portfolio by entering into a sponsored research agreement with UMass Medical School and an exclusive option agreement for rights to develop and commercialize novel AAV gene therapy product candidates utilizing a mutation independent minigene therapy approach for the treatment of vision loss in USH2A-related inherited retinal diseases (IRDs).

Figure 6: MiniCEP290 LCA10 early proof-of-concept (Source: corporate presentation)

On Oct. 28 the company announced initial topline data confirming that C5 inhibitor Zimura met its prespecified primary endpoint in reducing the rate of geographic atrophy growth in patients with dry age-related macular degeneration in a randomized, controlled phase 2b clinical trial. Reduction in the mean rate of GA growth over 12 months was 27.38% (p-value = 0.0072) for the Zimura 2 mg group as contrasted to sham control and 27.81% (p-value = 0.0051) for the Zimura 4 mg group as compared to sham control. The drug candidate appeared well tolerated after 12 months of administration with no Zimura-related inflammation or Zimura-related discontinuations (no ocular serious adverse events and no cases of endophthalmitis either). Incidence of choroidal neovascularization (CNV) in the untreated fellow eye was 10 patients (3.5%), three patients (2.7%) in the sham control group, six patients (9.0%) in the Zimura 2 mg group, and eight patients (9.6%) in the Zimura 4 mg group. Most frequently reported ocular adverse events were related to the injection procedure. Keep in mind the significance of this data considering that these patients have no treatment options currently.

Improvement in pre-specified endpoints (mean change in best corrected visual acuity and mean change in low luminance best corrected visual acuity from baseline to month 12) were not as promising (even inferior to placebo in some cases). However, as management pointed out in their presentation above, BCVA improvement is more important in conditions such as wet AMD as opposed to GA where the goal is simply to avoid blindness. Keep in mind that patients in the trial continue to be treated through month 18, so further data collection and readout in 2020 also will prove an important catalyst.

On Oct. 29 the company announced appointment of Abraham Scaria, PhD, to the position of Chief Scientific Officer (served previously at Genzyme, Sanofi and most recently at Casebia Therapeutics, leading multiple ocular gene therapy programs). Dr. Scarias 25-plus years of experience in the gene therapy arena will certainly prove helpful as the company's early-stage programs make their way into the clinic. This was followed up with another appointment in late November in the form of Guangping Gao, PhD, as Chief Strategist, Gene Therapy (has 30 years of research experience in gene-based treatments and is current President of the American Society of Gene and Cell Therapy (ASGCT). Dr. Gao's contributions to the field should not be understated (published 267 research papers, six book chapters and holds 135 patents with 239 additional patent applications pending).

For the third quarter of 2019, the company reported cash and equivalents of $94.9 million compared to net loss of $14.4 million. Research and development expenses rose slightly to $10.4 million, while G&A came in at $4.7 million. At the time, management estimated operational runway into 1H 2021 (not including $40 million of gross proceeds from secondary offering).

As for future catalysts, next year we can expect additional follow up on phase 2b data for the GA program and initiation of enrollment in the second pivotal study in Q1 2020. Data for Zimura in Stargardt disease also is expected at some point in 2020.

Keep in mind that competitor Apellis Pharmaceuticals (APLS) continues to enroll two phase 3 studies (DERBY and OAKS) for GA patients and progress here will be important to monitor as well.

Figure 7: Competitor Apellis' APL-2 FILLY phase 2 data slowed GA growth as well, graph provides point of comparison of mid-stage results (Source: corporate presentation)

On the conference call, management estimated that total expected cost of the second pivotal Zimura trial in GA could range between $30 million to $40 million plus additional external CMC cost for process development validation ranging from $10 million to $20 million (hinted that a partnership is a potential option on the table as well). Regarding market size, AMD is considered the most common cause of visual loss in developed countries with projection of 196 million in 2020 and 288 million in 2040 worldwide. Prevalence of GA in 2020 is estimated to be 1.5 million patients in the US with incidence of around 159,000/year. There are no currently FDA or EMEA approved treatment options available for these patients.

As for institutional investors of note, in December Venrock Healthcare Partners acquired a 5.60% stake in the company. While there has been a history of insider sales, President and CEO Glenn Sblendorio has made a few purchases as well (in May, November and December).

As for management lineup, President and CEO Glenn Sblendorio served prior as president and chief financial officer at The Medicines Company (NASDAQ:MDCO) from 2006 to 2016 and before that as executive vice president/chief financial officer of Eyetech Pharmaceuticals (was sold to OSI Pharmaceuticals for over $900 million in cash and stock deal). Chief Financial Officer David Carroll also came over from the Medicines Company. Chief Clinical Operations Officer also served prior at Eytech Pharmaceuticals as Vice President and Senior Vice President of Clinical Research and Development (played a key role in the development and approval of Macugen for the treatment of wet age-related macular degeneration).

To conclude, there are several elements in an investment here that we look for in ROTY (multiple ways to win via Zimura readouts and progression of gene therapy pipeline, good cash position post secondary, experienced management team, a derisking data set in GA, etc). Looking at prior news flow, I wish I had discovered this one earlier in the year when gene therapy programs were in licensed or even entry post Zimura GA data in October. However, even at the present market capitalization of $340 million (EV of around $200 million if cash is backed out), valuation seems to be on the cheap side relative to prospects in GA with additional indications and gene therapy pipeline giving investors long-term optionality. On a note of caution, burden of proof is on the company in detailed data readout for Zimura as well as with longer follow up to show maintained treatment effect and confirm prior data in the second pivotal trial.

For readers who are interested in the story and have done their due diligence, ISEE is a Speculative Buy and I expect continued upside in 2020 as this is both a revaluation and catalyst idea.

Risks include disappointing data readouts in 2020 for GA and Stargardt, setbacks in the clinic, regulatory setbacks for gene therapy pipeline and competition in certain spaces they are going after. When I think of LCA10 indication (and Usher for that matter), ProQR Theraeputics' (PRQR) sepofarsen and QR-421a come to mind. Allergan and Editas Medicine (EDIT) also are going after LCA10 with a CRISPR-based genome editing medicine (AGN-151587). A former winner mentioned in ROTY and public articles, Nightstar Therapeutics which was acquired by Biogen, also has a highly intriguing Stargardt disease program as well as earlier-stage asset NSR-BEST1 for Best disease. Going back to lead indication of GA for Zimura, keep in mind that multiple attempts with C5 in the past have failed, so burden of proof is on the company to truly show that "this time is different." Keep in mind that competitor APL-2 showed treatment effect maintained through 18 months - if phase 3 data for APL-2 disappoints, it could have an outsized impact on share price as well.

A comparison in valuation to Apellis Pharmaceuticals would be premature and essentially apples to oranges, considering the larger company has a pivotal readout due in PNH where there is rationale for optimism for the prospects of APL-2 as an add-on therapy to Soliris. AIHA and C3G are other indications being explored as well.

Currently Apellis sports a market cap around 6x that of IVERIC bio. One would think that as a revaluation idea alone (in the absence of data catalysts) and given clear signs of accumulation in the smaller company's stock, a revaluation of the latter to $500 million-plus market capitalization would be appropriate.

As for downside cushion and elements of de-risking, as mentioned prior cash position accounts for about a third of market capitalization and GA data for Zimura provides some cushion as well. It would appear that investors purchasing shares presently are getting the gene therapy pipeline essentially for free.

For our purposes in ROTY, again I wish I'd been savvy enough to catch this one earlier when enterprise value was ridiculously cheap (I often try to enter positions early on before a run up has taken place). That said, IVERIC bio still represents an attractive revaluation opportunity and I'm very interested to follow up on an update later in 2020 as lead gene therapy asset IC-100 makes its way into the clinic (would like to see management deliver on timeline projections and let us know when to expect the initial data set). Also, I feel that I have little edge as far as determining how pivotal data for competitor Apellis Pharmaceuticals' GA program will affect shares of IVERIC bio (am optimistic on positive outcome, but a comparison of side effect profiles will be key to determining who has an advantage in addressing this attractive market opportunity).

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Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: Disclaimer: Commentary presented is NOT individualized investment advice. Opinions offered here are NOT personalized recommendations. Readers are expected to do their own due diligence or consult an investment professional if needed prior to making trades. Strategies discussed should not be mistaken for recommendations, and past performance may not be indicative of future results. Although I do my best to present factual research, I do not in any way guarantee the accuracy of the information I post. I reserve the right to make investment decisions on behalf of myself and affiliates regarding any security without notification except where it is required by law. Keep in mind that any opinion or position disclosed on this platform is subject to change at any moment as the thesis evolves. Investing in common stock can result in partial or total loss of capital. In other words, readers are expected to form their own trading plan, do their own research and take responsibility for their own actions. If they are not able or willing to do so, better to buy index funds or find a thoroughly vetted fee-only financial advisor to handle your account.

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IVERIC bio: Promising Data In GA, Valuation Still Cheap With Multiple Catalysts In 2020 - Seeking Alpha

Millions of new scientific research papers are published every year, shedding light on everything from the evolution of stars to the ongoing impacts of climate change to the health benefits (or determents) of coffee to the tendency of your cat to ignore you. With so much research coming out every year, it can be difficult to know what is significant, what is interesting but largely insignificant, and what is just plain bad science. But over the course of a decade, we can look back at some of the most important and awe-inspiring areas of research, often expressed in multiple findings and research papers that lead to a true proliferation of knowledge. Here are ten of the biggest strides made by scientists in the last ten years.

The human family tree expanded significantly in the past decade, with fossils of new hominin species discovered in Africa and the Philippines. The decade began with the discovery and identification of Australopithecus sediba, a hominin species that lived nearly two million years ago in present-day South Africa. Matthew Berger, the son of paleoanthropologist Lee Berger, stumbled upon the first fossil of the species, a right clavicle, in 2008, when he was only 9 years old. A team then unearthed more fossils from the individual, a young boy, including a well-preserved skull, and A. sediba was described by Lee Berger and colleagues in 2010. The species represents a transitionary phase between the genus Australopithecus and the genus Homo, with some traits of the older primate group but a style of walking that resembled modern humans.

Also discovered in South Africa by a team led by Berger, Homo naledi lived much more recently, some 335,000 to 236,000 years ago, meaning it may have overlapped with our own species, Homo sapiens. The species, first discovered in the Rising Star Cave system in 2013 and described in 2015, also had a mix of primitive and modern features, such as a small brain case (about one-third the size of Homo sapiens) and a large body for the time, weighing approximately 100 pounds and standing up to five feet tall. The smaller Homo luzonensis (three to four feet tall) lived in the Philippines some 50,000 to 67,000 years ago, overlapping with several species of hominin. The first H. luzonensis fossils were originally identified as Homo sapiens, but a 2019 analysis determined that the bones belonged to an entirely unknown species.

These three major finds in the last ten years suggest that the bones of more species of ancient human relatives are likely hidden in the caves and sediment deposits of the world, waiting to be discovered.

When Albert Einstein first published the general theory of relativity in 1915, he likely couldnt have imagined that 100 years later, astronomers would test the theorys predictions with some of the most sophisticated instruments ever builtand the theory would pass each test. General relativity describes the universe as a fabric of space-time that is warped by large masses. Its this warping that causes gravity, rather than an internal property of mass as Isaac Newton thought.

One prediction of this model is that the acceleration of masses can cause ripples in space-time, or the propagation of gravitational waves. With a large enough mass, such as a black hole or a neutron star, these ripples may even be detected by astronomers on Earth. In September 2015, the LIGO and Virgo collaboration detected gravitational waves for the first time, propagating from a pair of merging black holes some 1.3 billion light-years away. Since then, the two instruments have detected several additional gravitational waves, including one from a two merging neutron stars.

Another prediction of general relativityone that Einstein himself famously doubtedis the existence of black holes at all, or points of gravitational collapse in space with infinite density and infinitesimal volume. These objects consume all matter and light that strays too close, creating a disk of superheated material falling into the black hole. In 2017, the Event Horizon Telescope collaborationa network of linked radio telescopes around the worldtook observations that would later result in the first image of the environment around a black hole, released in April 2019.

Scientists have been predicating the effects of burning coal and fossil fuels on the temperature of the planet for over 100 years. A 1912 issue of Popular Mechanics contains an article titled Remarkable Weather of 1911: The Effect of the Combustion of Coal on the ClimateWhat Scientists Predict for the Future, which has a caption that reads: The furnaces of the world are now burning about 2,000,000,000 tons of coal a year. When this is burned, uniting with oxygen, it adds about 7,000,000,000 tons of carbon dioxide to the atmosphere yearly. This tends to make the air a more effective blanket for the earth and to raise its temperature. The effect may be considerable in a few centuries.

Just one century later, and the effect is considerable indeed. Increased greenhouse gases in the atmosphere have produced hotter global temperatures, with the last five years (2014 to 2018) being the hottest years on record. 2016 was the hottest year since the National Oceanic and Atmospheric Administration (NOAA) started recording global temperature 139 years ago. The effects of this global change include more frequent and destructive wildfires, more common droughts, accelerating polar ice melt and increased storm surges. California is burning, Venice is flooding, urban heat deaths are on the rise, and countless coastal and island communities face an existential crisisnot to mention the ecological havoc wreaked by climate change, stifling the planets ability to pull carbon back out of the atmosphere.

In 2015, the United Nations Framework Convention on Climate Change (UNFCCC) reached a consensus on climate action, known as the Paris Agreement. The primary goal of the Paris Agreement is to limit global temperature increases to 1.5 degrees Celsius over pre-industrial levels. To achieve this goal, major societal transformations will be required, including replacing fossil fuels with clean energy such as wind, solar and nuclear; reforming agricultural practices to limit emissions and protect forested areas; and perhaps even building artificial means of pulling carbon dioxide out of the atmosphere.

Ever since the double-helix structure of DNA was revealed in the early 1950s, scientists have hypothesized about the possibility of artificially modifying DNA to change the functions of an organism. The first approved gene therapy trial occurred in 1990, when a four-year-old girl had her own white blood cells removed, augmented with the genes that produce an enzyme called adenosine deaminase (ADA), and then reinjected into her body to treat ADA deficiency, a genetic condition that hampers the immune systems ability to fight disease. The patients body began producing the ADA enzyme, but new white blood cells with the corrected gene were not produced, and she had to continue receiving injections.

Now, genetic engineering is more precise and available than ever before, thanks in large part to a new tool first used to modify eukaryotic cells (complex cells with a nucleus) in 2013: CRISPR-Cas9. The gene editing tool works by locating a targeted section of DNA and cutting out that section with the Cas9 enzyme. An optional third step involves replacing the deleted section of DNA with new genetic material. The technique can be used for a wide range of applications, from increasing the muscle mass of livestock, to producing resistant and fruitful crops, to treating diseases like cancer by removing a patients immune system cells, modifying them to better fight a disease, and reinjecting them into the patients body.

In late 2018, Chinese researchers led by He Jiankui announced that they had used CRISPR-Cas9 to genetically modify human embryos, which were then transferred to a womans uterus and resulted in the birth of twin girlsthe first gene-edited babies. The twins genomes were modified to make the girls more resistant to HIV, although the genetic alterations may have also resulted in unintended changes. The work was widely condemned by the scientific community as unethical and dangerous, revealing a need for stricter regulations for how these powerful new tools are used, particularly when it comes to changing the DNA of embryos and using those embryos to birth live children.

Spacecraft and telescopes have revealed a wealth of information about worlds beyond our own in the last decade. In 2015, the New Horizons probe made a close pass of Pluto, taking the first nearby observations of the dwarf planet and its moons. The spacecraft revealed a surprisingly dynamic and active world, with icy mountains reaching up to nearly 20,000 feet and shifting plains that are no more than 10 million years oldmeaning the geology is constantly changing. The fact that Plutowhich is an average of 3.7 billion miles from the sun, about 40 times the distance of Earthis so geologically active suggests that even cold, distant worlds could get enough energy to heat their interiors, possibly harboring subsurface liquid water or even life.

A bit closer to home, the Cassini spacecraft orbited Saturn for 13 years, ending its mission in September 2017 when NASA intentionally plunged the spacecraft into the atmosphere of Saturn so it would burn up rather than continue orbiting the planet once it had exhausted its fuel. During its mission, Cassini discovered the processes that feed Saturns rings, observed a global storm encircle the gas giant, mapped the large moon Titan and found some of the ingredients for life in the plumes of icy material erupting from the watery moon Enceladus. In 2016, a year before the end of the Cassini mission, the Juno spacecraft arrived at Jupiter, where it has been measuring the magnetic field and atmospheric dynamics of the largest planet in the solar system to help scientists understand how Jupiterand everything else around the sunoriginally formed.

In 2012, the Curiosity rover landed on Mars, where it has made several significant discoveries, including new evidence of past water on the red planet, the presence of organic molecules that could be related to life, and mysterious seasonal cycles of methane and oxygen that hint at a dynamic world beneath the surface. In 2018, the European Space Agency announced that ground-penetrating radar data from the Mars Express spacecraft provided strong evidence that a liquid reservoir of water exists underground near the Martian south pole.

Meanwhile, two space telescopes, Kepler and TESS, have discovered thousands of planets orbiting other stars. Kepler launched in 2009 and ended its mission in 2018, revealing mysterious and distant planets by measuring the decrease in light when they pass in front of their stars. These planets include hot Jupiters, which orbit close to their stars in just days or hours; mini Neptunes, which are between the size of Earth and Neptune and may be gas, liquid, solid or some combination; and super Earths, which are large rocky planets that astronomers hope to study for signs of life. TESS, which launched in 2018, continues the search as Keplers successor. The space telescope has already discovered hundreds of worlds, and it could find 10,000 or even 20,000 before the end of the mission.

The decade began with a revolution in paleontology as scientists got their first look at the true colors of dinosaurs. First, in January 2010, an analysis of melanosomesorganelles that contain pigmentsin the fossilized feathers of Sinosauropteryx, a dinosaur that lived in China some 120 to 125 million years ago, revealed that the prehistoric creature had reddish-brown tones and stripes along its tail. Shortly after, a full-body reconstruction revealed the colors of a small feathered dinosaur that lived some 160 million years ago, Anchiornis, which had black and white feathers on its body and a striking plume of red feathers on its head.

The study of fossilized pigments has continued to expose new information about prehistoric life, hinting at potential animal survival strategies by showing evidence of countershading and camouflage. In 2017, a remarkably well-preserved armored dinosaur which lived about 110 million years ago, Borealopelta, was found to have reddish-brown tones to help blend into the environment. This new ability to identify and study the colors of dinosaurs will continue to play an important role in paleontological research as scientists study the evolution of past life.

In November 2018, measurement scientists around the world voted to officially changed the definition of a kilogram, the fundamental unit of mass. Rather than basing the kilogram off of an objecta platinum-iridium alloy cylinder about the size of a golf ballthe new definition uses a constant of nature to set the unit of mass. The change replaced the last physical artifact used to define a unit of measure. (The meter bar was replaced in 1960 by a specific number of wavelengths of radiation from krypton, for example, and later updated to define a meter according to the distance light travels in a tiny fraction of a second.)

By using a sophisticated weighing machine known as a Kibble balance, scientists were able to precisely measure a kilogram according to the electromagnetic force required to hold it up. This electric measurement could then be expressed in terms of Plancks constant, a number originally used by Max Planck to calculate bundles of energy coming from stars.

The kilogram was not the only unit of measure that was recently redefined. The changes to the International System of Units, which officially went into effect in May 2019, also changed the definition for the ampere, the standard unit of electric current; the kelvin unit of temperature; and the mole, a unit of amount of substance used in chemistry. The changes to the kilogram and other units will allow more precise measurements for small amounts of material, such as pharmaceuticals, as well as give scientists around the world access to the fundamental units, rather than defining them according to objects that must be replicated and calibrated by a small number of labs.

In 2010, scientists gained a new tool to study the ancient past and the people who inhabited it. Researchers used a hair preserved in permafrost to sequence the genome of a man who lived some 4,000 years ago in what is now Greenland, revealing the physical traits and even the blood type of a member of one of the first cultures to settle in that part of the world. The first nearly complete reconstruction of a genome from ancient DNA opened the door for anthropologists and geneticists to learn more about the cultures of the distant past than ever before.

Extracting ancient DNA is a major challenge. Even if genetic material such as hair or skin is preserved, it is often contaminated with the DNA of microbes from the environment, so sophisticated sequencing techniques must be used to isolate the ancient humans DNA. More recently, scientists have used the petrous bone of the skull, a highly dense bone near the ear, to extract ancient DNA.

Thousands of ancient human genomes have been sequenced since the first success in 2010, revealing new details about the rise and fall of lost civilizations and the migrations of people around the globe. Studying ancient genomes has identified multiple waves of migration back and forth across the frozen Bering land bridge between Siberia and Alaska between 5,000 and 15,000 years ago. Recently, the genome of a young girl in modern Denmark was sequenced from a 5,700-year-old piece of birch tar used as chewing gum, which also contained her mouth microbes and bits of food from one of her last meals.

This decade included the worst outbreak of Ebola virus diseases in history. The epidemic is believed to have begun with a single case of an 18-month-old-boy in Guinea infected by bats in December 2013. The disease quickly spread to neighboring countries, reaching the capitals of Liberia and Sierra Leone by July 2014, providing an unprecedented opportunity for the transmission of the disease to a large number of people. Ebola virus compromises the immune system and can cause massive hemorrhaging and multiple organ failure. Two and a half years after the initial case, more than 28,600 people had been infected, resulting in at least 11,325 deaths, according to the CDC.

The epidemic prompted health officials to redouble their efforts to find an effective vaccine to fight Ebola. A vaccine known as Ervebo, made by the pharmaceutical company Merck, was tested in a clinical trial in Guinea performed toward the end of the outbreak in 2016 that proved the vaccine effective. Another Ebola outbreak was declared in the Democratic Republic of the Congo in August 2018, and the ongoing epidemic has spread to become the deadliest since the West Africa outbreak, with 3,366 reported cases and 2,227 deaths as of December 2019. Ervebo has been used in the DRC to fight the outbreak on an expanded access or compassionate use basis. In November 2019, Ervebo was approved by the European Medicines Agency (EMA), and a month later it was approved in the U.S. by the FDA.

In addition to a preventative vaccine, researchers have been seeking a cure for Ebola in patients who have already been infected by the disease. Two treatments, which involve a one-time delivery of antibodies to prevent Ebola from infecting a patients cells, have recently shown promise in a clinical trial in the DRC. With a combination of vaccines and therapeutic treatments, healthcare officials hope to one day eradicate the viral infection for good.

Over the past several decades, physicists have worked tirelessly to model the workings of the universe, developing what is known as the Standard Model. This model describes four basic interactions of matter, known as the fundamental forces. Two are familiar in everyday life: the gravitational force and the electromagnetic force. The other two, however, only exert their influence inside the nuclei of atoms: the strong nuclear force and the weak nuclear force.

Part of the Standard Model says that there is a universal quantum field that interacts with particles, giving them their masses. In the 1960s, theoretical physicists including Franois Englert and Peter Higgs described this field and its role in the Standard Model. It became known as the Higgs field, and according to the laws of quantum mechanics, all such fundamental fields should have an associated particle, which came to be known as the Higgs boson.

Decades later, in 2012, two teams using the Large Hadron Collider at CERN to conduct particle collisions reported the detection of a particle with the predicted mass of the Higgs boson, providing substantial evidence for the existence of the Higgs field and Higgs boson. In 2013, the Nobel Prize in Physics was awarded to Englert and Higgs for the theoretical discovery of a mechanism that contributes to our understanding of the origin of mass of subatomic particles, and which recently was confirmed through the discovery of the predicted fundamental particle. As physicists continue to refine the Standard Model, the function and discovery of the Higgs boson will remain a fundamental part of how all matter gets its mass, and therefore, how any matter exists at all.

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The Top Ten Scientific Discoveries of the Decade - Smithsonian

NEW YORK, Dec. 31, 2019 /PRNewswire/ --

Global Heart Transplantation Therapeutics Market: About this market This heart transplantation therapeutics market analysis considers sales from immunosuppressants and supplementary medications products. Our study also finds the sales of heart transplantation therapeutics in Asia, Europe, North America, and ROW. In 2019, the immunosuppressants segment had a significant market share, and this trend is expected to continue over the forecast period. Factors such as mechanism of actions (MoA) of these drugs to treat and prevent organ rejection will play a significant role in the immunosuppressants segment to maintain its market position. Also, our global heart transplantation therapeutics market report looks at factors such as rising prevalence of cardiovascular diseases and heart failures, availability of drugs in multiple dosage forms, and high dependency of immunosuppressants for heart transplantation. However, adverse effects of immunosuppressants, demand-supply imbalance for organ transplantation, and long-term complications associated with heart transplantation may hamper the growth of the heart transplantation therapeutics industry over the forecast period.

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Global Heart Transplantation Therapeutics Market: Overview High dependency on immunosuppressants for heart transplantation Various advances in surgical techniques, especially organ transplants and the prevalence of severe coronary artery disease and dilated cardiomyopathy are leading to an increase in the need for heart transplantation surgeries. However, heart transplantation can lead to several types of heart transplant rejection such as acute cellular rejection, acute antibody rejection, and coronary artery vasculopathy chronic rejection. This is driving the need for heart transplantation therapeutics to prevent transplant rejection. Immunosuppressants are used to prevsent transplant rejection caused by immune system. Thus, the high dependency on immunosuppressants for heart transplantation will lead to the expansion of the global heart transplantation therapeutics market at a CAGR of almost 3% during the forecast period. Advent of techniques for organ transplantation The number of heart transplantation procedures is substantially high, especially in developed countries, due to the high prevalence of risk factors for heart impairment. Currently, the market is witnessing rapid development in the field of regenerative therapies, such as cell therapy, gene therapy, and tissue engineering, which also involve the development of heart and/or other organs. The introduction of novel approaches for heart regeneration and transplantation is one of the key heart transplantation therapeutics market trends. Organ printing is a modern technology, which is a layer-by-layer additive robotic bio-fabrication of 3D functional living macro tissues and organ constructs using tissue spheroids as building blocks. This development is expected to have a positive impact on the overall market growth.

Competitive Landscape With the presence of several major players, the global heart transplantation therapeutics market is fragmented. This robust vendor analysis is designed to help clients improve their market position, and in line with this, this report provides a detailed analysis of several leading heart transplantation therapeutics manufacturers, that include AbbVie Inc., Astellas Pharma Inc., Cadila Healthcare Ltd., F. Hoffmann-La Roche Ltd., Mylan NV, Novartis AG, Pfizer Inc., Sun Pharmaceutical Industries Ltd., Teva Pharmaceutical Industries Ltd., and Veloxis Pharmaceuticals A/S. Also, the heart transplantation therapeutics market analysis report includes information on upcoming trends and challenges that will influence market growth. This is to help companies strategize and leverage on all forthcoming growth opportunities.

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The global heart transplantation therapeutics market at a CAGR of almost 3% during the forecast period - PRNewswire

For all the flak the pharmaceutical industry has taken for its exorbitant pricing practices, there's no getting around the fact that it's been a pretty stunning decade for medical progress.

Multiple new categories of medicines have moved from dreams and lab benches into the market and peoples lives, and investors who came along for the ride often reaped extraordinary profits. The Nasdaq Biotech Index is up 360% over the last 10 years to the S&P 500's 190%. And thats without mentioning the hundreds of billions of dollars in takeovers that rewarded shareholders with windfalls.

As 2020 approaches, it's worth highlighting how far we've come in the past 10 years in developing new therapies and approaches to treating disease, even as politicians grapple with how to rein in health-care costs without breaking an ecosystem that incentivizes the search for new discoveries. Here are some of the decades biggest medical breakthroughs:

Cell therapies: First approved in the U.S. two years ago, these treatments still sound like science fiction. Drugmakers harvest immune cells from patients, engineer them to hunt tumors, grow them by the millions into a living drug, and reinfuse them. Yescarta from Gilead Siences Inc. and Novartis AGs Kymriah the two treatments approved so far can put patients with deadly blood cancers into remission in some cases. At the beginning of the decade, academics were just beginning early patient tests.

Its still early days for the technology, and some issues are holding these drugs back. There are significant side effects, and the bespoke manufacturing process is expensive and time-consuming. That has contributed to a bruising price tag: Both of the approved medicines cost over $350,000 for a single treatment. And for now, cell therapy is mostly limited to very sick patients who have exhausted all other alternatives.

Luckily, more options are on their way. Some drugmakers are focused on different types of blood cancers. Others hope to mitigate side effects or create treatments that can be grown from donor cells to reduce expenses and speed up treatment. In the longer run, companies are targeting trickier solid tumors. Scientists wouldn't be looking so far into the future without this decades extraordinary progress.

Gene therapies: Researchers have spent years trying to figure out how to replace faulty DNA to cure genetic diseases, potentially with as little as one treatment. Scientific slip-ups and safety issues derailed a wave of initial excitement about these therapies starting in the 1990s; the first two such treatments to be approved in Europe turned out to be commercial flops.

This decade, the technology has come of age. Luxturna, a treatment developed by Spark Therapeutics Inc. for a rare eye disease, became the first gene therapy to get U.S. approval in late 2017. Then in May came the approval of Novartis AGs Zolgensma for a deadly muscle-wasting disease. The drugs have the potential to stave off blindness and death or significant disability with a single dose, and, unsurprisingly, Big Pharma has given them a substantial financial endorsement. Roche Holding AG paid $4.7 billion to acquire Spark this year, while Novartis spent $8.7 billion in 2018 to buy Zolgensma developer Avexis Inc.

Dozens of additional therapies are in development for a variety of other conditions and should hit the market in the next few years. They offer the tantalizing potential not just to cure diseases, but to replace years of wildly expensive alternative treatment. If drugmakers can resist the temptation to squeeze out every ounce of value by doing things like charging $2.1 million for Zolgensma, theres potential for these treatments to save both lives and money.

RNA revolution: The above treatments modify DNA; this group uses the bodys messaging system to turn a patients cells into a drug factory or interrupt a harmful process. Two scientists won a Nobel Prize in 2006 for discoveries related to RNA interference (RNAi), one approach to making this type of drug, showing its potential to treat difficult diseases. That prompted an enormous amount of hype and investment, but a series of clinical failures and safety issues led large drugmakers to give up on the approach. Sticking with it into this decade paid off.

Alnylam Inc. has been working since 2002 to figure out the thorny problems plaguing this class of treatments. It brought two RNAi drugs for rare diseases to the market in the past two years and has more on the way. The technology is also moving from small markets to larger ones: Novartis just paid $9.7 billion to acquire Medicines Co. for its Alnylam-developed drug that can substantially lower cholesterol with two annual treatments.

Ionis Pharmaceuticals Inc. and Biogen Inc. collaborated on Spinraza, a so-called antisense drug that became the first effective treatment for a deadly rare disease. It was approved in late 2016 and had one of the most impressive drug launches of the decade. And Moderna Therapeutics rode a wave of promising messenger RNA-based medicines to the most lucrative biotechnology IPO of all time in 2018. From pharma abandonment to multiple approvals and blockbuster sales potential in under 10 years. Not bad!

Cancer immunotherapy: Scientists had been working on ways to unleash the human immune system on cancers well before the 2010s without much luck. Checkpoint inhibitors drugs that release the brakes on the body's defense mechanisms have since produced outstanding results in a variety of cancers and are the decades most lucrative turnaround story.

Merck got a hold of Keytruda via its 2009 acquisition of Schering-Plough, but it was far from the focus of that deal. Once Bristol-Myers Squibb & Co. produced promising results for its similar drug, Opdivo, Merck started a smart development plan that has turned Keytruda into the worlds most valuable cancer medicine. Its now available to treat more than 10 types of the disease, and has five direct competitors in the U.S. alone. Analysts expect the category to exceed $25 billion in sales next year.

If anything, the drugs may have been too successful. Copycat efforts are pulling money that could fund more innovative research. There are thousands of trials underway attempting to extend the reach of these medicines by combining them with other drugs. Some are based more on wishful thinking than firm scientific footing. Still, the ability to shrink some previously intractable tumors is a considerable advance. If drugmakers finally figure out the right combinations and competition creates pricing pressure that boosts access, these medicines will do even more in the years to come.

Conquering hepatitis C: From a combined economic and public-health standpoint, a new group of highly effective hepatitis C medicines may outstrip just about anything else on this list so far. Cure rates for earlier treatments werent especially high; they took some time to work and had nasty side effects. The approval of Gileads Sovaldi in 2013, followed in time by successor drugs such as AbbVie Inc.s Mavyret, have made hepatitis C pretty easily curable in a matter of weeks. For Gilead, getting to market rapidly with its drug proved enormously profitable; it raked in over $40 billion in revenue in just three years.

Hepatitis C causes liver damage over time that can lead to transplants or cancer. The existence of a rapid cure is a significant long-term boon even if the initial pricing on the drugs made them, in some cases, prohibitively expensive. Sovaldi notoriously cost $1,000 per pill at launch and over $80,000 for a course of treatment. The good new is, treatments have become a lot more affordable, which should allow this class of drugs to have a broad and lasting positive health impact.

Hepatitis C is one of the relatively few markets where the drug-pricing system has worked well. As competing medicines hit the market, the effective cost of these treatments plummeted. That, in turn, made the drugs more accessible to state Medicaid programs and prison systems, which operate on tight budgets and care for populations with higher rates of hepatitis C infection. Louisiana has pioneered the use of a Netflix model, under which the state paid an upfront fee for unlimited access to the drug. Its an arrangement that will help cure thousands of patients, and other states are likely to follow its lead.

Many of the medicines highlighted in this column have list prices in the six figures, a trend thats helped drive up Americas drug spending by more than $100 billion since 2009. Building on this decades medical advances is going to lead to even more effective medicines that will likely come with steeper prices. Id like to hope that policymakers will come up with a solution that better balances the need to reward innovation with the need to keep medicines accessible. That would really be a breakthrough.

Max Nisen at mnisen@bloomberg.net

@2019Bloomberg

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Gene therapy to conquering hepatitis C: A decade of medical breakthroughs - Business Standard

As Newtons famous quote, standing on the shoulders of giants, this year, science has made considerable advances, building on many feats achieved in the past years. New discoveries, insights and inventions in the areas of astronomy, biology, medicine, paleontology and physics marked the year. Here is a selected pick of ten such breakthroughs in science witnessed in 2019.

1. Detailing the Denisovans

An early sketch of a Denisovan teen [Image Credits: Maayen Harel]

This year revealed some fantastic facts about our ancient ancestors, the Denisovans, who lived about 100,000 years ago. So far, we knew about them through scrap fossils from the Denisova Cave in Siberia, Russia. This year, researchers found a fossilised jawbone in the Tibetian plateau, which on DNA analysis showed that it belonged to the Denisovans, who were the regions first hominin inhabitants. It was also believed earlier that Denisovans were closely related to Neanderthals than to present-day humans. On the contrary, genomic analysis of the fossils from the Denisova Cave showed that they were closer to humans than to Neanderthals. But, how did our ancestors look like? Based on patterns of chemical changes in their DNA, researchers have reconstructed the anatomy of Denisovans. The findings reveal that some traits, like a sloping forehead, long face and large pelvis resemble Neanderthals, while others, like a large dental arch and very wide skull, are unique. Based on these findings, they even reconstructed the face of a teenage Denisovan girl.

2. An elusive cure to Ebola

Electron micrograph of an Ebola virus virion [Image Credits: CDC/Cynthia Goldsmith]

Ebola, a deadly viral disease that shook the African continent, affects humans and other primates and a cure for this disease has eluded science so far. Although an experimental vaccine is being developed, without a therapeutic cure, those infected are doomed to die. This year, two drugs that were tested during an outbreak in the Democratic Republic of the Congo may have hopes as they dramatically increased patients chances of survival. The two drugs, named REGN-EB3 and mAb-114, contain a cocktail of antibodies that are injected into the bloodstream of those infected. These drugs have shown a success rate of about 90% , bringing hopes to those battered by the disease.

3. The first image of a blackhole

The first captured image of a black hole [Image Credit: Event Horizon Telescope Collaboration]

Black holes, the most dense objects of our Universe, have been awe-inspiring for a century. However, we did not even know how they lookedbut all that changed this year. Scientists used a combination of telescope observations around the globe to reveal the first ever photograph of a supermassive black hole present at the heart of the distant galaxy Messier 87 in the Virgo constellation. The image, which captures the shadow of the black hole, shows a black hole that is 55 million light-years from Earth and has a mass of 6.5 billion times that of the Sun. Researchers believe that this epic photograph opens a new window into the study of black holes, their event horizons, and gravity.

4. Conquering Quantum Computing

Photograph of the Sycamore processor. [Image Credits: Erik Lucero, Research Scientist and Lead Production Quantum Hardware, Google]

Although physicists have been working on realising the concept of quantum computing for over three decades, it wasnt until this year that there was something tangible. Physicists and Engineers at Google claim to have developed the first functional quantum computer that can perform a set of computations in 200 seconds, which would have otherwise taken the worlds fastest supercomputer 10,000 years! This quantum computer has a 54-qubit processor, named Sycamore, which is comprised of quantum logic gates.

5. Beating malnutrition in the gut

Escherichia coli, a common bacteria found in the human gut [Image Credits: Photo by Eric Erbe, digital colorization by Christopher Pooley, both of USDA, ARS, EMU]

While it was long known that microbes in our gut played a vital role in our health and well-being, two studies published during the year showed how they could be used to address malnutritiona condition that affects millions of children around the world. The researchers analysed the types of microbes present in the gut of healthy and malnourished children and focused on boosting crucial gut microbes in the children using affordable, culturally acceptable foods.

6. Pushing the limits of gene editing

The DNA Double Helix [Image Credits: Image by Arek Socha from Pixabay]

After tasting success and controversies last year for genetically editing babies, researchers in China this year reported to have cloned five genetically edited macaques for research purposes for the first time. These monkeys have reduced sleep, increased movements in the night, increased anxiety and depression, and schizophrenia-like behaviors. Although it raises ethical questions, the researchers believe that cloned monkeys could replace the wild monkeys used in laboratories today. In the UK, scientists used gene therapy to arrest a form of age-related blindness and in the US, CRISPR, the gene editing software, was used to treat cancer.

7. The rampant loss of worlds ice

Meltwater on the ice shelf next to McMurdo Station, Antarctica.[Image Credit: Nicholas Bayou, UNAVCO]

With the rising global temperature, ice on the Earths surface is melting at a rapid rate. In Greenland, the ice sheets are melting seven times faster than they did in the 90s. Greenland has lost 3.8 trillion tonnes of ice since 1992, a quantity - enough to push global sea levels up by 10.6 millimetres. In Antartica, studies have detected significant changes in the thickness of the floating ice shelves, which hold the land-based ice in place. As a result, there could be more ice moving from the land into the sea. Similar loss of ice has been reported in the Alps and the Himalayas. The rising sea levels are estimated to displace 300 million people all over the world, affecting coastal cities and their livelihoods.

8. Taking a closer look at the Moon

The far side of the moon that is invisible to Earth [ Image Credits: NASA Apollo 16 photograph AS16-3021]

This year, China's National Space Administration (CNSA) achieved the first soft landing on the far side of the Moon with its Chang'e 4 mission. This mission will attempt to determine the age and composition of an unexplored region of the Moon. India also launched its second lunar mission, Chandrayaan 2, to map and study the variations in the lunar surface composition, and the location and abundance of water. The mission consisted of an orbiter, the Vikram lunar lander and the Pragyan rover. However, Vikram crashed during landing, in its attempt to land closer to the lunar south pole.

9. Biodiversity on the brink of extinction

A frog from the Western Ghats

This year, an extensive report from the United Nations Intergovernmental Science-Policy Platform on Biodiversity and Ecosystem Services (IPBES) found that of the estimated 8 million species of animals and plants on the planet, about a million face the threat of extinction, many within decades. About 40% of amphibians, a third of marine life and about 10% of the insects are at the brink of extinction. The report mentions that changes in land and sea use, exploitation of organisms; climate change, pollution and invasive alien species as primary reasons behind this situation.

10. Chronicling the final moments of dinosaurs

Image by enriquelopezgarre from Pixabay

It is well known that the dinosaurs, giant reptiles that once ruled the planet, went extinct about 66 million years ago when an asteroid crashed into Earth at the Chicxulub crater in Mexico. This year, scientists detailed fallouts of the impact that resulted in a mass extinction by examining the topography of the centre of the crater. When the asteroid struck, the melt rocks and breccia deposited at the bottom of the crater within minutes and over a few hours, another 90 metres were deposited. There was also a tsunami and a wildfire that followed the impact, which emitted sulphur aerosols that cooled the earth and blocked much of the sunlight.

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The Best of Science in 2019 - Research Matters

Gene Therapy market analysis report speaks about the manufacturing process. The process is analysed thoroughly with four points Manufacturers, regional analysis, Segment by Type & Applications and the actual process of whole Gene Therapy industry.

A complete analysis of the competitive landscape of the Gene Therapy Market is provided in the report. This section includes company profiles of market key players. The profiles include contact information, gross, capacity, product details of each firm, price, and cost of Gene Therapy Industry are covered.

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Gene Therapy Market Description: Gene therapy is an experimental treatment that involves introducing genetic material into a persons cells to fight or prevent disease. Researchers are studying gene therapy for a number of diseases, such as severe combined immuno-deficiencies, hemophilia, Parkinsons disease, cancer and even HIV, through a number of different approaches. A gene can be delivered to a cell using a carrier known as a vector. The most common types of vectors used in gene therapy are viruses. The viruses used in gene therapy are altered to make them safe, although some risks still exist with gene therapy. The technology is still in its infancy

Gene Therapy Market Segment by Manufacturers, this report covers

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Today, Gene Therapy is used for Cancer Diseases, Hematological Disease and Hereditary Disease. Cancer Diseases is the largest application of Gene Therapy and its market share exceed 78% in 2018.The global Gene Therapy market is valued at xx million USD in 2018 and is expected to reach xx million USD by the end of 2024, growing at a CAGR of xx% between 2019 and 2024.The Asia-Pacific will occupy for more market share in following years, especially in China, also fast growing India and Southeast Asia regions.North America, especially The United States, will still play an important role which cannot be ignored. Any changes from United States might affect the development trend of Gene Therapy.Europe also play important roles in global market, with market size of xx million USD in 2019 and will be xx million USD in 2024, with a CAGR of xx%.This report studies the Gene Therapy market status and outlook of Global and major regions, from angles of players, countries, product types and end industries; this report analyzes the top players in global market, and splits the Gene Therapy market by product type and applications/end industries.

Gene Therapy Market Segment by Regions, regional analysis covers

Gene Therapy Market report provides application, type impact on market. Also research report covers the present scenario of Gene Therapy Market Consumption forecast, by regional market, type and application, with sales and revenue, from 2019 to 2024.

Gene Therapy Market Segment by Type, covers

Gene Therapy Market Segment by Applications, can be divided into

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Gene Therapy Market 2020 Business Revenue, Future Growth, Trends Plans, Top Key Players, Business Opportunities, Industry Share, Global Size Analysis...

Amyotrophic lateral sclerosis (ALS), sometimes called Lou Gehrigs disease, is a neurodegenerative disease affecting nerve cells in the brain and spinal cord. Researchers at the University of California San Diego School of Medicine published research describing a new way to deliver a gene-silencing vector to mice with ALS. The therapy resulted in long-term suppression of the disease if the treatment was given before the disease started. It also blocked disease progression in the mice if symptoms already appeared.

The study was published in the journal Nature Medicine.

At present, this therapeutic approach provides the most potent therapy ever demonstrated in mouse models of mutated SOD1 gene-linked ALS, said senior author Martin Marsala, professor in the Department of Anesthesiology at UC San Diego School of Medicine. In addition, effective spinal cord delivery of AAV9 vector in adult animals suggests that the use of this new delivery method will likely be effective in treatment of other hereditary forms of ALS or other spinal neurodegenerative disorders that require spinal parenchymal delivery of therapeutic gene(s) or mutated-gene silencing machinery, such as in C9orf72 gene mutation-linked ALS or in some forms of lysosomal storage disease.

ALS appears in two forms, sporadic and familial. The most common form is sporadic, responsible for 90 to 95% of all cases. Familial ALS makes up 5 to 10% of all cases in the U.S., and as the name suggests, is inherited. Studies have shown that a least 200 mutations of the SOD1 gene are linked to ALS.

In healthy individuals, the SOD1 gene provides instructions for an enzyme called superoxide dismutase. This enzyme is used to break down superoxide radicals, which are toxic oxygen molecules that are a byproduct of normal cellular processes. It is believed that the mutations in the gene cause ineffective removal of superoxide radicals or potentially cause other toxicities resulting in motor neuron cell death.

The new research involves injecting shRNA, an artificial RNA molecule that can turn off, or silence, a targeted gene. This delivers shRNA to cells by way of a harmless adeno-associated virus (AAV). In the research, they injected the viruses carrying shRNA into two locations in the spinal cord of adult mice expressing an ALS-causing mutation of the SOD1 gene. They were performed just before disease onset or after the laboratory animals started showing symptoms.

The researchers have tested the approach in adult pigs, whose have spinal cord dimensions closer to those in humans. They found that by using an injector developed for adult humans, the procedure could be performed without surgical complications and in a reliable fashion.

The next step will be more safety studies with a large animal model.

While no detectable side effects related to treatment were seen in mice more than one year after treatment, the definition of safety in large animal specimens more similar to humans is a critical step in advancing this treatment approach toward clinical testing, Marsala said.

About 5,000 people are diagnosed with ALS in the U.S. each year, with about 30,000 people living with the disease. There are symptomatic treatments, but no cure. Most patients die from the disease two to five years after diagnosis.

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Experimental Gene Therapy Shows Promise for Preventing and Treating Lou Gehrig's Disease in Mice - BioSpace

Q4 2019 has been the best quarter (IBB up 22.5% XBI up 26.7%) for biotech investors since Q1 2015, which was the top of 2014-2015 rally. This 20+% rally is largely attributed to Fed's overall easing stance, increased mergers & acquisitions, positive regulatory backdrop, solid trial results and better-than-expected corporate earnings. 2019 deal activity has increased significantly, and large Biopharma companies are actively conducting mergers and acquisitions in order to deliver higher long-term return for their investors.

Based on data from Chimera Research Group (Biotech M&A - 2019 Deals), there were 28 biotech M/A deals in 2019, comparing to only 16 last year. The accumulative deal value is $203.7B, which includes two mega deals: BMY buying Celgene for $74B and ABBV buying Allergan for $63B. In 2018, total deal value was only $48.2B. Going into 2020, I am optimistic about the high volume of biotech deal activity will continue.

According to SVB Leerink Research, large Biopharma acquirers currently have $225B dry powder available for M&A deals. While I don't expect we have another year with over $200B total deal value, a year with $100-150B is very likely.

Based the EY M&A Firepower report, a couple of large Biopharma companies such as GILD, PFE, SNY, PFE, NVS, still haven't made any significant M&A deals in 2019. Based on the chart above, these five companies have about $15B to $25B of dry powder. Companies like GILD, MRK, AMGN, or NVS, might do a mega deal, acquiring a large-cap biotech company in a transaction valued more than $15B. While mega deals are hard to predict, I will focus on deals valued at less than $10B so that this would not use up all the dry powder of those large Biopharma companies mentioned at the start of the section.

Therefore, I am picking 5 best Mid-cap biotech acquisition targets in this post with deal value less than $10B each.

(Source: EY 2019 M&A Firepower Report)

With a market cap of $4.9B, Global Blood Therapeutics is engaged in the innovation and commercialization of drugs to serve the needs of sickle cell disease (NYSE:SCD). SCD is an inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin, leading to the formation of abnormal hemoglobin known as sickle hemoglobin. GBT went up 60% over 3 weeks since the company confirmed FDA approval of Oxbryta (voxelotor), company's main drug to treat sickle cell disease in adults and children 12 years of age and older.

Oxbryta, an oral therapy taken once daily, is the first approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of SCD.

FDA Approves Oxbryta (Voxelotor), the First Medicine Specifically Targeting the Root Cause of Sickle Cell Disease

Following suit, the European Medicines Agency (NYSEMKT:EMA) has also included voxelotor in its Priority Medicines (PRIME) program, while the European Commission (NYSE:EC) has designated voxelotor as an orphan medicinal product for the treatment of patients with SCD. Based on GBT's EHA corporate update in June, Oxbryta is addressing the needs of over 100,000 SCD patients in U.S. and 60,000 patients in Europe. The annual cost of SCD care is $200,000 per patient. An approved drug means R&D risks are largely absent and GBT is attractive to large Biopharma companies which seek to expand their drug pipelines without development risks.

Source

Additionally, GBT started HOPE-KIDS2 which is to confirm there is a decreased risk of stroke for children 2 to 15 years old with Oxbryta. GBT also announced a collaboration with Syros Pharmaceuticals (SYRS) to discover, develop and commercialize novel therapies for sickle cell disease and beta thalassemia. On Dec 18, GBT secured $150M non-dilutive term loan to enhance its cash position for further development and commercialization, which clears any near-term dilution risks and places company in a great spot to negotiate a good deal with potential acquirers.

uniQure (QURE) is developing a set of gene therapy treatments for various diseases, and its lead product candidate AMT-061 is in Phase 3 trials targeting hemophilia B. In July, uniQure reports updated AMT-061 and AMT-060 data in patients with hemophilia B. Topline data from HOPE-B pivotal study will come out in 2020. Positive clinical outcomes will provide the basis for QURE to potentially be the first biotech to have a one-off treatment for patients with Hemophilia B. The Hemophilia B market could reach $8 billion by 2026, making for a potential blockbuster status.

Source

In June 2019, Bloomberg reported that QURE is exploring options including a potential sale or partnerships.

"QURE is drawing interest from pharmaceutical companies looking to expand in gene therapy, according to the report, and a deal would bring access to a pipeline of experimental treatments for hemophilia, Huntington's disease and other disorders."

UniQure considering potential sale - Bloomberg

Source

Gene therapy is one of biotech's hottest area in recent years.

"After the Federal Trade Commission cleared the way last week for it to acquire Spark Therapeutics and its portfolio of treatments for genetically driven diseases, Roche followed up on Dec 23 with a massive $1.2 billion collaboration with Sarepta Therapeutics for the biotech's experimental Duchenne muscular dystrophy gene therapy."

Roche hands $1B to Sarepta in major return to gene therapy deals

"Japanese drugmaker Astellas on Monday became the latest large pharmaceutical company to buy its way into a burgeoning gene therapy field, announcing a $3 billion deal to buy the San Francisco-based biotech Audentes Therapeutics."

Astellas joins gene therapy race with $3B Audentes buy

With management's intention for a sale, QURE will likely be acquired in 2020. In 2019 Q3 earnings release (Source), management stated that, "Strong Balance Sheet with September 30, 2019 Cash and Cash Equivalents of $403 Million Expected to Fund Operations into Mid-2022." Currently QURE is valued at a market cap of $3.3B. A possible deal with premium of 75-100% like Roche's offer for Spark Therapeutics could get approved by QURE's management and shareholders.

On Dec 13, Intercept Pharmaceuticals has filed a marketing application in Europe seeking approval of obeticholic acid (OCA) for the treatment of fibrosis due to nonalcoholic steatohepatitis (NYSEARCA:NASH). An AdCom meeting has been tentatively scheduled for April 22, 2020 related to its U.S. application, necessitating an extension of the agency's current action date of March 26, 2020. New interim data confirmed excellent efficacy and its main drug OCA 25 mg showed robust improvement in liver fibrosis (by 1 stage) with no worsening of NASH at 18 months in the Phase 3 REGENERATE studies. It also cleared doubts about OCA's side effects.

Source

NASH, or nonalcoholic steatohepatitis, is defined as fatty, inflamed liver that is not due to alcohol abuse. In western countries, NASH tends to be associated with obesity, often with Type 2 diabetes. The NIH estimates that 3-12% of American adults have NASH. If the accurate number is 6%, that implies more than 10 million patients in the US. As one of the most promising players in NASH, ICPT is the best acquisition target for Gilead (GILD) to expand its dominance in the $60B NASH market or other large Biopharma companies to have a piece of the NASH pie.

Currently, ICPT is trading at a market cap of $4B with annualized sales of $250M which is growing at 32% year-over-year. As of 9/30/2019, ICPT has $714M cash on hand, which eliminated any near-term equity dilution risks. If you want more information, there are many great articles written on ICPT by other SA contributors where they listed ICPT's strengths in NASH in details.

bluebird bio is a clinical-stage biotech company which builds integrated product platforms that encompass gene therapy, cancer immunotherapy and gene editing for the treatment of cancer and rare diseases. Recently, BLUE announced a positive, long-term follow-up of 5 years for beta-thalassemia patients who have been transfusion-free for an extended period of time. Two weeks ago, BLUE and BMY announced positive topline results from a Phase 2 clinical trial, KarMMa, evaluating CAR T therapy idecabtagene vicleucel (ide-cel) (bb2121) in treatment-resistant multiple myeloma patients. In early 2020, the first commercial patient will be dosed with ZYNTEGLO for transfusion-dependent beta thalassemia in the European Union.

(Source)

Similar to QURE, BLUE is another promising mid-cap biotech company focused on gene therapy and gene editing. With previous buyouts of Kite Pharma, AveXis, Sparkand Audentes, large pharma companies have shown their interests in gene therapy and are optimistic about the future of gene editing. In Nov, BLUE was upgraded to Outperform with a target price of $119 by SVB Leerink.

Leerink sees "the transition to a commercial gene therapy company in early 2020 offering a balance between launch execution as a risk vs. high probability of success trial readouts and regulatory decisions in 2020 as potentially appealing catalysts for BLUE over the next 12 months," Foroohar writes.

bluebird bio upgraded at SVB Leerink on valuation

BLUE has a diverse pipeline which includes multiple Phase 2 and Phase 3 data readouts targeting sever genetic diseases in the next 2 years. On top of that, the Company has also started some clinic trials on Oncology. Comparing to other biotech companies focusing solely on gene therapy, BLUE is a more attractive acquisition target due to its diverse pipeline and huge potential.

(Source)

2019 has been a great year for BLUE regarding its multiple clinical successes. However, the share price is still down 8.7% YTD even with the Q4 biotech rally. A market cap of $4.9B is significantly underestimating the upside potential of company's pipeline and talents. As of 9/30/2019, the Company still has $1.41B cash on hand which is sufficient to last till mid-2021 with a quarterly net loss of $200M. I can easily see a big biopharma company to acquire BLUE for $10-12B for its various drugs in the pipeline targeting both severe genetic diseases and oncology and this purchase would generate massive returns for the next decade.

Amicus Therapeutics is a biopharmaceutical company that develops orally-administered, small molecule drugs to treat human genetic diseases. The drugs, called pharmacological chaperones, are being developed to treat Fabray, Gaucher, and Pompe disease. In its October analyst day presentation, FOLD has guided for peak sales of $1.0 billion for its only commercialized Fabry disease therapy, Galafold. Galafold is expected to reach an annual revenue potential of more than $500 million in 2023. This will imply a five-year CAGR of more than 40%.

(Source)

In 2019 Q3 earnings call, the CEO John Crowley stated,

Galafold continues to be one of the most successful launches for a rare disease medicine ever and remains the cornerstone of our success, with $48.8 million in third quarter revenue. We're also now treating more than 1,000 patients at a 90% plus compliance and adherence rate, and we have upwardly revised full-year 2019 guidance of now $170 million to $180 million. All of the global launch metrics that we track are on target or exceeding targets, including new patient starts compliance and adherence to therapy, reimbursement and access, new country approvals and a broadening prescriber base, among others.

Positive data from Phase 1/2 trial evaluating investigational therapy, AT-GAA, seems to establish Amicus' position in the Pompe disease treatment landscape. In February 2019, AT-GAA secured Breakthrough Therapy Designation from FDA for treatment of late-onset Pompe disease. FOLD is expected to complete enrolling patients in the Phase 3 PROPEL study going into 2020. Data from this trial is anticipated in the first half of 2021. If the trial meets its primary endpoints and drug is approved, FOLD is estimating an annual peak sales opportunity of $1.0 billion - $2.0 billion with AT-GAA. FOLD was also able to achieve positive interim results from a phase 1/2 study using AAV-CLN6 gene therapy to treat patients with Batten disease.

(Source)

Currently, FOLD is trading at a market cap of $2.55B. As of 9/30/2019, FOLD has $514M cash on hand. With exceptional growth in Galafold sales going forward, the company has enough cash going into 1H2022. Considering FY23 Galafold's sales of $500M, the stock is trading at 4x P/S excluding cash. FOLD is an attractive acquisition target for any large biopharma companies which are interested in treating rare disease.

Investing in mid-cap biotech companies is highly risky. This article only is only my personal speculation about which mid-cap biotech companies might get acquired in 2020 with a big fat premium. Speculation for M&A is not a solid basis for long-term investing. If readers are okay with the downside of investing in midcap biotech companies mentioned above, I would recommend buy some common stock at dips.

For company-specific risks, please see disclosures in 10-K and other regulatory filings for a fuller understanding of the risks associated with investing in the particular company.

Disclosure: I am/we are long GBT, QURE. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: I am/we are also long ICPT.

See more here:
5 Best Mid-Cap Biotech Acquisition Targets - Seeking Alpha

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome. Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy. "I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency." Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells. The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors." The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured. "This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville. "But these results are really exciting." In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden. For decades, the DNA of living organisms such as corn and salmon has been modified. But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs. The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself. "It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Cures

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy. Scientists practising the technique insert a normal gene into cells containing a defective gene. It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumor-killing super white blood cells for a cancer patient. Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union. They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and an inherited blindness. Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution. "Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster." Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period." "We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face grueling negotiations with their insurance companies. They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection. "You cannot do this in a community hospital close to home," said her doctor. However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers. They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Bioterrorism

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine. Last year in China, scientist He Jiankui triggered an international scandal -- and his excommunication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans. The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process. "That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations. "It's very easy to do if you don't care about the consequences," Musunuru added. Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability. There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US. The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online. "Not everyone is a biologist or scientist," she said. And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops? Charpentier thinks that technology generally tends to be used for the better. "I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

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Year in Review: Gene Therapy Technology and a Milestone 2019 for Medical Research - News18

The European Medicines Agency has validated BioMarins application to market its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A.

As such, the company said it expects the agencys review of the therapy in January next year under accelerated assessment.

The EMA granted access to its Priority Medicines (PRIME) regulatory initiative in 2017 for valoctocogene roxaparvovec and recently granted BioMarin's request for accelerated assessment of the MAA, potentially shortening the review period.

The submission is based on an interim analysis of study participants treated in an ongoing Phase III study with material from the to-be-commercialised process and updated three-year Phase I/II data.

It marks the first marketing application to be filed in Europe for a gene therapy product for any type of hemophilia.

BioMarin also announced the filing of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for the treatment, with the review expected to being in February.

"We are pleased that the agency has recognised the potential scientific advancement that valoctocogene roxaparvovec could bring to people with severe hemophilia A," said Hank Fuchs, president, Global Research and Development at BioMarin.

"We continue to move thoughtfully and urgently through the regulatory review process to deliver a treatment that we believe has the potential to make a meaningful difference to people with hemophilia A.

Go here to read the rest:
BioMarin's haemophilia gene therapy moves forward in the EU - PharmaTimes

For all the flak the pharmaceutical industry has taken for its exorbitant pricing practices, theres no getting around the fact that its been a pretty stunning decade for medical progress.

Multiple new categories of medicines have moved from dreams and lab benches into the market and peoples lives, and investors who came along for the ride often reaped extraordinary profits. The Nasdaq Biotech Index is up 360% over the last 10 years to the S&P 500s 190%. And thats without mentioning the hundreds of billions of dollars in takeovers that rewarded shareholders with windfalls.

As 2020 approaches, its worth highlighting how far weve come in the last decade in developing new therapies and approaches to treating disease, even as politicians grapple with how to rein in healthcare costs without breaking an ecosystem that incentivizes the search for new discoveries. Here are some of the decades biggest medical breakthroughs:

First approved in the U.S. two years ago, these treatments still sound like science fiction. Drugmakers harvest immune cells from patients, engineer them to hunt tumors, grow them by the millions into a living drug, and reinfuse them. Yescarta from Gilead Sciences Inc. and Novartiss Kymriah the two treatments approved so far can put patients with deadly blood cancers into remission in some cases. At the beginning of the decade, academics were just beginning early patient tests.

Its still in the early days for the technology, and some issues are holding these drugs back. There are significant side effects, and the bespoke manufacturing process is expensive and time-consuming. That has contributed to a bruising price tag: Both of the approved medicines cost over $350,000 for a single treatment. And for now, cell therapy is mostly limited to very sick patients who have exhausted all other alternatives.

Luckily, more options are on their way. Some drugmakers are focused on different types of blood cancers. Others hope to mitigate side effects or create treatments that can be grown from donor cells to reduce expenses and speed up treatment. In the longer run, companies are targeting trickier solid tumors. Scientists wouldnt be looking so far into the future without this decades extraordinary progress.

Researchers have spent years trying to figure out how to replace faulty DNA to cure genetic diseases, potentially with as little as one treatment. Scientific slip-ups and safety issues derailed a wave of initial excitement about these therapies starting in the 1990s; the first two such treatments to be approved in Europe turned out to be commercial flops.

This decade, the technology has come of age. Luxturna, a treatment developed by Spark Therapeutics Inc. for a rare eye disease, became the first gene therapy to get U.S. approval in late 2017. Then in May came the approval of Novartiss Zolgensma for a deadly muscle-wasting disease. The drugs have the potential to stave off blindness and death or significant disability with a single dose, and, unsurprisingly, Big Pharma has given them a substantial financial endorsement. Roche Holding paid $4.7 billion to acquire Spark this year, while Novartis spent $8.7 billion in 2018 to buy Zolgensma developer Avexis Inc.

Dozens of additional therapies are in development for a variety of other conditions and should hit the market in the next few years. They offer the tantalizing potential not just to cure diseases, but to replace years of wildly expensive alternative treatment. If drugmakers can resist the temptation to squeeze out every ounce of value by doing things like charging $2.1 million for Zolgensma, theres potential for these treatments to save both lives and money.

The above treatments modify DNA; this group uses the bodys messaging system to turn a patients cells into a drug factory or interrupt a harmful process. Two scientists won a Nobel Prize in 2006 for discoveries related to RNA interference, or RNAi, one approach to making this type of drug, showing its potential to treat difficult diseases. That prompted an enormous amount of hype and investment, but a series of clinical failures and safety issues led large drugmakers to give up on the approach. Sticking with it into this decade paid off.

Alnylam Inc. has been working since 2002 to figure out the thorny problems plaguing this class of treatments. It brought two RNAi drugs for rare diseases to the market in the last two years and has more on the way. The technology is also moving from small markets to larger ones: Novartis just paid $9.7 billion to acquire Medicines Co. for its Alnylam-developed drug that can substantially lower cholesterol with two annual treatments.

Ionis Pharmaceuticals Inc. and Biogen Inc. collaborated on Spinraza, a so-called antisense drug that became the first effective treatment for a deadly rare disease. It was approved in late 2016 and had one of the most impressive drug launches of the decade. And Moderna Therapeutics rode a wave of promising messenger RNA-based medicines to the most lucrative biotechnology initial public offering of all time in 2018. From pharma abandonment to multiple approvals and blockbuster sales potential in under 10 years. Not bad!

Scientists had been working on ways to unleash the human immune system on cancers well before the 2010s without much luck. Checkpoint inhibitors drugs that release the brakes on the bodys defense mechanisms have since produced outstanding results in a variety of cancers and are the decades most lucrative turnaround story.

Merck got Keytruda via its 2009 acquisition of Schering-Plough, but it was far from the focus of that deal. Once Bristol-Myers Squibb & Co. produced promising results for its similar drug, Opdivo, Merck started a smart development plan that has turned Keytruda into the worlds most valuable cancer medicine. Its now available to treat more than 10 types of the disease, and has five direct competitors in the U.S. alone. Analysts expect the category to exceed $25 billion in sales next year.

If anything, the drugs may have been too successful. Copycat efforts are pulling money that could fund more innovative research. There are thousands of trials underway attempting to extend the reach of these medicines by combining them with other drugs. Some are based more on wishful thinking than firm scientific footing. Still, the ability to shrink some previously intractable tumors is a considerable advance. If drugmakers finally figure out the right combinations and competition creates pricing pressure that boosts access, these medicines will do even more in the years to come.

From a combined economic and public-health standpoint, a new group of highly effective hepatitis C medicines may outstrip just about anything else on this list so far. Cure rates for earlier treatments werent especially high; they took some time to work and had nasty side effects. The approval of Gileads Sovaldi in 2013, followed in time by successor drugs such as AbbVie Inc.s Mavyret, have made hepatitis C pretty easily curable in a matter of weeks. For Gilead, getting to market rapidly with its drug proved enormously profitable; it raked in over $40 billion in revenue in just three years.

Hepatitis C causes liver damage over time that can lead to transplants or cancer. The existence of a rapid cure is a significant long-term boon even if the initial pricing on the drugs made them, in some cases, prohibitively expensive. Sovaldi notoriously cost $1,000 per pill at launch and over $80,000 for a course of treatment. The good news is that treatments have become a lot more affordable, which should allow this class of drugs to have a broad and lasting positive health effect.

Hepatitis C is one of the relatively few markets where the drug-pricing system has worked well. As competing medicines hit the market, the effective cost of these treatments plummeted. That, in turn, made the drugs more accessible to state Medicaid programs and prison systems, which operate on tight budgets and care for populations with higher rates of hepatitis C infection. Louisiana has pioneered the use of a Netflix model, under which the state paid an upfront fee for unlimited access to the drug. Its an arrangement that will help cure thousands of patients, and other states are expected to follow its lead.

Many of the medicines highlighted in this column have list prices in the six figures, a trend thats helped drive up Americas drug spending by more than $100 billion since 2009. Building on this decades medical advances is going to lead to even more effective medicines that will probably come with steeper prices.

Id like to hope that policymakers will come up with a solution that better balances the need to reward innovation with the need to keep medicines accessible.

That would really be a breakthrough.

View original post here:
The 2010s were a decade of medical breakthroughs - Los Angeles Times

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By Vector: Viral vector Retroviruses Lentiviruses Adenoviruses Adeno Associated Virus Herpes Simplex Virus Poxvirus Vaccinia Virus Non-viral vector Naked/Plasmid Vectors Gene Gun Electroporation Lipofection By Gene Therapy: Antigen Cytokine Tumor Suppressor Suicide Deficiency Growth factors Receptors Other By Application: Oncological Disorders Rare Diseases Cardiovascular Diseases Neurological Disorders Infectious disease Other Diseases

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CONTINUE

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Gene Therapy Market 2020: New Innovative Solutions to Boost Global Growth with New Technology, Busin - PharmiWeb.com

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There are 13 chapters to present the Gene Therapy for Age-related Macular Degeneration Market.

Table of contents 1. Report Overview2. Global Growth Trends3. Market Share by Manufacturers4. Gene Therapy for Age-related Macular Degeneration Market Size by Type5. Gene Therapy for Age-related Macular Degeneration Market Size by Application6. Production by Regions7. Gene Therapy for Age-related Macular Degeneration Consumption by Regions8. Company Profiles9. Gene Therapy for Age-related Macular Degeneration Market Forecast10. Value Chain and Sales Channels Analysis11. Opportunities & Challenges, Threat and Affecting Factors12. Key Findings13. Appendix

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Gene Therapy for Age-related Macular Degeneration Market Strategic Business Report 2019-2025 (RetroSense Therapeutics, REGENXBIO, AGTC, and many...