Archive for the ‘Gene Therapy Research’ Category

Whoever comes out on top of the current race to gain pioneering approvals for new AAV-delivered gene therapies will have to look over their shoulders to watch the next tech wave forming on the horizon for gene therapy 2.0.

One of those next-gen players, Generation Bio, just brought in $110 million of venture cash to cover the cost of the rest of their preclinical journey toward something completely new in the field. The latest round brings the biotech which now has about 80 staffers up to $235 million in total since its inception about 3 years ago. That will fuel the rest of its preclinical stage of development as it looks to break into human studies in the back half of 2021.

That kind of 4-plus year timeline before the first human dosing could test the endurance level of a venture player. But Generation CEO Geoff McDonough looks over the past 2 years advancing a new lipid nanoparticle delivery system for their closed-end DNA therapies working to the day when gene therapies can be produced and sold for far less than the $2 million-or-so price tag today and sees lots of fast-paced advances.

I think the reality is we didnt have an expectation at the outset (on timelines), McDonough tells me. Recognizing the novel work needed to build the platform, the investors knew it would take time and money to bring them up to a GMP level.

I would say for a 40-year problem, adds the CEO, 2 years seems pretty good.

The founding tech at Generation was designed to do what AAV treatments do in the nucleus, offering enduring expression, while allowing manufacturing at a biologic scale with a more economical, capsid-free production method. Taking a page from the tech handbooks at companies like Alnylam and Moderna, theyre building a gene therapy that they believe can do much better than the fragile, one-time-only pioneers. And without the $1 million production cost that keeps wholesale prices in the low 7-figure range.

Theyre looking for much greater economy, eventually taking these therapies to much broader ailments and out of the realm of rare diseases with a new approach that they believe can be infinitely redosable on an as-needed basis.

Thats the big picture.

Generations team is working on 2 lead programs for hemophilia A and phenylketonuria (PKU) to go into IND-enabling studies. Theyve now identified Wilson disease and Gaucher disease as likely starting points for the next steps as they move past the liver to skeletal muscle and the retina and then other tissues. And McDonough the former CEO at Sobi is looking down the road 12 to 18 months when hed like to turn to the public markets with an IPO to fund the first clinical-stage work.

In the meantime, hed like to concentrate on opening another new chapter of the company on the dealmaking side.

It felt very important not to partner initially, says McDonough. The investors wanted to retain ownership of platform. We just had tremendous good fortune we didnt need to do that for finance reasons. But now that they have a better grasp of the technology and what needs to be done, its time to partner probably later in the year.

T. Rowe Price funds and accounts led the round, with Farallon and Wellington Management Company jumping in alongside. Existing investors Atlas Venture, Fidelity, Invus, Casdin, Deerfield, Foresite Capital and an entity associated with SVB Leerink came back to stay in the syndicate. Cowen served as exclusive placement agent for the offering.

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With $110M to add to the bankroll, Generation Bio sets its sights on engineering a revolution in the gene therapy field - Endpoints News

Advance Market Analytics recently introduced Global Gene Therapy Market Report tracks the major market procedures including Market Overview, Business Revenue, Introduction, Gross profit & business strategies opted by key market players. The report also focuses on market size, volume and value, shipment, price, interview record, business distribution etc. These data help the consumer know about the competitors better. It also covers different industries clients information, which is very important to understand the market

According to AMA, the Global Gene Therapy market is expected to see growth rate of 19.3%

Summary: Genes are composed of DNA (Deoxyribonucleic Acid) that contains important information for making proteins which are vital for optimal human body function. Certain gene alterations result in these proteins not being made properly & so can lead to genetic disorders. Gene therapy is a new technique which uses genes to prevent or treat disease. Over the coming years, this technique may permit doctors to treat a disorder by inserting a gene into a patients cells rather than using drugs or surgery. This new therapy works by repairing, repressing, or replacing dysfunctional genes which cause disease with aiming of reestablishing normal function. Gene therapy is a striking area for drug development. With the right target & approach, it can address the original cause of a severe diseases.

The major players in Global Gene Therapy Market: Novartis International AG (Switzerland), Kite Pharma, Inc. (United States), Spark Therapeutics Inc. (United States), GlaxoSmithKline PLC (United Kingdom), Bluebird bio Inc. (United States), Genethon (France), Transgene SA (France), Applied Genetic Technologies Corporation (United States), Oxford BioMedica (United Kingdom)

These major players have adopted various organic as well as inorganic growth strategies such as mergers & acquisitions, new product launches, expansions, agreements, joint ventures, partnerships, and others to strengthen their position in this market.

Free Sample Report + All Related Graphs & Charts of Global Gene Therapy Market: https://www.advancemarketanalytics.com/sample-report/16796-global-gene-therapy-market

Market Drivers

Restraints

Opportunities

This report describes the development of the industry by upstream & downstream, industry overall and development, key companies, as well as type segment & market application and so on, and makes a scientific prediction for the development industry prospects on the basis of analysis, finally, analyses opportunities for investment in the industry at the end of the report.

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The Global Gene Therapy Market segments and Market Data Break Down are illuminated below:

Type (Somatic Cell Gene Therapy, Germ Cell Gene Therapy), Application (Neurological Disorders, Rare Diseases, Oncological Disorders, Cardiovascular Diseases, Infectious disease, Other Diseases), Vector Type (Viral Vector, Non-viral Vector)The regional analysis of Global Gene Therapy Market is considered for the key regions such as Asia Pacific, North America, Europe, Latin America and Rest of the World. North America is the leading region across the world. Whereas, owing to rising no. of research activities in countries such as China, India, and Japan, Asia Pacific region is also expected to exhibit higher growth rate the forecast period 2019-2025.

What Global Gene Therapy Market Report Contributes?

In short, the report is a vital guide for understanding the Global Gene Therapy industry accomplishments to the extent each significant perspective like all around learning of the genuine players and benefactors influencing the Global Gene Therapy Market advertise. The examination moreover bases on current Global Gene Therapy point of view, bargains edge, inconspicuous components of the Global Gene Therapy showcase movement.

Key highlights of the Study:

Comprehensive information about factors that will challenge the growth

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There are 15 Chapters analyzing in detail the Global Gene Therapy market.

Chapter 1, to describe Global Gene Therapy Introduction, product scope, market overview, market opportunities, market risk, market driving force;

Chapter 2, to analyze the top manufacturers of Global Gene Therapy, with sales, revenue, and price of Global Gene Therapy, in 2012 to 2018;

Chapter 3, to display the competitive situation among the top manufacturers, with sales, revenue and market share in 2012 to 2018;

Chapter 4, to show the global market by regions, with sales, revenue and market share of Global Gene Therapy, for each region, from 2012 to 2023;

Chapter 5, 6, 7,8 and 9, to analyze the key regions, with sales, revenue and market share by key countries in these regions;

Chapter 10 and 11, to show the market by type and application, with sales market share and growth rate by type, application, from 2012 to 2023;

Chapter 12, Global Gene Therapy market forecast, by regions, type and application, with sales and revenue, from 2018 to 2023;

Chapter 13, 14 and 15, to describe Global Gene Therapy sales channel, distributors, traders, dealers, Research Findings and Conclusion, appendix and data source.

Finally, this report covers the market landscape and its growth prospects over the coming years, the Report also brief deals with the product life cycle, comparing it to the relevant products from across industries that had already been commercialized details the potential for various applications, discussing about recent product innovations and gives an overview on potential regional market shares.

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Gene Therapy Market is expected to see growth rate of 19.3% - BulletintheNews

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The market is driven by the increasing adoption of RNAi technology for undruggable targets. In addition, recent advances in whole-genome RNAi screening is anticipated to boost the growth of the RNAi technology market.

RNAi technology is emerging as a promising therapeutic platform for the treatment of various diseases. This is due to efficient delivery mechanisms, high specificity in the targeted section, and the knockdown approach and safety of RNAi-based therapies. Moreover, RNAi technology has made every gene in the human genome as a druggable target, which is creating new opportunities for vendors to develop a wide range of targeted therapies. This is attracting many drug manufacturers to focus on the development of RNAi-based targeted cancer therapies. Several early-phase trials have reported clinical responses in the case of RNAi therapies directed against targets such as Kinesin spindle protein (KSP) and vascular endothelial growth factor (VEGF). Therefore, the increasing adoption of RNAi technology for undruggable targets is expected to fuel market growth during the forecast period.

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Major Five RNAi Technology Market Companies:

Alnylam Pharmaceuticals Inc.

Alnylam Pharmaceuticals Inc. operates in a single reporting segment, the discovery, development, and commercialization of RNAi therapeutics. The company offers ONPATTRO (patisiran) and GIVLAARITM (givosiran), and other RNAi therapeutics such as Fitusiran, Inclisiran, Lumasiran, and Vutrisiran in late stage (Phase 2 to Phase 4) clinical development pipeline.

Arbutus Biopharma Corp.

Arbutus Biopharma Corp. offers RNAi based therapeutic solutions for drug development, discovery, and commercialization to patients suffering from hepatitis B infection. Some of the key offerings of the company include LNP Delivery Platform and RNA Trigger Technologies.

Arrowhead Pharmaceuticals Inc.

Arrowhead Pharmaceuticals Inc. uses RNAi-based technologies in the development of therapeutics for treating intractable diseases. The company also offers licenses for its patented RNAi technology through collaboration. Targeted RNAi Molecule (TRiM) Platform is the key offering of the company.

Bio-Rad Laboratories Inc.

Bio-Rad Laboratories Inc. operates the business across segments such as Life Science and Clinical Diagnostics. Some of the key offerings of the company include siLentFect Lipid Reagent for RNAi and siLentMer.

Dicerna Pharmaceuticals Inc.

Dicerna Pharmaceuticals Inc. develops RNAi-based therapeutics using GalXC RNAi platform. The company also offers licensed use of their platforms through collaboration. GalXC Technology Platform is the key offering of the company.

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Related Reports on Healthcare include:

Global Cancer Biologics Market Global cancer biologics market by product (monoclonal antibodies, vaccines, cell and gene therapy, and others) and geography (Asia, Europe, North America, and ROW).

Global RNA and DNA Extraction Kit Market Global RNA and DNA extraction kit market by product (RNA extraction kit and DNA extraction kit) and geography (Asia, Europe, North America, and ROW).

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Global RNAi Technology Market 2020-2024 | Evolving Opportunities with Alnylam Pharmaceuticals Inc. and Arbutus Biopharma Corp. | Technavio - Business...

The comprehensive report published by Persistence Market Research offers an in-depth intelligence related to the various factors that are likely to impact the demand, revenue generation, and sales of the CNS Gene Therapy Market. In addition, the report singles out the different parameters that are expected to influence the overall dynamics of the CNS Gene Therapy Market during the forecast period 2018 2028.

As per the findings of the presented study, the CNS Gene Therapy Market is poised to surpass the value of ~US$ XX by the end of 2029 growing at a CAGR of ~XX% over the assessment period. The report includes a thorough analysis of the upstream raw materials, supply-demand ratio of the CNS Gene Therapy in different regions, import-export trends and more to provide readers a fair understanding of the global market scenario.

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The report segregates the CNS Gene Therapy Market into different segments to provide a detailed understanding of the various aspects of the market. The competitive analysis of the CNS Gene Therapy Market includes valuable insights based on which, market players can formulate impactful growth strategies to enhance their presence in the CNS Gene Therapy Market.

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CNS Gene Therapy Market: Facts, Figures and Analytical Insights, 2018 2028 - Expert Recorder

The Gene Therapy Market Report provides key strategies followed by leading Gene Therapy industry manufactures and Sections of Market like- product specifications, volume, production value, Feasibility Analysis, Classification based on types and end user application areas with geographic growth and upcoming advancement. The Gene Therapy market report provides comprehensive outline of Invention, Industry Requirement, technology and production analysis considering major factors such as Revenue, investments and business growth.

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The well-established Key players in the market are:

Pfizer Inc. Novartis AG Bayer AG Sanofi GlaxoSmithKline plc. Amgen Inc. Boehringer Ingelheim International GmbH uniQure N.V. bluebird bio, Inc. Celgene Corporation Others

This report for Gene Therapy Market discovers diverse topics such as regional market scope, product market various applications, market size according to specific product, sales and revenue by region, manufacturing cost analysis, Industrial Chain, Market Effect Factors Analysis, market size forecast, and more.

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Reports include the following segmentation: By Disease Indication Cancer Genetic disorders Cardiovascular diseases Ophthalmology Neurological conditions OthersBy Type of Vectors Viral vectors Non-viral vectorsBy Type of Cells Somatic cells Germline cellsBy Region North Americao U.S.o Canadao Mexico Europeo UKo Franceo Germanyo Russiao Rest of Europe Asia-Pacifico Chinao South Koreao Indiao Japano Rest of Asia-Pacific LAMEAo Latin Americao Middle Easto Africa

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Gene Therapy Market Growth to 2025 - Instanews247

Searching for new methods of attacking serious central nervous system diseases, Biogen has signed a deal that could be run into the hundreds of millions of dollars to essentially license a transcriptional map of the brain and scan it for new drug targets.

The partnership, worth $15 million upfront and far more in milestones, is with a new bioinformatics startup called CAMP4 Therapeutics. Founded in 2018 year by the Whitehead Institutes Richard Young and Harvard Medical Schools Leonard Zon, CAMP4 takes genes considered affiliated with a disease and maps out the various ways cells express those genes and turn them on or off. They then take that map and in the biological equivalent of stretching a war map across a table in a bunker mark up all the different methods of attack.

The more we know about a gene, it doesnt mean we can drug that gene, CEO Josh Mandel-Brehm, who worked at Biogen before joining CAMP4, told Endpoints News. Many genes are not quote-un-quote druggable, so then we have an interesting problem: I think I understand the genetics of the disease, but how do I drug it? And thats where our map solves for this problem.

Alongside the $15 million upfront payment, CAMP4 will be eligible for up to $96 million in milestones for each of the initial targets it supplies, and up to $173 million for each subsequent one.

Biogen is not disclosing what genes or diseases they will target, but the collaboration will focus on microglial cells. These macrophages are found in the central nervous system and have long been considered a key factor in neurodegenerative diseases such as Alzheimers and Parkinsons.

Biogen will bring CAMP4 the genes it believes are central to diseases in the central nervous system, and CAMP4 will inform them all the ways the gene is being expressed and where Biogen might go about turning up or down the expression.

So you say hey I want to move this particular gene for this disease, how should I do that? I want to change the expression of it,' Mandel-Brehm said, describing how they work with partners. Well show you the different nodes, if you will: hey you can drug this, you can drug this, you can drug this. Its modality agnostic.

CAMP4, founded out of Polaris Partners, is one of a couple of new biotechs that build on a discovery Young had back in 2015. The longtime transcriptional biologist published work showing that cells modify gene expression using a series of insulated neighborhoods, 3-D loops of DNA that control gene expression within them. CAMP4 maps those (along with other aspects of the transcriptome). Flagship Pioneerings new Omega Therapeuticsalso tries to drug them.

Since launching in 2018, CAMP4 has done extensive work on mapping genes for liver diseases, including NASH, as part of an in-house development program. Theyve also partnered with synthetic RNAi company Alnylam and are working to expand their maps of cells in the brain, heart, muscles, immune system, blood and kidneys, which they could then license out or use to develop drugs in-house.

The way I think about the diseases were going after is BFP: Big Fucking Problems,Mandel-Brehm said, citing NASH and other diseases that lack effective treatments. And Its well-validated and understood: If youre drugging something thats tied to the gene, youre much more likely to be successful.

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Targeting the BFPs of the CNS, Biogen licenses map of genetic neighborhoods in the brain - Endpoints News

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Major Players Are:BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire PLC, Sangamo Therapeutics, Inc., and Freeline Therapeutics

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Hemophilia Gene Therapy Market- Growth Opportunities by Manufacturers, Regions, Type and Application; Trends Forecast to 2026 - Expert Recorder

Polaris Partners has snagged Eli Lilly vet Darren Carroll as a partner. Carroll spent 22 years at Eli Lilly before making the switch to the investment firms Boston office. Most recently, Carroll served as SVP of corporate business development at Lilly, where he oversaw the $8.1 billion acquisition of Loxo Oncology and the divestiture of animal health company Elanco. Carroll is credited with forming Lilly Asia Ventures and was the founding CEO of Lilly spinout InnoCentive.

Mirati Therapeutics a keenly watched rival to Amgen in the KRAS G12C field has made some changes in its executive team. The biotech welcomed former Spark Therapeutics exec Daniel Faga as COO and Bristol-Myers Squibb vet Benjamin Hickey as CCO. In addition, the company promoted Vickie Reed to SVP, finance and chief accounting officer and Jessica Corson as VP, business development both joined in 2013.

MBX Biosciences has tapped co-founder and board director Kent Hawryluk to run the company as president and CEO. Hawryluk hops over after a stint as CBO of Avidity Biosciences, which he also co-founded. As a partner of Twilight Venture Partners, his previous experience includes helping launch Marcadia Biotech (acquired by Roche) and MB2 (acquired by Novo Nordisk). In addition to Hawryluk, the company has brought on Greg Davis as VP, product development. Davis most recently had a stint as VP of CMC, regulatory quality at Calibrium (acquired by Novo Nordisk).

Months after stepping down as the North America president of Sobi, Rami Levinhas been appointed to be CEO at Saniona focused on the treatment of eating disorders and diseases of the central nervous system. Levin will succeed Jrgen Drejer, who will transition to the role of CSO. Prior to his 5-year tenure at Sobi, Levin headed marketing at Merck Serono.

J&J-partnered Arrowhead Pharmaceuticalshas tappedJames Hassard as the companys first CCO to begin mapping a commercial path for its RNAi therapies. Most recently, Hassard served as SVP of marketing and market access at Coherus BioSciences. Prior to that, he held stints at Medivation, Amgen and Schering Plough.

As its lead therapy clears a pivotal Phase II, bringing it closer to its first BLA,ADC Therapeutics has tapped Joseph Camardo as head of medical affairs. In his new role, Camardo will be responsible for ensuring optimal medical impact for loncastuximab tesirine (ADCT-402) among patients with B-cell non-Hodgkin lymphoma. Camardo joins the Swiss biotech after a stint as SVP of Celgenes global health, which followed positions at Forest Research Institute and Wyeth Research (now Pfizer).

After scrapping their Phase I program and having their CSO hit the exit last May, German biotech Affimedhas brought on Andreas Harstrick as CMO. Most recently, Harstrick served in the same role at Molecular Partners. Harstrick is an alumnus of ImClone, Lilly Oncology and Merck Serono.

Dyve Biosciences has named Pfizer vet Chuck Harbert as CSO following the recent clearance the company received to begin a Phase II trial for their drug, DYV-700. Harbert spent three decades at Pfizer before transitioning to his new role. During his time at the pharma giant, Harbert served as VP, US exploratory development and strategic planning and had roles at Pfizer Central Research. In addition, Harbert was the co-inventor of Zoloft.

Biocon Biologics, a subsidiary of Biocon, has appointed MB Chinappa as CFO. Chinappas appointment comes at the same time as private equity fund True Norths acquisition of 2.44% of the company in a deal worth $74 million. Chinappa makes the jump from one of Biocons other subsidiaries, Syngene, where he was president (finance) and CFO. Chinappa led the Syngenes IPO in 2015.

Momenta Pharmaceuticalshas added the title of CFO to the companys CBO Young Kwon. Kwon joined the company in 2011 and prior to that, he worked at Biogen and Advanced Technology Ventures.

AVEO which had their drug tivozanib spurned once again by the FDA two months ago has appointed Erick Lucera to the position of CFO. Prior to joining the company, Lucera served in the same role at Valeritas. Before that, he was the CFO, treasurer and secretary of Viventia Bio (acquired by Eleven Biotherapeutics and now Sesen Bio).

Candel Therapeutics focused on the development of viral immunotherapies for multiple tumor indications has brought on Genzyme vet Sandra Poole as COO. Poole was most recently COO of LogicBio Therapeutics, after servingas EVP of technical operations and commercial development at ImmunoGen. During her time at Genzyme, Poole oversaw biologics manufacturing.

Antengene, a US-China biotech backed by Celgene, has snagged one of its top execs in China. John Chin, Celgenes general manager in the country, has joined as chief business officer just a few days before former Celgene CEO Mark Alles was appointed to the board of directors. Chins resume spans Aventis, Bristol-Myers Squibb and Merck.

Rubius Therapeutics has appointed autoimmunity and translational immunology expert Laurence Turka as the biotechs first CSO, joining its mission to pioneer a new class of medicines called red cell therapeutics. Turka hops over to the company from Rheos Medicines, where he was a co-founder and CSO. Turka was an entrepreneur-in-residence at Third Rock Ventures, turning to the VC life after an academic career that put him at the head of the American Society of Transplantation and a professor at Harvard Medical School and Massachusetts General Hospital.

Abeona Therapeutics which had some good news last month when the FDA lifted its hold on the companys butterfly disease therapy is parting ways with its executive chairman Steven Rouhandeh, revealed in an SEC filing.

Eiger BioPharmaceuticals has appointed Eldon Mayer as EVP and CCO. Mayer joins just as the company has initiated an NDA for its drug ionafarnib to treat Hutchinson-Gilford progeria syndrome (HGPS or progeria) and progeroid laminopathies. Mayer had the same job at Rigel Pharmaceuticals. Previously, Mayer was the SVP of commercial operations at Questcor Pharmaceuticals (acquired by Mallinckrodt).

Cancer-focused Cellectar Biosciences has named Igor Grachev as CMO. Grachev previously served as global development leader and head of innovative clinical trials initiative, R&D for TEVA Pharmaceuticals. His prior employers include GE Healthcare, Novartis, GSK, Merck, Schering PLough, Sanofi-Aventis and BioClinica.

The immunology experts at IFM Therapeutics who brought in $55.5 million in new venture backing last month has wooedMichael Cooke from Magenta Therapeutics as CSO. BeforeMagenta, Cooke was a founding scientist at the Genomics Institute of the Novartis Research Foundation.

Anti-edema therapies-focused Aeromics has brought on Joseph Schindler as its first CMO. Schindler is an associate professor of neurology and of neurosurgery at Yale University School of Medicine, where he is the clinical chief of the division of vascular neurology. He is also the director of the comprehensive stroke program and telestroke services at Yale New Haven Hospital.

Indias Vyome Therapeutics which back in 2016 raised $14 million for its R&D work on skin diseases has named Craig Tooman COO and CFO. Prior to joining Vyome, Tooman served as the CEO of Aratana Therapeutics. In addition, Tooman has served as founder and CEO of Avanzar Medical and CFO at Ikaria and Enzon Pharmaceuticals.

Baltimore-based startup WindMIL Therapeutics spun out of John Hopkins has named Bristol-Myers vet Karen LaRochelle as SVP, corporate & business development and promoted Patrick Dougherty to SVP, strategy, planning & operations. LaRochelle previously served as CBO of PsiOxus Therapeutics and during her time at BMS served as global head of negotiations and head of business development in China. Prior to his time at WindMIL, Dougherty served as chief of staff to the SVP R&D pipeline for pharmaceuticals R&D at GSK.

Vor Biopharma the biotech co-founded by Siddhartha Mukherjee to pioneer a new type of cancer cell therapy has made two new additions to its team. Amy Mendel joins as chief legal officer and Tania Philipps joins as VP of people. Mendel was the VP & associate general counsel for Ziopharm Oncology while Philipps was the VP and head of human resources at Tango Therapeutics.

JSR life sciences company Selexis has promoted Yemi Onakunle to the position of CBO. Onakunle joined the company in 2012 from Diosynth RTP, where he was the director of commercial development. His other stints include roles at Lonza Custom Manufacturing and Bachem Americas.

Immuneering whose current pipeline focuses on diseases such as cancer cachexia and metastasis has brought on Howard Kaufman as head of research and development. Kaufman, the former president of the Society for Immunotherapy of Cancer (SITC), was most recently CMO at Replimune Group. Kaufmans hiring comes a few weeks after the appointment of Scott Barrett to CMO at Immuneering.

CAR-T-focused Innovative Cellular Therapeutics (ICT) has welcomed Christopher Ballas onboard as SVP of manufacturing. Ballas career has brought him to Rocket Pharmaceuticals, Cook Medical and WuXi AppTec.

Allurion Technologies has recruited Whitney Cypes, former senior director of marketing for Insulet Corporation, as VP of global marketing and Chris Aronson, formerly of Restoration Robotics, as VP of North American sales. In addition, Benoit Chardon was promoted to EVP of commercial.

Eliot Forster-led cancer biotech F-star Therapeutics has appointed Edward Benz, the president and CEO emeritus of the Dana-Farber Cancer Institute, and Minerva exec Geoff Race to the board of directors.

Elias Zerhouni, the former R&D head at Sanofi and ex-NIH director, has taken up a seat at Waypoint Capitals board. The Geneva firm is led by Swiss billionaire and former Serono chief Ernesto Bertarelli and devised an $832 million buyout deal to take allergy drugmaker Stallergenes Greer private.

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Eli Lilly vet jumps aboard Polaris Partners; Mirati shakes up its leadership team with new hires and promotions - Endpoints News

For the past 12 years, Lovelace Biomedical has won competitive contracts for the Gene Therapy Resource Program (GTRP) at the National Heart Lung and Blood Institute (NHLBI), and has also received awards from the National Institute of Health (NIH), to perform studies to investigate and find new models for rare diseases with gene therapy studies. Now, reaching and focusing on commercial contracts, Lovelace continues to build on their history, and as a leader in pre-clinical studies with gene therapy as an important sector of their expertise.

Presenting the webinar is Lovelace's Chief Scientific Officer Jacob McDonald, Ph.D. and Dr. Janet Benson, who has led the Lovelace Gene Therapy Pharm/Tox program since 2007.

The webinar "Nonclinical Development in Gene Therapy, building on 12 years of the Lovelace Biomedical Center of Excellence in Gene Therapy" will be held on February 6, 2020 at 1 pm ET.

Join the Webinar

About Lovelace BiomedicalLovelace Biomedical is a contract research organization that helps pharmaceutical and biotechnology companies advance their complex drug development studies from the preclinical stage, and on to clinical trials. For over 70 years the organization has leveraged its multidisciplinary expertise in toxicology, gene therapy, medical countermeasures, and more, to provide excellence in preclinical research and fully understand the behavior of its clients' investigational products.

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Lovelace Biomedical to host webinar on their history and expertise in the rapidly growing field of Gene Therapy - PRNewswire

An abnormality in the SOD1 gene is linked to some inherited cases of amyotrophic lateral sclerosis (ALS). So could turning off the mutated gene halt the disease? An international research team led by the University of California San Diego School of Medicine showed the potential of that strategy in mice by using a gene therapy approach.

A one-time injection of a gene-silencing RNA delivered by an adeno-associated virus (AAV) vector into the spinal cord prevented the onset of ALS in presymptomatic mice, and it blocked disease progression in rodents that had already developed symptoms. The team reported the findings in the journal Nature Medicine.

The SOD1 gene codes for an enzyme called superoxide dismutase. Normally, the enzyme breaks down superoxide radicals that are produced during cell metabolism. But in ALS, SOD1 mutations can create misfolded SOD1 protein, as toxic oxygen molecules persist, leading to the death of motor neurons.

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The UC San Diego-led team postulated that a short hairpin RNA (shRNA)an artificial RNA molecule that can silence gene expressioncould be utilized to block the dysfunctional SOD1 gene.

Other researchers had tried delivering shRNA-bearing vectors into the blood via intravenous injection. In mouse models of ALS, disease progression was indeed slowed, but the approach only extended survival by about three months. In a more recent study, scientists used intrathecal injection into the cerebrospinal fluid, but the animals lived only two months longer despite being treated immediately after birth.

For the current study, the UCSD researchers injected the shRNA-containing AAV therapy into the spinal subpial space at cervical and lumbar spine levels.

The team observed impressive results. Remarkably, SOD1-mutated mice treated before disease onset never developed disabilities related to motor neuron functions when followed to an average age of 462 days. That means they didn't lose functions like grip strength ororientation reflexes. The control animals, by contrast,started showing symptomsat about 306 days and reached the end-stage of ALS about three months later.

Further analysis showed that the therapy suppressed the accumulation of misfolded SOD1 protein and almost completely preserved motor neuron cells.

In mice that had already entered the symptomatic stage, the injection also blocked disease progression and further motor neuron degeneration, the team reported.

At present, this therapeutic approach provides the most potent therapy ever demonstrated in mouse models of mutatedSOD1gene-linked ALS, the studys senior author, Martin Marsala of UCSD, said in a statement.

RELATED:Biogen's antisense ALS drug shows promise in early clinical trial

Several other strategies have been developed aimed at decreasing the production of mutated SOD1 protein. Swiss biotech Neurimmune has a recombinant antibody called -miSOD1, which the company developed based on memory B cells that are found in healthy elderly people and that protect against misfolded SOD1. In mouse models of ALS, the drug extended the animals lives by up to two months.

Antisense oligonucleotide therapy isanother potential modality for fighting neurodegenerative disease. Biogen recently showed its antisense drug tofersen (BIIB067) was well tolerated in ALS patients in a small phase 1 study. At its highest dose, the drug cutSOD1 protein levels in spinal fluid and the patients performed well on certain clinical function tests.

Marsala and colleagues now plan to run additional studies of their spinal subpial shRNA approach in a large animal model to determine the optimal, safe dosage of the treatment.

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, Marsala said in the statement.

Originally posted here:
Halting ALS with a gene therapy approach - FierceBiotech

NEW YORK, Jan. 6, 2020 /PRNewswire/ --

INTRODUCTIONAdvanced therapy medicinal products, such as cell and gene therapies, have revolutionized healthcare practices. The introduction of such treatment options has led to a paradigm shift in drug development, production and consumption. Moreover, such therapies have actually enabled healthcare providers to treat several difficult-to-treat clinical conditions. In the past two decades, more than 30 such therapy products have been approved; recent approvals include Zolgensma (2019), RECELL System (2018), AmnioFix (2018), EpiFix (2018), EpiBurn (2018), Alofisel (2018), LUXTURNA (2017), Yescarta (2017), and Kymriah (2017). Further, according to a report published by The Alliance for Regenerative Medicine in 2019, more than 1,000 clinical trials are being conducted across the globe by over 900 companies. In 2018, around USD 13 billion was invested in this domain, representing a 73% increase in capital investments in this domain, compared to the previous year. It is worth highlighting that, based on an assessment of the current pipeline of cell therapies and the historical clinical success of such products, it is likely that around 10-20 advanced therapies are approved by the US FDA each year, till 2025.

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The commercial success of cell and advanced therapies is not only tied to whether they are capable of offering the desired therapeutic benefits, but also on whether the developers are able to effectively address all supply chain requirements. The advanced therapy medicinal products supply chain is relatively more complex compared to the conventional pharmaceutical supply chain. As a result, there are a number of risks, such as possible operational inefficiencies, capacity scheduling concerns, process delays leading to capital losses, and deliverable tracking-related issues, which need to be taken into consideration by therapy developers. This has generated a need for bespoke technological solutions, which can be integrated into existing processes to enable the engaged stakeholders to oversee and manage the various aspects of the cell and advanced therapies supply chain, in compliance to global regulatory standards. Over the years, several innovative, software-enabled systems, offering supply chain orchestration and needle-to-needle traceability, have been developed. The market has also recently witnessed the establishment of numerous partnerships, most of which are agreements between therapy developers and software solutions providers. Further, given the growing demand for cost-effective personalized medicinal products, and a myriad of other benefits of implementing such software solutions, the niche market is poised to grow significantly in the foreseen future.

SCOPE OF THE REPORTThe 'Cell and Advanced Therapies Supply Chain Management Market: Focus on Technological Solutions, 2019-2030' report features an extensive study of the growing supply chain management software solutions market. The focus of this study is on software systems, including cell orchestration platforms (COP), enterprise manufacturing systems (EMS), inventory management systems (IMS), laboratory information management systems (LIMS), logistics management systems (LMS), patient management systems (PMS), quality management systems (QMS), tracking and tracing software (TTS), and other such platforms that are being used to improve / optimize various supply chain-related processes of cell and advanced therapies.

Amongst other elements, the report features: A detailed assessment of the current market landscape, featuring a comprehensive list of over 160 technological platforms that are being used to manage the cell and advanced therapies supply chain, along with information on the different types of software systems (COP, EMS, IMS, LIMS, LMS, PMS, QMS, TTS, and others), their key specifications and benefits (chain of identity and custody, compatibility and integration, data management and analytics, regulatory compliance, reliability and security, scalability, software-as-a-service, traceability, user-friendliness, workflow management, and others), affiliated modes of deployment (cloud and on-premises), scale of management (small enterprise, mid-size enterprise and large enterprise), end users (biobanks, cell therapy labs, hospitals, research institutes, commercial organizations, and others), applications (ordering and scheduling, sample collection, manufacturing, logistics, and patient verification and treatment follow-up), regulatory certifications / accreditations (21 CFR Part 11, CLIA, FACT-JACIE, GAMP 5, GDPR, HIPAA, and others), and key support services offered (customization, installation / implementation, maintenance, training / technical support, upgradation, validation and testing, and others). An insightful company competitiveness analysis, taking into consideration the supplier power (based on their employee base and years of experience in the industry) and portfolio-related parameters, such as number of software solutions offered, affiliated modes of deployment, scale of management, end users, applications, regulatory certifications / accreditations, support services offered, and key platform specifications and benefits. Comprehensive profiles of industry players that are currently offering software solutions for supply chain management, featuring an overview of the company, its financial information (if available), and a detailed description of its software system(s). Each profile also includes a list of recent developments, highlighting the key achievements, partnership activity, and the likely strategies that may be adopted by these players to fuel growth, in the foreseen future. A detailed review of the cell and advanced therapies supply chain, offering insights on the processes associated with various stages, such as donor eligibility assessment, sample collection, manufacturing, logistics, and patient verification and treatment follow-up, along with information on cost requirements and existing opportunities for improvement in the supply chain management practices. A qualitative assessment of the current and long-term needs of different stakeholders (patients, healthcare providers, collection centers, manufacturers, logistics service providers and regulators / payers) involved in the cell and advanced therapies supply chain, featuring a summary of the diverse needs and areas of concern, along with our opinion (based on past and prevalent trends) on how the industry is preparing to address such issues. An analysis of the investments made at various stages of development, such as seed financing, venture capital financing, debt financing, grants, capital raised from IPOs and subsequent offerings received by companies that are engaged in this field. An analysis of the partnerships that have been established in the domain, in the period between 2014 and Q3 2019, covering software licensing agreements, mergers and acquisitions, product development agreements, product integration agreements, distribution agreements, asset purchase agreements, and other relevant deals. A detailed analysis of the platform utilization use cases where aforementioned software systems were leveraged by various stakeholders in the domain, in the period between 2014 and Q3 2019, highlighting the ways in which companies have implemented such systems to improve / optimize various supply chain-related processes of cell and advanced therapies. An in-depth analysis of the cost saving potential across various processes of the cell and advanced therapies supply chain that can be brought about by the implementation of bespoke and integrated technological solutions / software systems. A case study on COPs, featuring insights on their key functions and implementation strategies, while also considering their strategic position and connectivity with other adjacent systems within the cell and advanced therapies supply chain. In addition, it provides a brief discussion on the growing popularity of COPs on the social media platform, Twitter.

One of the key objectives of the report was to understand the primary growth drivers and estimate the future size of the supply chain management software solutions market. Based on multiple parameters, such as number of cell and advanced therapies under development, expected pricing, likely adoption rates, and potential cost saving opportunities from different software systems, we have developed informed estimates of the evolution of the market, over the period 2019-2030. In addition, we have provided the likely distribution of the current and forecasted opportunity across [A] different software systems (COP, EMS, IMS, LIMS, LMS, PMS, QMS, and TTS), [B] applications (donor eligibility assessment, sample collection, manufacturing, logistics, and patient verification and treatment follow-up), [C] modes of deployment (cloud and on-premises), [D] end users (biobanks, cell therapy labs, hospitals, research institutes, and commercial organizations), and [E] key geographical regions (North America, Europe and Asia-Pacific). In order to account for the uncertainties associated with some of the key parameters and to add robustness to our model, we have provided three market forecast scenarios portraying the conservative, base and optimistic tracks of the industry's evolution.

The opinions and insights presented in this study were influenced by discussions conducted with several stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals: Bryan Poltilove (Vice President and General Manager, Thermo Fisher Scientific) Jacqueline Barry (Chief Clinical Officer, Cell and Gene Therapy Catapult) Jill Maddux (Director, Cell and Gene Therapy Product Strategy, McKesson) and Divya Iyer (Senior Director, Corporate Strategy and Business Development, McKesson) Martin Lamb (Chief Business Officer, TrakCel)

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

RESEARCH METHODOLOGYThe data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include Annual reports Investor presentations SEC filings Industry databases News releases from company websites Government policy documents Industry analysts' viewsWhile the focus has been on forecasting the market till 2030, the report also provides our independent view on various non-commercial trends emerging in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market gathered from various secondary and primary sources of information.

CHAPTER OUTLINESChapter 2 is an executive summary of the insights captured in our research. It offers a high-level view on the likely evolution of the cell and advanced therapies supply chain solutions market in the mid to long term.

Chapter 3 is an introductory chapter that presents a general overview of the cell and advanced therapies domain, along with details on the various types of such products and the current therapy landscape. It features a detailed discussion on the cell and advanced therapies supply chain and key challenges related to different processes / stakeholders involved in this segment of the industry. The chapter also provides information on various software-enabled systems, highlighting their key advantages and associated challenges. Further, the chapter describes the key growth drivers and roadblocks related to the use of such solutions in the cell and advanced therapies supply chain, offering insights on the emergence of advanced, digital technologies in the domain, as well.

Chapter 4 includes information on more than 160 technological platforms that are currently being used for managing the critical intricacies within the cell and advanced therapies supply chain. It features detailed information on the different types of software systems (COP, EMS, IMS, LIMS, LMS, PMS, QMS, TTS, and others), their key specifications and benefits (chain of identity and custody, compatibility and integration, data management and analytics, regulatory compliance, reliability and security, scalability, software-as-a-service, traceability, user-friendliness, workflow management, and others), affiliated modes of deployment (cloud and on-premises), scale of management (small enterprise, mid-size enterprise and large enterprise), end users (biobanks, cell therapy labs, hospitals, research institutes, commercial organizations, and others), applications (ordering and scheduling, sample collection, manufacturing, logistics, and patient verification and treatment follow-up), regulatory certifications / accreditations (21 CFR Part 11, CLIA, FACT-JACIE, GAMP 5, GDPR, HIPAA, and others), and key support services offered (customization, installation / implementation, maintenance, training / technical support, upgradation, validation and testing, and others).

Chapter 5 features an insightful competitiveness analysis of the software solutions providers based on various parameters, such as number of software systems offered, affiliated modes of deployment, scale of management, end users, applications, regulatory certifications / accreditations, support services offered, and key platform specifications and benefits. In the chapter, stakeholder entities have been plotted on a 2X2 matrix, having a company's Supplier Power (based on its employee base and years of experience in the industry) and Company Competitiveness as the two axes.

Chapter 6 includes elaborate profiles of companies that are currently offering core supply chain management software systems, such as EMS, IMS, LIMS, LMS, PMS, QMS, and TTS (shortlisted on the basis of company competitiveness analysis scores); each profile features an overview of the company, its financial information (if available), and a detailed description of the platform(s). Each profile also includes a list of recent developments, highlighting key achievements, partnership activity and the likely strategies that may be adopted by these players to fuel growth in the foreseen future.

Chapter 7 presents a brief introduction to COPs, along with insights into their key functions and implementation strategies, while also considering their strategic position and connectivity with other adjacent systems within the entire cell and advanced therapies supply chain. Further, the chapter includes a brief discussion on the growing popularity of COP on the social media platform, Twitter, and presents a snapshot of how the public opinion about such technological solutions has evolved in the period 2014-2018. In addition, the chapter features detailed profiles of all the industry players that are currently offering COPs for supply chain management. For each of these companies, we have presented detailed profiles, featuring an overview of the company, its financial information (if available), and a detailed description of the platform(s). Each profile also includes a list of recent developments, highlighting key achievements, partnership activity and the likely strategies that may be adopted by these players to fuel growth in the foreseen future.

Chapter 8 provides information on funding instances and investments that have been made within the cell and advanced therapies supply chain management market. The chapter includes details on various types of investments (such as seed financing, venture capital financing, debt financing, grants, capital raised from IPOs and subsequent offerings) received by companies between 2014 and Q3 2019, highlighting the growing interest of the venture capital community and other strategic investors in this domain.

Chapter 9 features an elaborate discussion and analysis of partnerships / collaborations that have been established in the domain in the period between 2014 and Q3 2019. It includes a brief description of various types of partnership models (such as software licensing agreements, mergers and acquisitions, product development agreements, product integration agreements, distribution agreements, asset purchase agreements, and others) that have been employed by stakeholders within this domain. It also consists of a schematic representation showcasing the players that have established the maximum number of alliances in this industry. Furthermore, we have provided a world map representation of all the deals inked in this field, highlighting those that have been established within and across different continents.

Chapter 10 presents a detailed analysis of platform utilization use cases where the aforementioned software systems were leveraged by various stakeholders in the domain, in the period between 2014 and Q3 2019. It includes a brief discussion on the ways in which companies have implemented such software solutions to improve / optimize various supply chain-related processes of cell and advanced therapies.

Chapter 11 provides a qualitative assessment of the current and long-term needs of different stakeholders (patients, healthcare providers, collection centers, manufacturers, logistics service providers and regulators / payers) involved in the cell and advanced therapies supply chain. It also presents a summary of the diverse needs and areas of concern, along with our opinion (based on past and prevalent trends) on how the industry is preparing to address the aforementioned issues.

Chapter 12 presents a detailed review of the cell and advanced therapies supply chain, offering insights on the processes associated with various stages, such as donor eligibility assessment, sample collection, manufacturing, logistics, and patient verification and treatment follow-up, along with information on cost requirements and existing opportunities for improvement in the supply chain management practices.

Chapter 13 features an insightful analysis, highlighting the cost saving potential across various processes of the cell and advanced therapies supply chain that can be brought about by the implementation of bespoke and integrated technological solutions / software systems.

Chapter 14 features a comprehensive market forecast, highlighting the future potential of the supply chain management software solutions market till 2030, based on multiple parameters, such as number of cell and advanced therapies in development, expected pricing, likely adoption rates, and cost saving opportunity from different software systems. In addition, we have provided the likely distribution of the current and forecasted opportunity across [A] different software systems (COP, EMS, IMS, LIMS, LMS, PMS, QMS, and TTS), [B] applications (donor eligibility assessment, sample collection, manufacturing, logistics, and patient verification and treatment follow-up), [C] modes of deployment (cloud and on-premises), [D] end users (biobanks, cell therapy labs, hospitals, research institutes, and commercial organizations), and [E] key geographical regions (North America, Europe and Asia-Pacific). In order to account for the uncertainties associated with some of the key parameters and to add robustness to our model, we have provided three market forecast scenarios portraying the conservative, base and optimistic tracks of the industry's evolution.

Chapter 15 is a collection of executive insights of the discussions that were held with various key stakeholders in this market. The chapter provides a brief overview of the companies and details of interviews held with Bryan Poltilove (Vice President and General Manager, Thermo Fisher Scientific), Jacqueline Barry (Chief Clinical Officer, Cell and Gene Therapy Catapult), Jill Maddux (Director, Cell and Gene Therapy Product Strategy, McKesson) and Divya Iyer (Senior Director, Corporate Strategy and Business Development, McKesson), and Martin Lamb (Chief Business Officer, TrakCel).

Chapter 16 is a summary of the overall report. In this chapter, we have provided a list of key takeaways from the report, and expressed our independent opinion related to the research and analysis described in the previous chapters.

Chapter 17 is an appendix, which provides a comprehensive list of technological solutions / software systems that are being used to effectively manage and streamline the supply chain processes of the overall healthcare industry.

Chapter 18 is an appendix, which provides tabulated data and numbers for all the figures provided in the report.

Chapter 19 is an appendix, which provides the list of companies and organizations mentioned in the report.

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Cell and Advanced Therapies Supply Chain Management Market: Focus on Technological Solutions, 2019-2030 - PRNewswire

Astellas Pharma recentlyagreed to acquire Audentes Therapeutics, a move it expects will result in faster development of potentially best-in-class therapies for rare neuromuscular diseases, including muscular dystrophy (MD).

Audentes vectorized exon-skipping technology which uses a modified adeno-associated virus (AAV) vector to allow cells to skip over mutated sections of genes will complement Astellas own work, Kenji Yasukawa, president and CEO of Astellas, said in a press release.

Recent scientific and technological advances in genetic medicine have advanced the potential to deliver unprecedented and sustained value to patients, and even to curing diseases with a single intervention, Yasukawa said.

Audentes has developed a robust pipeline of promising product candidates which are complementary to our existing pipeline, including its lead program AT132, he added. By joining together with Audentes talented team, we are establishing a leading position in the field of gene therapy with the goal of addressing the unmet needs of patients living with serious, rare diseases.

The technology uses the modified AAV vector to deliver small molecules antisense oligonucleotides complementary to the RNA sequence of a gene of interest, which allow cells to skip over mutated exons while they are producing proteins.

Exons are the coding regions of genes that provide instructions to make proteins.

Audentes had started developing several therapies for Duchenne muscular dystrophy (DMD) based on its exon-skipping technology. These include AT702, AT751 and AT753.

All three treatment candidates use the same AAV delivery vector. However, as they target different DMD gene exons, the potential therapies are intended for distinct subgroups of patients. AT702 is designed to skip exon 2 and is meant for those who either have duplications in exon 2 or mutations in exons 1-5. AT751 is designed for those with mutations in exon 51, and AT753 for people with alterations in exon 53.

Audentes had also started developing and testing AT466, an experimental treatment for myotonic dystrophy type 1.

The acquisition also gives Astellas direct access to AT132, Audentes lead gene therapy candidate for the treatment ofX-linked myotubular myopathy.

AT132 uses an AAV8 viral vector to deliver a functional copy of the MTM1 gene to muscle cells. This enables the production of myotubularin, an important enzyme for the development and maintenance of muscle cells.

Matthew R. Patterson, chairman and CEO of Audentes, said his company is very pleased with the agreement. With its focus on innovative science and a global network of research, development and commercialization resources, we believe that operating as part of the Astellas organization optimally positions us to advance our pipeline programs and serve our patients, he said.

Under the terms of the agreement, Audentes will become an independent subsidiary of Astellas and will have access to scientific resources to accelerate the development and manufacturing of the combined product pipeline. The transaction, worth $3 billion, is expected to take place early this year.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that make up the lining of blood vessels found in the umbilical cord of newborns.

Total Posts: 42

Jos is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimers disease.

Originally posted here:
New MD Treatments the Main Goal of Astellas, Audentes Merger - Muscular Dystrophy News

PUNE, India, Jan. 6, 2020 /PRNewswire/ -- Immunotherapy is forecast to become the oncology treatment of choice by 2026 with an estimated 60% of previously treated cancer patients likely to adopt immunotherapy in this timeframe. Multiple treatment lines, combination therapy and the opportunity for repeat treatment are likely to accelerate fast growth. Cancer immunotherapy also expands into multiple indications and our analysis indicates that key immunotherapies including anti-PD-1 drugs, dendritic cell vaccines, T-cell therapies and cancer vaccines are all driving the market. The rising incidence and prevalence of numerous cancers globally is a significant accelerator of growth. This is due to more sensitive early detection techniques, higher patient awareness and a growing aging population. Furthermore, the FDA's pro-science attitude will accelerate development and regulatory approval for these drugs. To that end, the cancer immunotherapy market is forecast to hit $115 billion by 2023. Overall strong growth rates are expected due to a significant unmet need and increasing trends of hematological cancers.

Browse Complete Report of Global Cancer Immunotherapy Market Analysis & Forecast to 2023 Report at https://www.reportsnreports.com/reports/2713825-global-cancer-immunotherapy-market-analysis-forecast-to-2023-antibody-drug-conjugates-adcs-bispecific-monoclonal-antibodies-cancer-vaccines-cytokines-interferons-chimeric-antigen-receptor-car-t-cell-thera-inhibitors.html

Prior to the launching of Yervoy, the five-year survival rate for patients with early stage melanoma was 98%; but the five-year survival rate for late-stage melanoma was just 16%. Yervoy has been reported to have a survival rate of 25% when tested alone. When tested as part of a combination therapy treatment with Bristol's nivolumab, the two-year survival rates rose to 88% for patients with late-stage cancer. Increase in patient survival rates brought about by cancer immunotherapy treatment is similar to that seen when bone marrow transplantation changed our conception on how blood cancer was treated. Other key therapeutic players in this market include Opdivo (nivolumab), Keytruda (pembrolizumab), Tecentriq (atezolizumab), Ibrance (palbociclib) the newly approved Bavencio (avelumab) and Imfinzi (durvalumab) and of course the first CAR-T therapies Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel).

Opdivo (nivolumab) from BMS is one of the most exciting agents in the immunotherapy space, and is indicated for melanoma, lung cancer, kidney cancer, blood cancer, head and neck cancer, and bladder cancer. It was given a fast-track approval on December 22, 2014. The majority of immune-oncology agents are anti-programmed death-1 (PD-1) monoclonal antibodies, which will certainly guide the market over the coming years. Projects that currently are valuable include combined immunotherapies on our knowledge of CD137 and PD-1/PDL1 mechanisms. A study on a novel effector activating monoclonal antibody known as IMAB362 for the treatment of solid cancers is also exciting. Other projects comparing CAR-T cell effectiveness against T-cells that target CD19 or mesothelin are interesting in a preclinical setting. Of course, Novartis gained the first CAR-T FDA approval for Kymriah (tisagenlecleucel, CTL019), in August 2017, for children and young adults with B-cell ALL. In October 2017, Yescarta (axicabtagene ciloleucel) from Kite Pharma for adult patients large B-cell lymphoma was also given FDA approval. This is a major boost for the global and US immunotherapy, and gene therapy markets.

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What Are CAR-T Therapies? How Will They Impact the Market?

CAR T (chimeric antigen receptor T) cells are engineered specificity using antibody fragments directed to the tumor cell, and also T-cell CD8/CD3 plasma membrane proteins that elicit specific activity towards the tumor cell, via intracellular signaling pathways. To date publications have revealed a number of effective intracellular molecules in the engineered T cell including CD28, 4-1BB (CD137) and CD3 zeta.

These engineered T cells have numerous advantages including:

Story continues

To date, the main challenges associated with CAR T therapy include manufacturing, regulations, pricing and toxicity in patients. Currently there are over 100 recruiting CAR-T clinical trials globally, mainly in the US, China and Europe. To date a number of CAR T Cells (autologous/allogeneic) trials are demonstrating clinical benefit to patients, but others have demonstrated toxicity such as cytokine release syndrome. In July 2017, an FDA advisory panel determined that the benefits of CAR T outperform the risks. Kymriah (tisagenlecleucel) by Novartis is indicated to treat children and young adults with acute leukemia and performed well in the ELIANA trial. The FDA's Oncologic Drugs Advisory Committee (ODAC) recommended this agent for approval and became the first CAR-T cell therapy on the US market. In October 2017, Yescarta (axicabtagene ciloleucel) from Kite Pharma for adult patients large B-cell lymphoma was also given FDA approval.

The CAR-T industry is addressing unmet needs in specific relapsed cancers, and trials have indicated that some patients show long term activity and high remission rates, but there is a large proportion of patients with toxicities such as cytokine release syndrome and neurotoxicity. The main players within the CAR-T market are Novartis, Juno Therapeutics, Kite Pharma and Cellectis. The market is moving ahead, backed by years of R&D, from both academia and industry, investors capitol and small clinical studies. From now on, Kelly Scientific forecasts that CAR T therapy will become more streamlined, with faster manufacturing times as advances in technologies take hold and clinical trials provide more robust evidence that this immunotherapy is robust. These factors, plus strategies to reduce adverse reactions and toxicities and larger players like Novartis taking stage will push CAR-T therapy ahead. However, recent deaths in the Juno ROCKET trial are creating questions amongst investors. How will the CAR T space influence the total immunotherapy industry going forward? This comprehensive report scrutinizes the total market and provides cutting-edge insights and analysis.

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Within the cancer therapeutics space, which today is worth over $100 billion globally, immunotherapeutic drugs have gained worldwide acceptance. This is because they are targeted therapeutics that have high specificity for cancer cells. Today, cancer immunotherapy drugs have captured nearly 50% of the overall oncology drugs market, generating about $75 billion in 2019 alone and are forecast to surpass $115 billion in 2023. This report describes the evolution of such a huge market in 20 chapters supported by over 180 tables and figures in 450 pages.

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Key Questions Answered in this Report

Another Related Research Titled: Cancer Biomarkers Market Insights 2019, Global and Chinese Analysis and Forecast to 2024 is a professional and in-depth study on the current state of the global Cancer Biomarkers industry with a focus on the Chinese market. The report provides key statistics on the market status of the Cancer Biomarkers manufacturers and is a valuable source of guidance and direction for companies and individuals interested in the industry. Overall, the report provides an in-depth insight of 2014-2024 global and Chinese Cancer Biomarkers market covering all important parameters. Get Free sample copy of this research report at https://www.reportsnreports.com/contacts/requestsample.aspx?name=2358970.

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Global Cancer Immunotherapy Market is Forecast to hit $115 Billion by 2023 - ReportsnReports - Yahoo Finance

GYSS 2020 gathers new and returning eminent scientists and talented young researchers worldwide to convene in Singapore, to spark new ideas and innovations

National Research Foundation Singapore will be organizing The Global Young Scientists Summit 2020 (GYSS 2020) in Singapore from January 14 to 17, with the theme of Advancing Science, Creating Technologies for a Better World.

The world is facing a litany of challenges, from growing antibiotic resistance to a looming shortage in data storage capacity.

GYSS 2020 gathers new and returning eminent scientists and talented young researchers worldwide to convene in Singapore, to spark new ideas and innovations.

To develop the solutions to these problems, young researchers need the help of their more experienced colleagues, and vice versa. Collaborations across disciplines and nations are also vital to spark new ideas and innovations. It will provide a platform for such conversations on science and research, technology innovation, and potential answers to global issues.

The event enables 320 outstanding young scientists to interact with 17 eminent leaders in science and technology, who gather in Singapore from across the world and from a wide variety of research fields, including physics, chemistry, medicine, mathematics, computer science and engineering.

Members of the public will have the opportunity to hear from the distinguished speakers at a series of free public lectures, meanwhile the delegates participate in plenary lectures, panel discussions and interactive group sessions.

The GYSS 2020 speakers include world-renowned recipients of the Nobel Prize, Fields Medal, Millennium Technology Prize and Turing Award.

Participating as speakers for the first time at the Summit are Sir Konstantin Novoselov (Nobel Prize in Physics, 2010), and Dr Kees Immink (Institute of Electrical and Electronics Engineers Medal of Honor, 2017).

Sir Novoselov and his colleague, Sir Andre Geim, who participated at GYSS 2017, isolated and mapped the properties of graphene, a wonder material that consists of a single layer of carbon atoms and is many times stronger than steel, lighter than paper, and an excellent conductor of heat and electricity.

Dr Kees has been one of the most prolific contributors to the field of consumer electronics in the late 20th century.

The GYSS 2020 will also feature a special guest speaker: Professor Alain Fischer, Chair of Experimental Medicine at the Collge de France in Paris.

Professor Fischer has been a pioneer in the fight to understand and treat genetic diseases that are related to the immune system, uncovering many genetic defects that disrupt the human immune system.

He was also one of the first scientists to successfully use gene therapy to treat a rare form of severe combined immunodeficiency, often called the bubble boy disease, after a wellknown patient who lived for years in a plastic bubble filled with filtered air.

The GYSS 2020 is the eighth edition of the event, and will span 15 plenary lectures, panel discussions and interactive small group sessions.

The participants will also go on site visits and engage in dialogue sessions with principal investigators and researchers to better understand the research opportunities in Singapore.

Ashish Rauniyar, a PhD research fellow at the Oslo Metropolitan University who will be attending GYSS 2020, said he is looking forward to meeting talented young researchers from around the world and engaging with the distinguished speakers: The opportunity to talk to them about their scientific experiences is an amazing opportunity.

As part of the summit, panel discussions among the eminent scientists will take place at public forums at local universities and schools, and at the National Library.

Read more here:
National Research Foundation Singapore to organize GYSS 2020 - BSA bureau

(MENAFN - Ameliorate Solutions) uniqure-gene-therapy.jpg" alt="Image result for Hemophilia Gene Therapy" width="380" height="270" />

The Research report on the Hemophilia Gene Therapy Market is complete guide for the new entrants in the industry; the report provides the market history of every product ever retailed by the company. It also provides history of the product types, sales, volume, technology, during the forecast period. The growth rate challenges and barriers are also explained in the Hemophilia Gene Therapy Market research report. The report shades light on the development rate of the strategies. Products and technologies used in the production, manufacturing and marketing of the report.

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Report Coverage

The report is designed to incorporate both qualitative and quantitative aspects of the Hemophilia Gene Therapy Market with respect to each of the regions and countries involved in the study. Furthermore, the report also caters the detailed information about the crucial aspects such as major drivers & restraining factors which will define the future growth of the market.

The report provides a list of all the key players in the Hemophilia Gene Therapy market along with a detailed analysis of the strategies, which the companies are adopting. The strategies mainly include new product development, research, and development, and also provides revenue shares, company overview, and recent company developments to remain competitive in the market.

As part of competitive analysis, the research study includes exhaustive company profiling of leading players of the global Hemophilia Gene Therapy market. All of the segments studied in the report are analyzed based on different factors such as market share, revenue, and CAGR.

The prominent players in the global Hemophilia Gene Therapy market are:

Spark Therapeutics, Ultragenyx, Shire PLC, Sangamo Therapeutics, Bioverativ, BioMarin, uniQure, Freeline Therapeutics

Hemophilia Gene TherapyMarket segment by Types:

Hemophilia AHemophilia B

Hemophilia Gene TherapyMarket segment by Applications: Hemophilia A Gene TherapyHemophilia B Gene TherapyTop of Form Global Hemophilia Gene Therapy Market Segmentation by Region:

North America, United States, Canada, Mexico, Asia-Pacific, China, India, Japan, South Korea, Australia, Indonesia, Malaysia, Philippines, Thailand, Vietnam, Europe, Germany, France, UK, Italy, Russia, Rest of Europe, Central & South America

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In addition, this report discusses the key drivers influencing market growth, opportunities, the challenges and the risks faced by key players and the market as a whole. It also analyses key emerging trends and their impact on present and future development.

The research includes historic data from 2014 to 2018, and forecast to 2026 which makes the reports an invaluable resource for industry executives, marketing, sales and product managers, consultants, analysts, and other people looking for key industry data in readily accessible documents with clearly presented tables and graphs.

Global Hemophilia Gene Therapy Market Overview, Drivers, Restraints and Opportunities, Segmentation overview

Global Hemophilia Gene Therapy Market competition by Manufacturers (2020-2026)

Production and Consumption by Regions

Complete profiling and analysis of Manufacturers (2020-2026)

Manufacturing cost analysis, Materials analysis, Region-wise manufacturing expenses

Industrial Chain, Sourcing Strategy and Downstream Buyers

Marketing Strategy Analysis, Distributors/Traders

Global Hemophilia Gene Therapy Market Effect Factors Analysis (2020-2026)

Global Hemophilia Gene Therapy Market Forecast (2020-2026)

Global Hemophilia Gene Therapy Market Research Findings and Conclusion

The following customization options are available for the report:

-Product Analysis

Product matrix, which gives a detailed comparison of the product portfolio of each company

-Regional Analysis

Further breakdown of the Rest of Europe Hemophilia Gene Therapy market into Russia, Norway, and Denmark

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Detailed analysis and profiling of additional market players (up to five)

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MENAFN06012020007010643ID1099511248

Original post:
Hemophilia Gene Therapy Market Clinical Research and Analysis Report, Forecast 2020-2026 - MENAFN.COM

Yanlan Li,1,2,* Xiangning Li,1,3,* Jiayao Qu,1,3 Dixian Luo,1,3 Zheng Hu1,3

1Translational Medicine Institute, the First Peoples Hospital of Chenzhou Affiliated to University of South China, Hunan 432000, Peoples Republic of China; 2Hunan Province Key Laboratory of Tumor Cellular and Molecular Pathology, Cancer Research Institute, University of South China, Hunan 421001, Peoples Republic of China; 3National & Local Joint Engineering Laboratory for High-Through Molecular Diagnosis Technology, The First Peoples Hospital of Chenzhou, Hunan 432000, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Zheng Hu; Dixian LuoTranslational Medicine Institute, National & Local Joint Engineering Laboratory for High-Through Molecular Diagnosis Technology, The First Peoples Hospital of Chenzhou, Hunan 432000, Peoples Republic of ChinaTel/Fax +86 735 2343902Email hu48005@163.com; luodixian_2@163.com

Purpose: Colorectal cancer (CRC) is one of the major contributors to cancer mortality and morbidity. Finding strategies to fight against CRC is urgently required. Mutations in driver genes of APC or -catenin play an important role in the occurrence and progression of CRC. In the present study, we jointly apply CRISPR/Cas9-sgRNA system and Single-stranded oligodeoxynucleotide (ssODN) as templates to correct a heterozygous TCT deletion mutation of -catenin present in a colon cancer cell line HCT-116. This method provides a potential strategy in gene therapy for cancer.Methods: A Cas9/-catenin-sgRNA-eGFP co-expression vector was constructed and co-transfected with ssODN into HCT-116 cells. Mutation-corrected single-cell clones were sorted by FACS and judged by TA cloning and DNA sequencing. Effects of CRISPR/Cas9-mediated correction were tested by real-time quantitative PCR, Western blotting, CCK8, EDU dyeing and cell-plated clones. Moreover, the growth of cell clones derived tumors was analyzed at nude mice xenografts.Results: CRISPR/Cas9-mediated -catenin mutation correction resulted in the presence of TCT sequence and the re-expression of phosphorylation -catenin at Ser45, which restored the normal function of phosphorylation -catenin including reduction of the transportation of nuclear -catenin and the expression of downstream c-myc, survivin. Significantly reduced cell growth was observed in -catenin mutation-corrected cells. Mice xenografted with mutation-corrected HCT-116 cells showed significantly smaller tumor size than uncorrected xenografts.Conclusion: The data of this study documented that correction of the driven mutation by the combination of CRISPR/Cas9 and ssODN could greatly remedy the biological behavior of the cancer cell line, suggesting a potential application of this strategy in gene therapy of cancer.

Keywords: CRISPR/Cas9, ssODN, targeted gene editing, -catenin, colon cancer

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

Continued here:
Cas9 Mediated Correction of -catenin Mutation and Restoring the | OTT - Dove Medical Press

Axovant Gene Therapies (NASDAQ:AXGT) was upgraded by Zacks Investment Research from a hold rating to a buy rating in a research note issued on Monday, Zacks.com reports. The brokerage presently has a $5.50 price target on the stock. Zacks Investment Researchs price objective would suggest a potential upside of 8.48% from the companys current price.

According to Zacks, Axovant Sciences Ltd. is a biopharmaceutical company which focuses on the acquisition, development and commercialization of therapeutics for the treatment of neurodegenerative disorders. Its product candidate includes RVT-101 which is in different clinical trial for the treatment of Alzheimers disease and other forms of dementia. Axovant Sciences Ltd. is based in Hamilton, Bermuda.

Several other brokerages also recently commented on AXGT. Chardan Capital increased their price target on Axovant Gene Therapies from $10.00 to $15.00 and gave the company a buy rating in a report on Monday, October 28th. ValuEngine upgraded Axovant Gene Therapies from a hold rating to a buy rating in a report on Friday. One equities research analyst has rated the stock with a hold rating and ten have issued a buy rating to the stock. Axovant Gene Therapies currently has an average rating of Buy and a consensus price target of $24.66.

AXGT stock traded up $0.16 during midday trading on Monday, hitting $5.07. 64,342 shares of the company traded hands, compared to its average volume of 72,781. Axovant Gene Therapies has a twelve month low of $3.81 and a twelve month high of $19.60. The company has a quick ratio of 1.41, a current ratio of 1.41 and a debt-to-equity ratio of 0.69. The business has a 50 day simple moving average of $5.18 and a 200-day simple moving average of $6.20. The stock has a market capitalization of $112.81 million, a price-to-earnings ratio of -0.63 and a beta of 1.18.

Axovant Gene Therapies (NASDAQ:AXGT) last announced its quarterly earnings results on Friday, November 8th. The company reported ($0.61) EPS for the quarter, beating the consensus estimate of ($1.15) by $0.54. Equities research analysts forecast that Axovant Gene Therapies will post -3.56 EPS for the current year.

Several institutional investors have recently made changes to their positions in AXGT. BlackRock Inc. acquired a new stake in shares of Axovant Gene Therapies in the second quarter worth $1,482,000. Jane Street Group LLC grew its stake in shares of Axovant Gene Therapies by 28.8% in the second quarter. Jane Street Group LLC now owns 46,455 shares of the companys stock worth $289,000 after acquiring an additional 10,375 shares during the last quarter. Barclays PLC acquired a new stake in shares of Axovant Gene Therapies in the third quarter worth $65,000. Finally, Tower Research Capital LLC TRC grew its stake in shares of Axovant Gene Therapies by 955.3% in the second quarter. Tower Research Capital LLC TRC now owns 4,221 shares of the companys stock worth $27,000 after acquiring an additional 3,821 shares during the last quarter. Institutional investors and hedge funds own 14.80% of the companys stock.

Axovant Gene Therapies Company Profile

Axovant Gene Therapies Ltd., a clinical-stage gene therapy company, focuses on developing a pipeline of product candidates for debilitating neurological and neuromuscular diseases. The company's current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis, Parkinson's disease, oculopharyngeal muscular dystrophy, amyotrophic lateral sclerosis, and frontotemporal dementia.

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Axovant Gene Therapies (NASDAQ:AXGT) Rating Increased to Buy at Zacks Investment Research - Riverton Roll

The first biotech IPO of 2020 is here, and its for a company founded by two marquee names in oncology.

Cambridge-based Black Diamond Therapeutics filed on Friday for an IPO worth up to $100 million, becoming the first biotech of the decade to announce their intention to go public. Theyll use their proceeds to bring theirnew oncogene approach into the clinic.

The fact that an oncology biotech will be the first IPO of the new decade should come as little surprise. The calendar may have changed but the basic incentives that have driven record investment into and revenue from cancer drugs havent. Last years first IPO Poseida Therapeutics was also a cancer-focused biotech.

The first company launched out of Versants Basel-based discovery engine in December 2018, Black Diamond leveraged a high-profile C-suite and emerging science to rapidly rake in cash: Nearly $200 million within a year of their emergence from stealth mode, including an $85 million Series C last month.

The biotech is run by David Epstein and Elizabeth Buck, two former developers of the cancer drug Tarceva. The tech is a form of targeted oncology called allosteric therapies. These therapies are similar to other oncogenic drugs, such as kinase inhibitors, that inhibit the main binding site of a protein fueling cancer. It just does so by inhibiting a different part of the protein that may have mutated.

Black Diamond has spent over a year mapping these mutations, before raising the Series C on the promise of pushing their BDTX-189 drug for HER2 and EGFR mutations into a Phase I/II trial.

They promise to use the bulk of their IPO proceeds for the same goal, with some of the rest going to a preclinical glioblastoma program.

I-Mab Biopharma

The same day as Black Diamonds filing, Shanghai-based oncology startup I-Mab Biopharma updated their filing with detailed information. Theyre expected to be the years first biotech to price, on January 16.

I-Mabsannouncementcame in October, but the new filing offers more detail into a company that hopes to become the first Chinese biotech to list on the Nasdaq exchange since Zai Lab debuted in 2017.

They expect to price between $13 and $14 per share and raise $87 million. They will use the proceeds largely to fund a long list of clinical trials, while slotting significant portions for building their own manufacturing facility in China and research facilities in the US.

The teeming number of assets has to do with I-Mabs unusual but thus-far successful model. The company has spread widely, licensing a long list of assets from more established biotechs. They conduct proof-of-concept trials in the US, and then use the data for trials inChina. Once the drug is clinically validated in the US, I-Mab retains Chinese rights for further development and global out-licensing.

Most of these assets are in oncology including lead multiple myeloma asset TJ202 although TJ101 is a long-acting hormone for pediatric growth deficiency and TJ301 is an IL-6 for autoimmune disorders.

Its a model thats already gained over $400 million in private funding. Their Nasdaq bid reflects the growing importance of China, not only as a burgeoning market for established pharma companies but as a growing hub for drug development.

See the article here:
The first biotech IPO of the new decade has landed and of course it's another cancer drug developer - Endpoints News

January 03, 2020 -Maximum Life Foundation (MaxLife), a non-profit organization focused on aging research, is providing a promising free gene therapy for ten patients with Alzheimers disease.

According to the Alzheimers Association, Alzheimers disease is the sixth leading cause of death in the US. Over five million Americans have the condition, leading to costs of $277 billion a year.

With this gene therapy, researchers have seen improvements in Alzheimers symptoms and the recovery of normal brain functions in experiments with mice. In human cell experiments, the therapy had the same effects through the rejuvenation of microglia, the brains first line of defense against infection, and neurons.

In August 2018, a patient received a low dose of the therapy with no adverse side effects. To date, the patients disease hasnt progressed.

MaxLife will grant 100 percent of the therapy costs to help bring pioneering gene therapy to cure this disease and make Alzheimers disease a thing of the past, said David Kekich, MaxLifes CEO.

Studies have proven that aging is the leading factor in many life-threatening diseases, including Alzheimers. This new gene therapy aims to treat the cellular degeneration caused by aging.

The new treatment is offered by Integrated Health Systems, a gene therapy facilitator that is seeking to treat other adult aging-related diseases with no known cure, including sarcopenia, chronic kidney disease, and atherosclerosis.

This technology could halt many of the big age-associated killers in industrialized countries, said Kekich. Compassionate care helps patients with no other option to get access to experimental therapies that may benefit both themselves and society as a whole.

Other healthcare organizations have stressed the need to leverage gene therapies and precision medicine to improve treatment for Alzheimers and other diseases. A recent study published in Frontiers in Aging Neuroscience discussed how precision medicine tactics will help improve cognitive disease treatment.

Taking a precision medicine approach, the question is no longer Does treatment work? but Who does treatment work for? Identifying the characteristics of non-responders becomes as important as responders in understanding the impact of a particular intervention, the team said.

Such an approach may result in considerable health benefits by allowing more effective selection of individuals for treatments based ona prioriknown profiles of disease risk and their potential response to treatment.

Researchers at Massachusetts General Hospital (MGH) also recently discovered that certain genetic variants may help protect individuals against Alzheimers disease, a finding that could hold important implications for precision medicine therapies.

The team studied a patient who carried a mutation in a gene known to cause early onset Alzheimers but didnt show signs of mild cognitive impairment until her seventies. This is nearly three decades after the typical age of onset. Evaluating this patient, and patients like her, could help researchers understand more about the progression of Alzheimers.

This single case opens a new door for treatments of Alzheimers disease, based more on the resistance to Alzheimers pathology rather than on the cause of the disease. In other words, not necessarily focusing on reduction of pathology, as it has been done traditionally in the field, but instead promoting resistance even in the face of significant brain pathology, said Yakeel T. Quiroz, PhD, clinical neuropsychologist and neuroimagingresearcher at MGH.

With the new gene therapy, MaxLife will add to the growing body of research exploring the use of precision medicine and genetics in chronic disease treatment.

If we can prove a benefit to patients that have no other option now, we can potentially treat Alzheimers disease in people in early to mid-stage Alzheimers, finally creating effective medicine at the cellular level, states Kekich. If successful, this treatment could potentially be used on other diseases such as Parkinsons and ALS.

To apply for a free therapy or for more information, click here.

Original post:
Free Gene Therapy Available for Patients with Alzheimer's - HealthITAnalytics.com

DURHAM Hemophilia. Cystic fibrosis. Duchenne muscular dystrophy. Huntingtons disease. These are just a few of the thousands of disorders caused by mutations in the bodys DNA. Treating the root causes of these debilitating diseases has become possible only recently, thanks to the development of genome editing tools such as CRISPR, which can change DNA sequences in cells and tissues to correct fundamental errors at the source but significant hurdles must be overcome before genome-editing treatments are ready for use in humans.

Enter the National Institutes of Health Common FundsSomatic Cell Genome Editing (SCGE)program, established in 2018 to help researchers develop and assess accurate, safe and effective genome editing therapies for use in the cells and tissues of the body (aka somatic cells) that are affected by each of these diseases.

Todaywith three ongoing grants totaling more than $6 million in research fundingDuke University is tied with Yale University, UC Berkeley and UC Davis for the most projects supported by the NIH SCGE Program.

In the 2019 SCGE awards cycle, Charles Gersbach, the Rooney Family Associate Professor of Biomedical Engineering, and collaborators across Duke and North Carolina State University received two grants: the first will allow them to study how CRISPR genome editing affects engineered human muscle tissues, while the second project will develop new CRISPR tools to turn genes on and off rather than permanently alter the targeted DNA sequence. This work builds on a 2018 SCGE grant, led by Aravind Asokan, professor and director of gene therapy in the Department of Surgery, which focuses on using adeno-associated viruses to deliver gene editing tools to neuromuscular tissue.

Duke engineers improve CRISPR genome editing with biomedical tails

There is an amazing team of engineers, scientists and clinicians at Duke and the broader Research Triangle coalescing around the challenges of studying and manipulating the human genome to treat diseasefrom delivery to modeling to building new tools, said Gersbach, who with his colleagues recently launched the Duke Center for Advanced Genomic Technologies (CAGT), a collaboration of the Pratt School of Engineering, Trinity College of Arts and Sciences, and School of Medicine. Were very excited to be at the center of those efforts and greatly appreciate the support of the NIH SCGE Program to realize this vision.

For their first grant, Gersbach will collaborate with fellow Duke biomedical engineering faculty Nenad Bursac and George Truskey to monitor how genome editing affects engineered human muscle tissue. Through their new project, the team will use human pluripotent stem cells to make human muscle tissues in the lab, specifically skeletal and cardiac muscle, which are often affected by genetic diseases. These systems will then serve as a more accurate model for monitoring the health of human tissues, on-target and off-target genome modifications, tissue regeneration, and possible immune responses during CRISPR-mediated genome editing.

Duke researchers: Single CRISPR treatment provides long-term benefits in mice

Currently, most genetic testing occurs using animal models, but those dont always accurately replicate the human response to therapy, says Truskey, the Goodson Professor of Biomedical Engineering.

Bursac adds, We have a long history of engineering human cardiac and skeletal muscle tissues with the right cell types and physiology to model the response to gene editing systems like CRISPR. With these platforms, we hope to help predict how muscle will respond in a human trial.

Gersbach will work with Tim Reddy, a Duke associate professor of biostatistics and bioinformatics, and Rodolphe Barrangou, the Todd R. Klaenhammer Distinguished Professor in Probiotics Research at North Carolina State University, on the second grant. According to Gersbach, this has the potential to extend the impact of genome editing technologies to a greater diversity of diseases, as many common diseases, such as neurodegenerative and autoimmune conditions, result from too much or too little of certain genes rather than a single genetic mutation. This work builds on previous collaborations between Gersbach, Barrangou and Reddy developing bothnew CRISPR systems for gene regulationandto regulate the epigenome rather than permanently delete DNA sequences.

Aravind Asokan leads Dukes initial SCGE grant, which explores the the evolution of next generation of adeno-associated viruses (AAVs), which have emerged as a safe and effective system to deliver gene therapies to targeted cells, especially those involved in neuromuscular diseases like spinal muscular atrophy, Duchenne muscular dystrophy and other myopathies. However, delivery of genome editing tools to the stem cells of neuromuscular tissue is particularly challenging. This collaboration between Asokan and Gersbach builds on their previous work in usingAAV and CRISPR to treat animal models of DMD.

We aim to correct mutations not just in the mature muscle cells, but also in the muscle stem cells that regenerate skeletal muscle tissue, explainsAsokan. This approach is critical to ensuring long-term stability of genome editing in muscle and ultimately we hope to establish a paradigm where our cross-cutting viral evolution approach can enable efficient editing in multiple organ systems.

Click through to learn more about theDuke Center for Advanced Genomic Technologies.

(C) Duke University

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Duke researchers land $6M in federal grants to advance gene editing - WRAL Tech Wire

New research has deepened the understanding of the underlying causes of Batten disease, including organs and cell types affected as well as crucial molecular mechanisms, which can help the design of novel therapies, a review study reports.

Researchers believe these new insights will be key to inform the targeting, timing, and strategies for future treatments.

The study, Pathomechanisms in the neuronal ceroid lipofuscinoses, was published in the journal Biochimica et Biophysica Acta (BBA) Molecular Basis of Disease.

Batten disease, also known as neuronal ceroid lipofuscinoses (NCLs), is a group of inherited neurodegenerative disorders that share certain clinical symptoms. The various forms of the disease are caused by different mutations and distinguished in part by the age at which symptoms appear.

All are lysosomal storage disorders (LSDs) characterized by the abnormal accumulation of fatty substances, known as ceroid and lipofuscin, inside cells in compartments called lysosomes, which are responsible for breaking down and recycling cell materials.

This buildup is particularly toxic to nerve cells (neurons) and leads to progressive deterioration of the brain, even though other tissues can also be affected.

Novel clinical and preclinical findings have deepened scientists understanding of what causes Batten disease and how these disorders progress over time.

The study reviews these insights, which could facilitate the development of new treatments that target each disease type.

Major advances have been made in therapies targeting the central nervous system, or CNS (comprising the brain and spinal cord), but NCLs should be considered diseases that can affect multiple organ systems, and not just the brain as has been the traditional view, the researchers wrote.

Evidence shows that multiple organs can be affected, with disease spreading to other body regions depending on disease type. Mapping all the tissues affected will be important to refine therapeutic delivery and timing, the researchers said.

Shrinkage, or atrophy, of the brain, accompanied by enlargement of the lateral ventricles (cavities within the brain filled with cerebrospinal fluid) is a common finding in Batten disease. But as the proteins affected in Batten disease are widely expressed in various tissues and cell types, it is likely that other organs are also affected by these disorders.

Observations in animal models and patients, for instance, suggest that the spine, as well as vision, the heart, and the bowel, are likely affected in multiple NCLs.

Identifying and targeting all organs and tissues involved, which so far have been overlooked, is important and could provide added benefit to treatment approaches for Batten disease, the scientists said.

Specific populations of neurons are more vulnerable to Batten disease. Early in the disease, interneurons neurons that connect sensory and motor neurons within the CNS are lost in several regions in the brain. Moreover, a type of neuron involved in controlling motor movement, called Purkinje cells, also seem to be particularly vulnerable.

While the reasons for this are still unclear, the unique biological and electrical properties of these neurons and their greater dependence on lysosomes could explain why they are more vulnerable to Batten disease.

Researchers have also been reconsidering the role played by glial cells, which are cells of the nervous system that provide protection and support to neurons.

Although Batten disease has been considered a disease of neurons, the abnormal accumulation of fatty and proteic substances that mark these disorders occurs in various cell types across the body, in addition to the nervous system.

A growing body of evidence suggests that the activation of astrocytes and microglia two types of glial cells precedes and more accurately predicts where neuronal loss is going to occur, when compared to the actual measurement of fatty material accumulation.

In cell models of CLN1 (known as infantile Batten disease) and CLN3 (known as juvenile Batten disease), astrocytes and microglia were seen to cause neuronal loss, which suggests that they have an important role in the development of Batten disease.

There is also evidence for an antibody-mediated immune response in Batten disease, with a possible autoimmune component a harmful immune response that attacks the bodys own tissues especially in CLN3.

In addition to their role in degrading cell waste, lysosomes are involved in other processes such as sensing nutrients and balancing the levels of calcium and metals, as well as the transport and communication between nerve cells.

Likely related to that is the fact that various Batten disease models are characterized by synaptic dysfunction a malfunction of the synapse, or the junctions between two nerve cells that allow them to communicate.

Other cellular pathways linked to lysosomes including autophagy (the self-eating waste disposal system of cells) and gene activation routes may also be abnormal in Batten disease and contribute to its development.

This information sheds light on potential mechanisms by which NCL mutations may lead to disease, beyond the role of lysosomes.

Various investigational therapies have gone through preclinical tests, including immunomodulatory agents, modulators of lysosomal function, agents that mimic the deficient enzyme in a particular NCL, and inhibitors of glutamate receptor (cell receptors important for transmitting signals between neurons). All these approaches have had varying degrees of success, the review stated.

Some medicines have been tested in patients, such as the immunosuppressive agent mycophenolate mofetil, sold under the brand name CellCept, which was evaluated in a Phase 2 study for CLN3 (NCT01399047).

Cystagon, a molecule that mimics PPT1 (the protein deficient in CLN1), has also been clinically tested in a Phase 4 clinical trial (NCT00028262). However, the benefits of both treatments have been only modest in patients.

This has further highlighted the importance of targeting [disease mechanisms] that are specific to each form of the disease, the researchers wrote.

They believe that targeting the known common defects in neuroinflammation and autophagy may help to develop add-on therapies that could greatly improve the therapeutic efficacy as compared to single-therapy strategies.

Moreover, the discovery of disease manifestations at unexpected sites within or outside the CNS will necessitate the development of therapies that can be targeted to these tissues successfully, the researchers wrote.

Defining the timing of disease in these different tissues, in relation to events in the CNS, will provide important information about effective therapeutic windows and is currently informing the design of various gene therapy clinical trials.

These include ongoing Phase 1/2 clinical trials at the Nationwide Childrens Hospital, in Ohio, testing Amicus Therapeutics gene therapies: AAV-CLN6 for CLN6 disease (NCT02725580) and AAV9-CLN3 for CLN3 (NCT03770572).

To evaluate a gene therapy for CLN2, safety and efficacy studies (NCT00151216, NCT01414985, and NCT01161576) are being conducted at Weill Cornell Medical College in New York.

Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.

Total Posts: 14

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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New Batten Research Key to Informing Potential Therapies, Review Says - Batten Disease News

Dublin, Jan. 03, 2020 (GLOBE NEWSWIRE) -- The "Cell and Advanced Therapies Supply Chain Management Market, 2019-2030: Focus on Technological Solutions" report has been added to ResearchAndMarkets.com's offering.

Cell and Advanced Therapies Supply Chain Management Market: Focus on Technological Solutions, 2019-2030 report features an extensive study of the growing supply chain management software solutions market.

The focus of this study is on software systems, including cell orchestration platforms (COP), enterprise manufacturing systems (EMS), inventory management systems (IMS), laboratory information management systems (LIMS), logistics management systems (LMS), patient management systems (PMS), quality management systems (QMS), tracking and tracing software (TTS), and other such platforms that are being used to improve / optimize various supply chain-related processes of cell and advanced therapies.

One of the key objectives of the report was to understand the primary growth drivers and estimate the future size of the supply chain management software solutions market. Based on multiple parameters, such as number of cell and advanced therapies under development, expected pricing, likely adoption rates, and potential cost saving opportunities from different software systems, we have developed informed estimates of the evolution of the market, over the period 2019-2030.

In addition, we have provided the likely distribution of the current and forecasted opportunity across:

Advanced therapy medicinal products, such as cell and gene therapies, have revolutionized healthcare practices. The introduction of such treatment options has led to a paradigm shift in drug development, production and consumption. Moreover, such therapies have actually enabled healthcare providers to treat several difficult-to-treat clinical conditions.

In the past two decades, more than 30 such therapy products have been approved; recent approvals include Zolgensma (2019), RECELL System (2018), AmnioFix (2018), EpiFix (2018), EpiBurn (2018), Alofisel (2018), LUXTURNA (2017), Yescarta (2017), and Kymriah (2017). Further, according to a report published by The Alliance for Regenerative Medicine in 2019, more than 1,000 clinical trials are being conducted across the globe by over 900 companies.

In 2018, around USD 13 billion was invested in this domain, representing a 73% increase in capital investments in this domain, compared to the previous year. It is worth highlighting that, based on an assessment of the current pipeline of cell therapies and the historical clinical success of such products, it is likely that around 10-20 advanced therapies are approved by the US FDA each year, till 2025.

The commercial success of cell and advanced therapies is not only tied to whether they are capable of offering the desired therapeutic benefits, but also on whether the developers are able to effectively address all supply chain requirements. The advanced therapy medicinal products supply chain is relatively more complex compared to the conventional pharmaceutical supply chain. As a result, there are a number of risks, such as possible operational inefficiencies, capacity scheduling concerns, process delays leading to capital losses, and deliverable tracking-related issues, which need to be taken into consideration by therapy developers.

This has generated a need for bespoke technological solutions, which can be integrated into existing processes to enable the engaged stakeholders to oversee and manage the various aspects of the cell and advanced therapies supply chain, in compliance to global regulatory standards. Over the years, several innovative, software-enabled systems, offering supply chain orchestration and needle-to-needle traceability, have been developed.

The market has also recently witnessed the establishment of numerous partnerships, most of which are agreements between therapy developers and software solutions providers. Further, given the growing demand for cost-effective personalized medicinal products, and a myriad of other benefits of implementing such software solutions, the niche market is poised to grow significantly in the foreseen future.

Amongst other elements, the report features:

In order to account for the uncertainties associated with some of the key parameters and to add robustness to our model, we have provided three market forecast scenarios portraying the conservative, base and optimistic tracks of the industry's evolution.

The opinions and insights presented in this study were influenced by discussions conducted with several stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals:

Key Topics Covered

1. PREFACE1.1. Scope of the Report1.2. Research Methodology1.3. Chapter Outlines

2. EXECUTIVE SUMMARY

3. INTRODUCTION3.1. Context and Background3.2. An Introduction to Cell and Advanced Therapies3.2.1. Classification of Advanced Therapy Medicinal Products3.2.2. Current Market Landscape3.3. Cell and Advanced Therapies Supply Chain3.3.1. Key Processes3.3.2. Challenges Associated with the Cell and Advanced Therapies Supply Chain3.4. Software Solutions for Cell and Advanced Therapies Supply Chain Management3.4.1. Cell Orchestration Platform3.4.2. Enterprise Manufacturing System3.4.3. Inventory Management System3.4.4. Laboratory Information Management System3.4.5. Logistics Management System3.4.6. Patient Management System3.4.7. Quality Management System3.4.8. Tracking and Tracing System3.5. Growth Drivers and Roadblocks3.6. Emergence of Digital Technologies in Supply Chain Management3.6.1. Blockchain Technology3.6.2. Internet of Things3.6.3. Augmented Reality3.6.4. Big Data Analytics3.6.5. Artificial Intelligence

4. CURRENT MARKET LANDSCAPE4.1. Chapter Overview4.2. Cell and Advanced Therapies Supply Chain Management: Overall Market Landscape4.2.1. Analysis by Type of Software Solution4.2.2. Analysis by Key Specification and Benefit4.3.3. Analysis by Application4.3.4. Analysis by End User4.3.5. Analysis by Mode of Deployment4.3.6. Analysis by Scale of Management4.3.7. Analysis by Regulatory Certifications / Accreditations4.3. Cell and Advanced Therapies Supply Chain Management: Developer Landscape4.2.1. Analysis by Year of Establishment4.2.2. Analysis by Location of Headquarters4.2.3. Analysis by Size of Company4.3.4. Analysis by Support Services Offered4.3.5. Leading Developers: Analysis by Number of Software Solutions

5. COMPANY COMPETITIVENESS ANALYSIS5.1. Chapter Overview5.2. Methodology5.3. Assumptions and Key Parameters5.4. Competitiveness Analysis: Overview of Supply Chain Management Software Solution Providers5.4.1. Small-sized Companies5.4.2. Mid-sized Companies5.4.3. Large Companies

6. CORE SUPPLY CHAIN MANAGEMENT SOFTWARE SOLUTIONS: COMPANY PROFILES6.1. Chapter Overview6.2. Brooks Life Sciences6.2.1. Company Overview6.2.2. Financial Information6.2.3. BiobankPro: Software Description6.2.4. Recent Developments and Future Outlook6.3. Cryoport6.3.1. Company Overview6.3.2. Financial Information6.3.3. Cryoportal: Software Description6.3.4. Recent Developments and Future Outlook6.4. MasterControl6.4.1. Company Overview6.4.2. MasterControl Platform: Software Description6.4.3. Recent Developments and Future Outlook6.5. SAP6.5.1. Company Overview6.5.2. Financial Information6.5.3. SAP S/4HANA: Software Description6.5.4. Recent Development and Future Outlook6.6. Savsu Technologies6.6.1. Company Overview6.6.2. Financial Information6.6.3. evo Cold Chain 2.0: Software Description6.6.4. Recent Development and Future Outlook6.7. TraceLink6.7.1. Company Overview6.7.2. Financial Information6.7.3. Digital Supply Chain Platform: Software Description6.7.4. Recent Developments and Future Outlook

7. CELL ORCHESTRATION PLATFORMS: EMERGING TRENDS AND PROFILES OF KEY PLAYERS7.1. Chapter Overview7.2. Supply Chain Orchestration Platforms7.2.1. Key Functions of Supply Chain Orchestration Platforms7.2.2. Advantages of Supply Chain Orchestration Platforms7.2.3. Supply Chain Orchestration Platform Implementation Strategies7.3. Supply Chain Orchestration Platform: Trends on Twitter7.3.1. Scope and Methodology7.3.2. Historical Trends in Volume of Tweets7.3.3. Popular Keywords7.4. Key Industry Players7.4.1. Be The Match BioTherapies7.4.2. Clarkston Consulting7.4.3. Haemonetics7.4.4. Hypertrust Patient Data Care7.4.5. Lykan Bioscience7.4.6. MAK-SYSTEM7.4.7. sedApta Group7.4.8. Stafa Cellular Therapy7.4.9. Title 21 Health Solutions7.4.10. TrakCel7.4.11. Vineti

8. FUNDING AND INVESTMENT ANALYSIS8.1. Chapter Overview8.2. Types of Funding8.3. Cell and Advanced Therapies Supply Chain Management: Recent Funding Instances8.3.1. Analysis by Number of Funding Instances8.3.2. Analysis by Amount Invested8.3.3. Analysis by Type of Funding8.3.4. Analysis by Number of Funding Instances and Amount Invested across Different Software Solutions8.3.5. Most Active Players: Analysis by Amount Invested8.3.6. Most Active Investors: Analysis by Participation8.3.7. Geographical Analysis by Amount Invested8.4. Concluding Remarks

9. PARTNERSHIPS AND COLLABORATIONS9.1. Chapter Overview9.2. Partnership Models9.3. Cell and Advanced Therapies Supply Chain Management: Recent Collaborations and Partnerships9.3.1. Analysis by Year of Partnership9.3.2. Analysis by Type of Partnership9.3.3. Analysis by Partner's Focus Area9.3.4. Analysis by Type of Software Solution9.3.5. Most Active Players: Analysis by Number of Partnerships9.3.6. Analysis by Regions

10. PLATFORM UTILIZATION USE CASES10.1. Chapter Overview10.2. Cell and Advanced Therapies Supply Chain Management: Recent Platform Utilization Use Cases10.2.1. Analysis by Year of Utilization10.2.2. Analysis by User's Focus Area10.2.3. Analysis by Type of Software Solution10.2.4. Most Active Players: Analysis by Number of Utilization Instances10.2.5. Most Active Players: Regional Analysis by Number of Utilization Instances

11. VALUE CHAIN ANALYSIS11.1. Chapter Overview11.2. Cell and Advanced Therapies Value Chain11.2. Cell and Advanced Therapies Value Chain: Cost Distribution11.3.1. Donor Eligibility Assessment11.3.2. Sample Collection11.3.3. Manufacturing11.3.4. Logistics11.3.5. Patient Verification and Treatment Follow-up

12. STAKEHOLDER NEEDS ANALYSIS12.1. Chapter Overview12.2. Cell and Advanced Therapies Supply Chain Management: Needs of Different Stakeholders12.2.1. Comparison of Stakeholder Needs

13. COST SAVINGS ANALYSIS13.1. Chapter Overview13.2. Key Assumptions and Methodology13.3. Overall Cost Saving Potential of Supply Chain Management Software Solutions, 2019-203013.3.1. Cost Saving Potential in Donor Eligibility Assessment, 2019-203013.3.2. Cost Saving Potential in Sample Collection, 2019-203013.3.3. Cost Saving Potential in Manufacturing, 2019-203013.3.4. Cost Saving Potential in Logistics, 2019-203013.3.5. Cost Saving Potential in Patient Verification and Treatment Follow-up, 2019-2030

14. MARKET FORECAST14.1. Chapter Overview14.2. Key Assumptions and Forecast Methodology14.3. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market, 2019-203014.3.1. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Application14.3.2. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by End User14.3.3. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Type of Software Solution14.3.4. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Mode of Deployment14.3.5. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Geography14.4. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Application, Type of Software Solution and Mode of Deployment14.4.1. Cell and Advanced Therapies Supply Chain Management Solutions Market for Donor Eligibility Assessment, 2019-203014.4.2. Cell and Advanced Therapies Supply Chain Management Solutions Market for Sample Collection, 2019-203014.4.3. Cell and Advanced Therapies Supply Chain Management Solutions Market for Manufacturing, 2019-203014.4.4. Cell and Advanced Therapies Supply Chain Management Solutions Market for Logistics, 2019-203014.4.5. Cell and Advanced Therapies Supply Chain Management Solutions Market for Patient Verification and Treatment Follow-up, 2019-2030

15. EXECUTIVE INSIGHTS15.1. Chapter Overview15.2. Thermo Fisher Scientific15.2.1. Company Snapshot15.2.2. Interview Transcript: Bryan Poltilove, Vice President and General Manager15.3. Cell and Gene Therapy Catapult15.3.1. Company Snapshot15.3.2. Interview Transcript: Jacqueline Barry, Chief Clinical Officer15.4. McKesson15.4.1. Company Snapshot15.4.2. Interview Transcript: Jill Maddux, Director, Cell and Gene Therapy Product Strategy, and Divya Iyer, Senior Director, Corporate Strategy and Business Development15.5. TrakCel15.5.1. Company Snapshot15.5.2. Interview Transcript: Martin Lamb, Chief Business Officer

16. CONCLUDING REMARKS16.1. Chapter Overview16.2. Key Takeaways

17. APPENDIX 1: LIST OF ADDITIONAL SUPPLY CHAIN MANAGEMENT SOFTWARE SOLUTIONS

18. APPENDIX 2: TABULATED DATA

19. APPENDIX 3: LIST OF COMPANIES AND ORGANIZATIONS

For more information about this report visit https://www.researchandmarkets.com/r/kw1hkc

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Read the original post:
Cell and Advanced Therapies Supply Chain Management Industry Report, 2019-2030 - GlobeNewswire

With the books being closed on 2019, its time to prognosticate about what the next year will hold in the biotech and pharmaceutical industry. And, to paraphrase a famous song from the 1980s, grab a set of shades, because the future looks bright.

BioSpace spoke to leaders from various corners of the industry who provided their insights into what the coming year is likely to hold for their particular sphere. Below are predictions for three spheres within the industry for the coming year.

Oncology

Immuno-oncology has been one of the research cornerstones in cancer research and that will continue. However, in 2020, traditional targeted therapies will continue to be of importance. Stephen Gately, chief executive officer of TD2 (Translational Drug Development), an oncology-focused contract research organization, said there are still roles for traditional targeted therapeutics for specific indications. Following Mercks acquisition of ArQule, Inc. and its kinase inhibitor discovery and development programs for cancer treatment, predicted the industry might see a refocus on understanding cancer and how it can respond to different drugs. He said Merck would not have spent $2.7 billion for ArQules Phase II Brutons tyrosine kinase inhibitor ARQ 531 if there was not a potential upside for the therapy, particularly as a treatment for B-cell cancers. If trial protocols are well-established and there are stricter guidelines for patient selections, Gately said the use of certain inhibitors are vastly superior to immunotherapies in development.

When it comes to clinical trials, Gately predicted there could be a change in how those are organized. He said the goal for companies is to attempt to get their assets into trials as quickly as possible but noted there is a heated and competitive battle for trial sites. As companies attempt to elbow their way to the front of the line, Gately said it is possible that the industry will begin to see more opportunities for economic incentives used to convince the clinical trial sites to take up trials sooner. If that is established, Gately said it will become a game-changer.

Cell and Gene Therapies

This past year saw the approval of gene therapies that can address devastating diseases. And that is likely to continue in 2020. But, safety will still be paramount. Ena Cratsenburg, chief business officer of Ginkgo Bioworks, said over the course of the next year, we will see drug developers use synthetic biology to improve their ability to develop therapeutics with unprecedented levels of activity and control. In 2020, synbio companies will solidify their positions as key enablers in bringing the most innovative medicines to consumers, Cratsenburg said.

Mark Sawicki, chief commercial officer at Cryoport, developer of the first shippers certified for the delivery of human advanced therapies, said that as the cell and gene therapies continue to show their importance for treating new diseases, 2020 will usher in a record year for biologics license applications and marketing authorization applications. Sawicki suggested that over the course of the coming year, eight or nine companies will file such applications to gain regulatory approval of their products. In addition to the high number of filing companies, he suggested that there will be multiple new products launching with three or four generating revenues in 2020. By 2023, Sawicki predicts there will be at least 22 commercial launches in the space.

Jason Steiner, vice president of business development and strategy at gene editing company Synthego, said in 2020, the gap between the pace of innovation in the cell and gene therapy landscape and the infrastructure required for commercialization and deployment will continue to widen before it converges again.Steiner said the development of engineered cell therapies will continue to move toward non-viral approaches in order to boost engineering sophistication while decreasing the time and cost of traditional engineering. He added that manufacturing consideration of cell and gene therapies will continue to move farther upstream. They will be more tightly integrated with early development to avoid commercialization bottlenecks that are being caused by a rapid increase in the pace of development and regulatory approval cycles.

Real World Evidence

The importance of Real World Evidence (RWE) will continue to grow in 2020. Jane Reed, director of life sciences at U.K.-based Linguamatics, said the industry will continue to harness RWE in its drug development programs. RWE was not a topic of conversation in the industry 10 years ago, Reed said, but now there is significant value seen from what RWE brings to the table. RWE relates to the collection of information about a drugs safety and efficacy outside the structure of a clinical trial. The data can be collected from a variety of sources, including electronic health records, wearable devices, lab tests and more. The pharma industry will need to not only continue to collect the data, but learn to use it in a way that improves the outcomes of the patients it serves. Reed said the industry has to show they are listening to the patient community and be flexible. From there, the industry will need to feed that patient-driven data into drug discovery efforts. To boost the collection of RWE, Reed said the use of targeted social media research will also increase the amount of data that companies have at their fingertips. As companies within the pharmaceutical industry continue to dig into the depths of disease states, particularly those of rare diseases, Reed said the use of RWE will prove to be increasingly important.

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What Does 2020 Have in Store for the Life Sciences? - BioSpace

Home Topics Bleeding Disorders

BioMarin has submitted a Biologics License Application (BLA) to the Food and Drug Administration (FDA) for valoctocogene roxaparvovec (BMN 270) for the treatment of hemophilia A in adults. This is the first marketing application submission for a gene therapy product for any type of hemophilia.

Valoctocogene roxaparvovec is an investigational adeno-associated virus (AAV) gene therapy that is administered as a single infusion to produce clotting factor VIII. The BLA submission is supported by interim analysis of a phase 3 study and 3-year phase 1/2 data. Results from the ongoing phase 1/2 study showed that bleed rate control and reduction in factor VIII usage was maintained for a third year following a single administration of valoctocogene roxaparvovec.

The FDA previously granted Breakthrough Therapy and Orphan Drug designations to valoctocogene roxaparvovec. The Company anticipates the BLA review to commence in February 2020.

We look forward to working with the FDA as we seek marketing authorization for the potential first gene therapy for hemophilia A, said Hank Fuchs, MD, President, Global Research and Development at BioMarin. Our hope is one day very soon to deliver a transformative treatment that has the potential to change the way hemophilia A is treated.

For more information visit biomarin.com.

This article originally appeared on MPR

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BLA Submitted for Gene Therapy to Treat Hemophilia A - Hematology Advisor

Throughout 2019, ALS News Today brought you daily coverage of key findings, treatment developments, clinical trials, and other events related to amyotrophic lateral sclerosis (ALS).

As a reminder of what mattered most to you in 2019, here are the top 10 most-read articles of last year with a brief description of what made them interesting and relevant to the ALS community.

We look forward to reporting more news to patients, family members, and caregivers dealing with ALS during 2020.

A tale of two preclinical studies showed that a gene therapy candidate targeting a key ALS mutation in the C9orf72 gene was able to lessen the buildup of toxic RNA clumps and reduce the activity of the mutated gene in cells collected from a patient with frontotemporal dementia (FTD) and a mouse model of ALS.

Developed by uniQure,the therapy is designed to silence thedisease-causing gene. It works by delivering microRNAs (miRNAs) RNA molecules that regulate gene expression that target the mutated C9orf72s RNA for degradation. The results supported the continuation of uniQures gene therapy program in ALS and FTD, the company said.

In the summer, a small study discovered an altered composition of gut microbes in people with ALS, which could drive digestive problems in those with the disease.

Using genetics, a research team in China found that fecal samples of people with ALS have an increase in harmful microbes of the phylum Firmicutes and a decrease in beneficial microorganisms called Bacteroidetes. The resulting poor gastrointestinal health may lead to a decline in the guts digestion and metabolism functions.

At the beginning of the year, U.S. biopharmaceutical MediciNova received a notice of allowance stating that its request for a patent covering a combination of its investigational therapy ibudilast (MN-166) plus Rilutek (riluzole) was being consideredby the U.S. Patent and Trademark Office.

Ibudilast is a small molecule that reduces the activity of immune cells in the brain while supporting the growth of motor neurons, those lost in people with ALS. In a Phase 2 trial (NCT02238626), the treatment was found to work well in combination with Sanofis approved therapy Rilutek, improving patients functional activity, quality of life, and muscle strength.

The notice of allowance was the final step toward patent registration. Such registration will provide patent protection until November 2035 to the combination, for use in treating ALS and other neurodegenerative diseases.

In June, MediciNova launched a Phase 2b/3 clinical trial to continue studying ibudilast as an add-on therapy to Rilutek. The multicenter, double-blind study (NCT04057898) will recruit approximately 230 participants, who will be randomly selected to receive either ibudilast plus Rilutek or a placebo plus Rilutek for 12 months.

The trials main goal is to study ibudilasts impact on ALS progression and functional disability by determining changes in the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score between the beginning and end of the treatment period. Secondary objectives include changes in patients muscle strength, quality of life, and respiratory function. The study also will evaluate the safety and tolerability profile of ibudilast.

Just a few months earlier, MediciNova had received approval from the U.S. Food and Drug Administration (FDA) to initiate this trial. Eligible patients must have had the disease for no more than 18 months and present just mild disability.

During the trial, patients will receive Rilutekfor at least 30 days before starting a regimen of either 100 mg per day of ibudilast or a placebo for a period of 12 months. Participants may then enter an extension phase in which they will be offered the ibudilast combination for an additional six months. Top-line data is expected by December 2021.

The Phase 3 trial evaluatingBrainStorm Cell Therapeuticss cell therapy candidateNurOwn completed patient enrollment in October. The 200 participants will receive three administrations of NurOwn, or a placebo, into the spinal canal every two months. The primary goals are to demonstrate the therapys safety and its ability to slow disease progression.

A cell-based therapy, NurOwn works by removing specific stem cells from patients and converting them to cells that produce molecules that promote nerve tissue growth and survival. The modified cells are then returned to the patient to stimulate nerve tissue growth. NurOwn aims to safely improve abilities like swallowing, speech, handwriting, and walking in people with ALS.

A long-term Phase 2/3 study examining vitamin B12 as a treatment for ALS found that ultra-high doses of methylcobalamin, the physiologically active form of this vitamin, may extend survival and slow the decline in functional capacity, compared with a placebo.

The study included 373 patients, diagnosed fewer than three years earlier, across 51 sites in Japan. However, the benefits were only seen in patients diagnosed less than one year before taking the supplements, and only a trend was observed.

Results from a Phase 1 clinical trial (NCT02870634) showed that CuATSM a small molecule able to selectively deliver copper to cells with damaged mitochondria slowed disease progression and improved the respiratory and cognitive function of people with ALS.

Damaged mitochondria are considered a hallmark of several neurodegenerative diseases, including ALS, and delivering copper is thought to restore the health of these organelles. Developer Collaborative Medicinal Development (CMD) began testing CuATSM in patients with sporadic and familial ALS in 2016.CMD is now planning to launch a randomized, placebo-controlled clinical trial for CuATSM to confirm these results.

In March, a study discovered several species of bacteria and fungi living in the central nervous system (CNS) of people with ALS, suggesting that patients have coexisting bacterial and fungal infections.

The study built on prior research suggesting that ALS might be caused by a fungal infection. Researchers then examined frozen CNS tissue from 11 ALS patients to assess whether bacterial infections accompany fungal infections. Bacterial DNA was found in different regions of the CNS and the presence of bacteria was confirmed in neural tissue samples.

Our most-read article of 2019reported that small nerve damage may serve as an ALS trigger. Such damage may accelerate motor symptoms in rats carrying a mutation in the SOD1 gene,one of the 40 genes associated with ALS development in humans.

While rats without the mutation completely recovered leg function four weeks after induced damage to the sciatic nerve, located in the leg, SOD1-mutated animals were unable to fully recover. These rats also lost function in the uninjured leg, likely as a consequence of sustained immune activation and more severe neurodegeneration.

The induced nerve damage mimics head injury and trauma in human patients, which could explain the higher prevalence of the disease among war veterans and professional athletes.

***

At ALS News Today, we hope these stories and our regular reporting throughout 2020 contribute to informing and improving the lives of everyone affected by ALS.

We wish all our readers a happy 2020.

Total Posts: 6

Ins holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Cincias e Tecnologias and Instituto Gulbenkian de Cincia. Ins currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.

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Top 10 ALS Stories of 2019 - ALS News Today