Archive for the ‘Gene Therapy Research’ Category

The Electrical Shielding Tape market is an intrinsic study of the current status of this business vertical and encompasses a brief synopsis about its segmentation. The report is inclusive of a nearly accurate prediction of the market scenario over the forecast period market size with respect to valuation as sales volume. The study lends focus to the top magnates comprising the competitive landscape of Electrical Shielding Tape market, as well as the geographical areas where the industry extends its horizons, in magnanimous detail.

The market report, titled Global Electrical Shielding Tape Market Research Report 2019 By Manufacturers, Product Type, Applications, Region and Forecast to 2025, recently added to the market research repository of details in-depth past and present analytical and statistical data about the global Electrical Shielding Tape market. The report describes the Electrical Shielding Tape market in detail in terms of the economic and regulatory factors that are currently shaping the markets growth trajectory, the regional segmentation of the global Electrical Shielding Tape market, and an analysis of the markets downstream and upstream value and supply chains.

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The report offers the market growth rate, size, and forecasts at the global level in addition as for the geographic areas: Latin America, Europe, Asia Pacific, North America, and Middle East & Africa. Also, it analyses, roadways and provides the global market size of the main players in each region. Moreover, the report provides knowledge of the leading market players within the Electrical Shielding Tape market. The industry-changing factors for the market segments are explored in this report. This analysis report covers the growth factors of the worldwide market based on end-users.

The key manufacturers covered in this Electrical Shielding Tape market report:

Daimler AGSchmitz CargobullGreat DaneUtility TrailerPaccarVolvoWabash National CorporationFontaineHyundai TransleadNavistarKogel Trailer GmbhSchwarzmuller GroupFAW SipingHuida HeavyCIMCSinotrukLiangshan Huayu

Segment by RegionsNorth AmericaEuropeChinaJapanSoutheast AsiaIndia

Segment by TypeUp To 50 T50-100 TAbove 100 T

Segment by ApplicationCement IndustryFood IndustryChemical IndustryLogistics IndustryOil and Gas IndustryOther

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In accordance with a competitive prospect, this Electrical Shielding Tape report dispenses a broad array of features essential for measuring the current Electrical Shielding Tape market performance along with technological advancements, business abstract, strengths and weaknesses of market position and hurdles crossed by the leading Electrical Shielding Tape market players to gain leading position. Other aspects such as customer base, sales reach, local coverage, production price trends, and production cost layout are also analyzed to bestow accurate rivalry perspective.

Pivotal highlights of Electrical Shielding Tape market:

The Electrical Shielding Tape market report includes a brief about the cost analysis, key raw material used, as well as the fluctuating price trends of the war material.

The suppliers of the raw material and their market concentration rate have also been enlisted.

The manufacturing cost structures, encompassing details about the raw material, manufacturing process analysis, as well as labor costs have been enumerated in the study.

Substantial details about the industry chain analysis, downstream buyers, and sourcing strategies have been elucidated.

A separate section has been designated for the analysis of the marketing strategy adopted, as well details about the distributors that are a part of the supply chain.

The report is inclusive of information regarding the channels adopted for the product marketing, marketing channel development trends, pricing and brand strategies, as well as target clientele.

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Gene Therapy Market 2020 Industry Size, Regions,Trends and Top Manufacturers Bluebird Bio, Sangamo, Spark Therapeutics, Dimension Therapeutics,...

Global Gunshot Detection System Marketwas valued US$ 920.2 Mn in 2017 and is expected to reach US$ 7120.7 Mn by 2026, at CAGR of 29.2% during forecast period.

The major growth drivers of the gunshot detection system market include the rise in demand for advanced security systems, increasing demand for security in various industry verticals, and rising criminal activities in urban areas. In addition, Increased incidences of gun-firing, especially at school premises, have led to various security measures, such as warning people in close proximity and alerting authorities to the location of the shooting all these factors will drive the growth of Gunshot Detection system market during the forecast period. Increasing government initiative side has led to the growth of the Gunshot Detection system across the globe. The latest trend for smart cities across the globe is resulting in the quantitative increase in demand of Gunshot across the nation.

Growing investment in border security is also likely to drive the market growth in the coming years, as gunshots systems can help border security forces to detect and stop any attempts of smuggling, infiltration, and illegal immigration. Also, with a little amendment of the system, it might be possible for the manufacturers to help forces identify criminals and detect illegal entry even in the absence of gunshots. Various law enforcement agencies across global are now deploying various strategies in order to control the increased incidents related to the mass shooting, which will further increase the demand and interest in advanced gunshot detection technology.

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At the same time, the factors that are limiting the market are the scarcity of gunshot detection professionals, the high installation cost of gunshot detection systems. Also, worldwide financial uncertainty and macroeconomic situations such as currency exchange rates and economic difficulties are some of the major challenges which are hampering the growth of Gunshot Detection System Market.

Based on the installation, the vehicle installations segment is expected to account for the largest share of the gunshot detection system market over the period. The growth in the vehicle installations segment is due to the upgradation of existing armored vehicle fleets. The increasing delivery of new armored vehicles is also leading to increasing demand for armored vehicles. Increasing soldier modernization programs are also contributing to the demand for overall GDS.

The global gunshot detection systems market is mainly dominating by Raytheon products, both in military and civilian applications. Shotspotter, which sells civilian versions of Raytheons gunshot detection system through the license, generated a revenue of around XX million in 2017. The product obtaining cost for gunshot detection systems is very low. The civilian usage of gunshot detection systems is gradually increasing across various countries, with the threat from random shooters and terrorists augmenting the demand from civilians.

North America is expected to drive the growth of the gunshot detection system market during the forecast period, 2017 to 2026. The gunshot detection system market in North America is expected to witness growth, because of the largest civilian gunfire deaths in the region, especially in the US where the firearm-related death rate is 25 times higher than other countries. Major US cities are planning to install indoor gunshot detection systems at universities, corporate office locations, and financial facilities, among others, over the next few years.

The objective of the report is to present a comprehensive assessment of the market and contains thoughtful insights, facts, historical data, industry-validated market data and projections with a suitable set of assumptions and methodology. The report also helps in understanding the Global Gunshot Detection System Market dynamics, structure by identifying and analysing the market segments and project the global market size. Further, the report also focuses on the competitive analysis of key players by product, price, financial position, product portfolio, growth strategies, and regional presence. The report also provides PEST analysis, PORTERs analysis, and SWOT analysis to address questions of shareholders to prioritizing the efforts and investment in the near future to the emerging segment in the Global Gunshot Detection System Market.

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Scope of Global Gunshot Detection System Market:

Global Gunshot Detection System Market, By Type:

Indoor OutdoorGlobal Gunshot Detection System Market,By Installation:

Fixed Installation Wearable Installation Vehicle Mounted InstallationGlobal Gunshot Detection System Market, By Application:

Military Law EnforcementGlobal Gunshot Detection System Market, By Region:

North America Europe Asia-Pacific South America Middle East & AfricaKey Players Operated in Market Include:

Raytheon Company Thales Group Battelle Memorial Institute Rafael Safran Electronics & Defense Rheinmetall AG ELTA Systems Ltd

MAJOR TOC OF THE REPORT

Chapter One: Gunshot Detection System Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Gunshot Detection System Market Competition, by Players

Chapter Four: Global Gunshot Detection System Market Size by Regions

Chapter Five: North America Gunshot Detection System Revenue by Countries

Chapter Six: Europe Gunshot Detection System Revenue by Countries

Chapter Seven: Asia-Pacific Gunshot Detection System Revenue by Countries

Chapter Eight: South America Gunshot Detection System Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Gunshot Detection System by Countries

Chapter Ten: Global Gunshot Detection System Market Segment by Type

Chapter Eleven: Global Gunshot Detection System Market Segment by Application

Chapter Twelve: Global Gunshot Detection System Market Size Forecast (2019-2026)

Browse Full Report with Facts and Figures of Gunshot Detection System Market Report at:https://www.maximizemarketresearch.com/market-report/global-gunshot-detection-system-market/29340/

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Global Gene Therapy Market 2019 exhibiting a CAGR of 22.07% till 2025 - Fusion Science Academy

From our Obsession

New thinking is required to serve an aging population.

Scientists know a lot about the hallmarks of different types of dementia. Alzheimers disease is characterized by buildups of amyloid and tau proteins. Vascular dementia is the result of gnarled and broken blood vessels that normally supply oxygen to the brain. Parkinsons disease and other Lewy Body dementias are caused by misshapen alpha-synuclein proteins in the brain.

Conventional wisdom has it that each of these dementias needs its own treatment. An anti-amyloid drug probably wouldnt work for someone who doesnt have amyloid buildups in their brain.

But to this day, there are no definitive treatmentsor preventive measuresfor any of the dozens of dementias out there. Which has led some researchers to take a more systematic approach: What if there were a single mechanism in the brain that, when faulty, leads to all kinds of dementias? And what if this mechanism, like a switch, could be flipped off?

Thats the thinking of Michael Fossel, the founder of the Michigan-based biotech startup Telocyte, which is developing treatments for Alzheimers. Today (Jan. 14), Fossel published a review articlepostulating that Alzheimers and other dementias are caused by a failing of a workhorse class of brain cells called glia. He also proposes that he and his colleagues at Telocyte, founded in 2015, have a solution: a gene therapy that could target these cells to keep dementia at bay.

The paper is theoreticalits a review, so its not presenting any original data. Its a new way of thinking, and a bold proposition. Its encouraging to see individuals like Dr. Fossel pulling together research and trying to come up with new theories, says Rebecca Edelmayer, the director of scientific engagement at the Alzheimers Association. The Alzheimers Association is a nonprofit and publisher of Alzheimers and Dementia, the journal in which Fossels review article appeared.

But while theories are important, Edelmayer says, they also need to be tested. Gene therapies are still relatively new. And theres reason to wonder about the safety of the gene the therapy would introduce: one that codes for the enzyme telomerase. Before scientists can even begin to test Fossels systematic theory of dementia, theyll need a lot of data demonstrating its safety.

Telomerase has been a focus of longevity research for years. Its an enzyme that lengthens telomeres, which are the genetic caps on the end of our chromosomes. Every time cells divide, telomeres shortenand when telomeres have been sufficiently shaved away, cells enter a state called senescence and stop dividing. Then, they self-destruct.

Shorter telomeres have been correlated with a whole host of age-related health issues: cancer, diabetes, and even forms of dementia. But its not the telomeres themselves that cause these issues, Fossel suggests. As my telomeres shorten, there are a lot of other things going on, too, he says.

The relative length of telomeres, we know, sends a signal to the rest of the cells DNA. As telomeres shorten with cell replication, cells change the way they carry out other genetic instructions, which can result in shoddy protein production. Its a process called the telomere positron effect (paywall), and scientists still dont fully understand it.

Fossel posits that when telomeres shrink in microglial cells, part of the brains immune system, other critical parts of their DNA degrade, tooand that genetic damage can result in many different dementias.

Telocytes gene therapy would aim to rebuild those glial telomeres. That would involve sending an active copy of the telomerase gene, TERT, into the cerebrospinal fluid, carried by a virus. The virus, which should be otherwise benign, isnt great at getting genetic material into specific cells: in mouse models, about 5% of the total therapy winds up in neurons, to no lasting effect, and about 1% winds up in microglial cells, Fossel says. But even with the TERT gene just floating around in the glial cell for a few weeks or months, it might be enough for telomerase to lengthen those end caps and trick the cell into expressing genes like it did in its younger days.

Usually, gene therapies work by introducing new genetic material that replaces a persons faulty or missing genetic code. Telocytes gene therapy, however, wouldnt be replacing a gene: Itd just be giving glial cells another copy of one they already have. All of our cells have the TERT gene embedded in their chromosomes. But the vast majority of cells (save for red blood cells, sperm or egg cells, and cells along parts of the digestive tract) have the gene switched permanently off.

Thats for good reason: Telomerase is active in most forms of cancer. Which is why many scientists fear that inserting a gene that codes for telomeraselike Telocytes gene therapyrisks causing cancer

My main concern is its safety, says Jue Lin, molecular biologist at the University of California San Francisco whose work focuses on studying telomere length and stress levels over time. We dont know whether the over-expression of telomerase will increase the risk of cancer. In the brain, particularly in the glial cells that Fossels proposed gene therapy would target, the cancer in question would likely be glioblastoma, a ravenously growing brain tumor.

Mouse models using telomerase gene therapy in the brain have been promising, with no notable incidence of cancerbut those experiments are imperfect. Mice express telomerase differently than humans do, Lin explains: They have a lot more telomerase, in more tissues than humans. Mice also dont live as long as we do, and cancer takes a long time to develop, Lin says.

And gene therapies carry the risk of a dangerous immune reaction to the virus carrying the therapeutic gene. The viruses used in gene therapy todayand the one Fossel proposes usingshould be safer than the ones used in the early days of gene therapy. Adeno-associated virus, or AAV for short, should elicit only the tiniest of immune responses. But scientists have recently voiced concerns about the long-term safety of gene therapies using AAV.

Given the risks, theres disagreement over whether the telomerase approach is worth pursuing. Its important and interesting to have an additional hypothesis, says Diego Forero, a researcher at the School of Health Sciences at the Fundacin Universitaria del rea Andina in Colombia. His work, which is independent of Fossels, focuses on exposing astrocytes, a type of glial cell in the brain, to telomerase, to see how theyll react. Hes found that telomerase is involved in other cellular functions, like a cells metabolism. In his opinion, its too early to say that Fossels theory should be tested.

Rather than focusing on the potential therapeutic application of telomerase in brain cells, Forero is interested in more basic, exploratory research. He thinks that applying it to a specific targetlike a cure for dementiawouldnt tell scientists enough about all the ways telomerase could affect brain cells.

Those calls for prudence can be frustrating for dementia patients facing a dearth of options. Even with no immediate plans to conduct clinical trials, Fossel says he has already had some 200 people with mild to moderate dementia reach out to him as willing participants. Theyre ineligible for most other clinical trials for dementia therapies, which tend to seek out participants who have risk factors of the disease but minimal symptomsor none at all.

People have faced terrible disease and said Im going to take my chances, says Arthur Caplan, a bioethicist at New York Universitys Langone Medical Center. With vulnerable populations desperate for treatment, peer review from independent scientists becomes even more important. Its critical that the data and research are conducted by parties that dont have a vested financial interest in a certain outcome.

These studies also need to have strong institutional review boards, Caplan says. These boards are required any time researchers are conducting experiments with human subjectsespecially when the risks are so high.

Libella Gene Therapeutics, a Kansas-based biotech startup, is beginning a clinical trial for a telomerase gene therapy to treat broad aging this year. However, its taken its work to Colombia, where the standards for institutional review boards arent as high as they are in the US. Its a tactic informally known as IRB shopping, and it raises eyebrows in the research community.

Were always open to new ideas and novel ways [to treat dementia, Edelmayer says. We have to leave no stone unturned. But, she continued, one of the biggest things we want to see is not just theories. We want to see them tested.

Continued here:
Could a gene therapy cure dementia? - Quartz

After a manufacturing specification delay, Bluebird Bio has officially launched Zynteglo, its first gene therapy, andtapped a new leader to run the operation.

Nicola Heffron, a former exec with Celgene, Shire and GlaxoSmithKline, has joined Bluebird as its European chief, Bloomberg reported, just as the company gets its rollout underway there. She is replacing Andrew Obenshain, whos moving up to the global leadership team.

Heffron is tasked with charting the course for Bluebirds first commercial launch of its first product, Zynteglo, to treatbeta thalassemia, a rare inherited disease marked by reduced production of oxygen-carrying hemoglobin in red blood cells.

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And Heffron knows a thing or two about blood disorders. Before Bluebird, she headed up global marketing for Celgenes myeloid portfolio, her LinkedIn profile shows.

Wednesday, Bluebird revealed that the $1.76 million-per-treatment gene therapy is now available in Germany. The University Hospitalof Heidelberg serves as the drugs first qualified treatment center inthe country, and Bluebird said its working with institutions that have expertise in stem cell transplant as well as in treating patients with [beta thalassemia] to establish more centers.

RELATED:Bluebird Bio readies Zynteglo launch as EU approves 'refined' manufacturing

During a presentation at the annual J.P. Morgan Healthcare Conference on Tuesday, Bluebird CEO Nick Leschly confirmed the companysvalue-based payment model for the costly onetime therapy. Under that arrangement,payments of 315,000 ($351,000) each are made in five installments over five years. Except for the first round of expenses, payers only pay the rest if Zynteglo delivers on its therapeutic promise.

So far, the reimbursement agreements Bluebird has penned using that model can coverabout half of Germanys patients, according to Leschly.

Novartis has also rolled out a similar program for its $2.1 million spinal muscular atrophy gene therapy Zolgensma.

Bluebird won its European nod for Zynteglo last year but delayed the launch after a manufacturing specification hiccup. The Cambridge, Massachusetts-based biotech has established a manufacturing network that includes both internal facilitiesand contract partners for itslentiviral vector and drug product, Leschly said Tuesday.

RELATED:ASH: Bluebird's multiple myeloma CAR-T follow-up shows promise in phase 1

In the U.S., Bluebird has started itsrolling submission for approval and is in talks with the FDA regarding the requirements and timing of the various components of the application. Its expecting to finish the process in the first half of 2020.

Outside of beta thalassemia, Bluebird is testing the same drug, also known as LentiGlobin, in sickle cell disease, which is also marked by an abnormality in hemoglobin.

According to phase 1/2 data presented at last years American Society of Hematology annual meeting in December, none of the 17 patients enrolled in group Cwhich used an improved stem cell harvest technique and a new manufacturing processrequired regular blood transfusions post-treatment. Nine patients had beenfollowed for at least six months at that data cutoff.

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Bluebird Bio taps ex-Celgene exec Heffron to lead its first gene therapy launch - FiercePharma

SAN RAFAEL, Calif., Jan. 13, 2020 /PRNewswire/ --BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a pioneer in treatments for the rare disease Phenylketonuria (PKU) and in gene therapy clinical research, announced today that both the U.S. Food and Drug Administration (FDA) and the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. have granted the Company Investigational New Drug (IND) status and approved its Clinical Trial Application (CTA), respectively, for its investigational gene therapy candidate BMN 307. BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU. BMN 307 will be evaluated to determine whether a single dose of treatment can restore natural Phe metabolism, normalize plasma Phe levels, and enable a normal diet in patients with PKU.

The Company expects to start dosing patients in PHEARLESS, a Phase 1/2 study, in the first quarter of 2020 with product made at commercial scale from its award-winning gene therapy manufacturing facility. The Company is actively preparing regulatory submissions to open additional clinical sites in other countries.BMN 307 represents a potential third PKU treatment option from BioMarin and its second gene therapy clinical program. Both the FDA and European Medicines Agency have granted BMN 307 Orphan Status.

BMN 307 follows BioMarin's first investigational gene therapy clinical program: valoctocogene roxaparvovec to treat severe hemophilia A, currently in Phase 3. Based on data from an interim analysis of the ongoing Phase 3 study, the European Medicines Agency validated a Marketing Authorization Application in the EU for valoctocogene roxaparvovec, and the review process has begun. The Company also has submitted a Biologics License Application to the FDA and anticipates the review to begin in February 2020.

"With BMN 307, we are joining together our expertise in PKU biology and the knowledge we have gained from developing the only two approved therapies for PKU with our understanding of gene therapy clinical development and manufacturing from our valoctocogene roxaparvovec experience," said Hank Fuchs, President, Worldwide Research and Development at BioMarin."BioMarin has stood with the PKU community for over 15 years and remains dedicated to continuing to increase the body of medical knowledge in this devastating disease."

PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down Phe, an amino acid that is commonly found in many foods. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychological issues, affecting the way a person thinks, feels, and acts. Due to the seriousness of these symptoms, in many countries infants are screened at birth to ensure early diagnosis and treatment to avoid intellectual disability and other complications. According to treatment guidelines, PKU patients should maintain lifelong control of their Phe levels.

BMN 307 Clinical Program

BioMarin's clinical program is composed of two key studies. PHEARLESS, a Phase 1/2 study, will evaluate the safety, efficacy, and tolerability of a single intravenous administration of BMN 307 in patients with PKU. The study consists of a dose-escalation phase, followed by a cohort expansion phase once an initially efficacious dose has been demonstrated. In addition, BioMarin is sponsoring an observational study, PHENOM, which has already started enrolling patients with PKU to measure both established and new markers of disease and clinical outcomes over time.

BioMarin's 15-Plus Year Commitment to PKU Research

For more than 15 years, BioMarin has been a pioneer in ongoing research to help improve the lives of PKU patients. BioMarin has treated approximately 7,000 PKU patients around the world. The company has two approved PKU therapies, and the investigational gene therapy BMN 307 is currently in development. BioMarin has conducted 40 clinical studies in PKU and has sponsored 38 external clinical studies. BioMarin researchers have authored 54 publications in medical and scientific journals on PKU and supported another 52 publications by external researchers.

About Gene Therapy

Gene therapy is a form of treatment designed to address a genetic problem by adding a normal copy of the defective gene. The functional gene is inserted into a vector containing a small DNA sequence that acts as a delivery mechanism, providing the ability to deliver the functional gene to targeted cells. The cells can then use the information from the normal gene to build the functional proteins that the body needs, potentially reducing or eliminating the cause of the disease.

Gene Therapy Manufacturing

BioMarin has leveraged its knowledge and experience in manufacturing complex biological products to design, construct and validate a state-of-the-art vector production facility in Novato, California. This facility is the site of production for both valoctocogene roxaparvovec and BMN 307. Manufacturing capabilitiesare an essential driver for our gene therapy programs and allows us to control quality, capacity, costs and scheduling enabling rapid development. Manufacture of BMN 307 was performed with a commercial ready process at scale in this facility. Production of BMN 307 with a commercial ready process at scale reduces risk associated with making process changes later in development and will speed overall development timelines significantly.

Ongoing process development efforts and experience gained at commercial scale have led to improvements in productivity and operational efficiency. The ability to scale out the facility with additional equipment combined with the improvements in productivity result in a doubling of overall potential capacity to 10,000 doses per year, combined for both products, depending on final dose and product mix. This improvement in productivity is anticipated to meet both commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well in to the future.

About Phenylketonuria

PKU, or PAH deficiency, is a genetic disorder affecting approximately 50,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most patients to adhere to the life-long strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.

To learn more about PKU and PAH deficiency, please visit http://www.PKU.com. Information on this website is not incorporated by reference into this press release.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases.The company's portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates.For additional information, please visitwww.biomarin.com. Information on such website is not incorporated by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the development of BioMarin's BMN 307 program generally, BioMarin's planned submissions to regulatory authorities for BMN 307, BioMarin's gene therapy manufacturing capabilities, the impact of using material manufactured at commercial scale in a clinical trial, the timing and results of BioMarin's planned Phase 1/2 trial of BMN 307, and the review of marketing applications for valoctocogene roxaparvovec. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others:results and timing of current and planned preclinical studies and clinical trials of BMN 307; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities; uncertainties inherent in research and development, including unfavorable new clinical data and additional analyses of existing clinical data; the results and timing of current and future clinical trials related to BMN 307; our ability to reproducibly and consistently manufacture sufficient quantities of BMN 307, the possibility that changes may be required to the current manufacturing process; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended September 30, 2019 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

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BioMarin, Pioneer in Phenylketonuria, to Begin Clinical Trial with BMN 307 Gene Therapy - PRNewswire

Precision BioSciences, a Durham-based genome editing company, has reached two regulatory milestones for its potential therapy against multiple myeloma, a chronic cancer of white blood cells.

The U.S Food and Drug Administration (FDA) has accepted the companys Investigational New Drug (IND) application and also granted the therapy Orphan Drug Designation, a status that gives companies tax reductions and other incentives to develop treatments for rare diseases.

The therapy, designated as PBCAR269A, is Precisions third allogeneic chimeric antigen receptor (CAR) T cell therapy candidate.

FDA acceptance of the IND for PBCAR269A further underscores the ongoing progress in our allogeneic CAR T pipeline, said Matt Kane, co-founder and chief executive officer of Precision BioSciences. We have now moved three CAR T programs from preclinical to clinical stage development since April 2019, and we look forward to continuing to advance our allogeneic CAR T portfolio to bring these novel therapeutic candidates to patients.

Matthew Kane

The company plans to begin a Phase 1 clinical trial of the therapy this spring at multiple sites using material produced at its own manufacturing facility in Durham. About 48 patients are expected to be enrolled.

For more information about the trial, visitwww.clinicaltrials.gov, and enter study identifier number NCT04171843.

In preclinical disease models, PBCAR269A has demonstrated no evidence of graft-versus-host disease at doses that resulted in potent anti-tumor activity, said Chris Heery, M.D., chief medical officer of Precision BioSciences. There remains significant unmet need in the treatment of relapsed/refractory multiple myeloma, and we are excited to begin clinical trials with an off-the-shelf CAR T therapy candidate in this setting.

The IND for PBCAR269A builds on the initial clinical data Precision presented in late 2019 for its lead program, a CAR T therapy for treating non Hodgkins lymphoma and B-cellacute lymphoblastic leukemia, and the FDAs acceptance of an IND for another CAR T therapy for treating non-Hodgkins lymphoma, chronic lymphocytic leukemia and small lymphocytic lymphoma.

Precision BioSciences harnesses T cells, a type of white blood cell that is vital to the adaptive immune systems ability to identify specific antigens and destroy pathogens. Through the companys CAR T technology, the T cells can be directed to kill cancer cells.

Precision produces the CAR T therapies by selecting T cells derived from healthy donors as starting material. Then, using its proprietary ARCUS genome-editing technology, the company modifies the donor T cells.

Scientists insert the CAR gene at the T cell receptor locus, enabling the T cell to target a specific marker on a cancer cell, while knocking out the T cell receptor to prevent the patients immune system from recognizing and attacking the T cells.

The company optimizes its CAR T therapy candidates for immune cell expansion in the body by maintaining a high proportion of certain types of CAR T cells throughout the manufacturing process and in the final product.

The process creates a consistent product that can be reliably and rapidly manufactured and is designed to prevent graft-versus-host disease, normally a major challenge when inserting foreign or altered cells or tissues into the body.

The company has posted a four-minutevideoon its website to explain CAR T therapy, using Samurai warriors as an analogy.

Last July Precision opened its Manufacturing Center for Advanced Therapeutics (MCAT), the first in-house current Good Manufacturing Process (cGMP)-compliant manufacturing facility in the United States dedicated to genome-edited, off-the-shelf chimeric antigen receptor CAR T cell therapy products.

Precision Bio facility

Given the potential output of our platform, weve known from the beginning that it was critical for us to address the need for scalable manufacturing of cell-therapy products in order to be able to effectively deliver them to patients, Kane said when the facility opened. In addition to our clinical work, it also has the potential to be a commercial launch facility with the capacity to generate up to 10,000 doses of CAR T cell therapies and 4,000 doses of gene therapies per year.

The facility can produce three different drug substances: allogeneic CAR T cells, messenger RNA and adeno-associated viral vectors. It was designed to meet regulatory requirements in the United States, Europe and Japan.

In addition to health care, Precisions ARCUS genome-editing platform has applications in food and agriculture.

In 2018 the company created a new name and brand identity, Elo Life Systems, for its food and agriculture business, previously known as Precision PlantSciences, based in Research Triangle Park.

Elo is using the ARCUS platform and other new technologies for applications in crop improvement, animal genetics, industrial biotechnology and sustainable agriculture.

Since it was spun out of Duke University in 2006, Precision raised about $300 million in venture capital, government grants and collaboration agreements. The company went public in March 2019, grossing $145.4 million in an initial public offering of stock.

The companys shares are listed on the Nasdaq Global Select Market under the ticker symbol DTIL, shorthand for the companys marketing tagline, Dedicated to improving life.

(C) N.C. Biotech Center

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Precision BioSciences hits two key FDA milestones in advancing gene therapy for cancer - WRAL Tech Wire

SAN FRANCISCO Pharmaceutical companies and their investors have grown accustomed to big news kicking off the year, specifically multibillion-dollar deals.

The last three J.P. Morgan Healthcare Conferences, considered a sort of Opening Day for the industry, were hallmarked by the acquisitions of Ariad Pharmaceuticals, Impact Biomedicines and Loxo Oncology. (And that's not including Bristol-Myers Squibb's $74 billion deal for Celgene the week before the meeting last year.)

In fact, Eli Lilly pressed Loxo for a quick buyout to have something flashy to announce at last year's conference. Such enthusiasm was noticeably absent this time around, though, resulting in a quieter first day than biotech shareholders had hoped for.

The Nasdaq Biotechnology Index fell almost 2%, with Sage Therapeutics, Clovis Oncology and other potential takeover targets trading down by market's close. Brad Loncar, a founder of biotech exchange-traded funds, noted on Twitter how even shares of MorphoSys fell despite the German drugmaker having the most positive news of the day.

While major M&A announcements seem unlikely for the rest of the week, industry experts still expect the challenges facing bigger companies will result in a healthy number of deals in 2020. In the meantime, biotechs will be busy trying to deliver on their development plans some of which were provided in more detail during Monday's presentations.

Connecticut-based Alexion Pharmaceuticals is best known for its high-priced rare disease drugs Soliris and Ultomiris. On Monday, the company gave an early look at full-year financials, reporting a top line revenue increase of more than 20% between 2018 and 2019. That growth correlates to, at the very least, roughly $4.96 billion in annual revenue, which would be slightly higher than the average analyst estimate.

For Stifel's Paul Matteis, more surprising than the revenue beat was Alexion's plan to treat four times as many U.S. neurology patients with Soliris and Ultomiris by 2025. If successful, the plan would create a "substantial upside" to revenue estimates, according to the analyst.

"This of course raises a number of natural questions," Matteis wrote in a note to investors, "such as where will this growth come from, and what does it assume (if anything) for additional neuro indications where Soliris/Ultomiris isn't derisked."

Shareholders, however, responded positively to the updates, sending Alexion shares up 4%.

Soliris is approved to treat several diseases, including a chronic neuromuscular illness known as gMG and a type of central nervous system inflammation abbreviated as NMOSD. Alexion says that, in less than two years time, these neurology indications have become its largest franchise by patient volume. By the end of 2019, almost 1,900 U.S. neurology patients were taking Soliris.

Ultomiris, a follow-on to Soliris, is under investigation as a treatment for gMG and NMOSD across a couple of late-stage studies. And on Tuesday, Alexion announced it will soon begin a Phase 3 study of the drug in ALS, with plans to enroll 350 adults in a 50-week trial.

Selling new paths to growth is particularly important for Alexion now, as the company has come under pressure from activist investor Elliott Advisors to seek a sale.

BioMarin could bring the first hemophilia gene therapy to market later this year. While waiting for regulators to confirm its approval application is under review, the California biotech announced Monday it has more than doubled capacity at a gene therapy plant. Altogether, the facility can make up to 10,000 doses each year of either the hemophilia treatment or a separate BioMarin gene therapy that's about to begin human testing.

That capacity level, according to executives, would allow the company to treat all U.S. hemophilia A patients in roughly two year's time. The update increases the competitive pressure on drugmakers with marketed products, such as Takeda and Novo Nordisk, as well as those working on rival hemophilia gene therapies. Swiss pharma giant Roche falls into both buckets, and could lose out on many patients because of BioMarin, according to a recent doctor survey from Citi Research.

Sarepta Therapeutics also had a manufacturing update, announcing that production for its experimental micro-dystrophin gene therapy is now large enough to be considered commercially viable.

RBC Capital Markets analyst Brian Abrahams called this a "critical manufacturing milestone" for Sarepta, one that shores up the timeline for a pivotal study scheduled to start sometime in the middle of the year.

Despite launching one of the industry's largest initial public offerings in 2018, Rubius Therapeutics has struggled out of the gate in getting its first clinical data. While its ambitions are large, the Flagship Pioneering-backed biotech failed to meet expectations it set for delivering early Phase 1 data from its lead asset, a PKU drug, by the end of 2019.

"We understand what we didn't do right in 2019, and we are doing it differently in 2020," CEO Pablo Cagnoni told a half-filled breakout room at the JPM conference. "We will deliver in 2020."

While Cagnoni and other executives emphasized cancer therapies set to enter clinical testing in 2020 and speedy progress on its own manufacturing plant, the biotech also admitted it still has not dosed a single PKU patient to date, calling into question the company's ability to execute.

Pressuring Rubius further is BioMarin's announcement that it will develop a PKU gene therapy, with plans to dose its first patient this quarter. BioMarin already sells two PKU drugs and could be a formidable competitor.

Shares in Rubius ticked down by about 5% Monday and have shed two-thirds of their value since the company went public.

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Alexion's neuro bet, Rubius' trial troubles and biotech's gene therapy milestones - BioPharma Dive

SAN FRANCISCO, Jan. 14, 2020 /PRNewswire/ --GenScript Biotech Corp., a leading global biotechnology group and a pioneer in the field of gene synthesis, today held its inaugural "Global Forum on Cell & Gene Therapy and the Booming China Market," during the JP Morgan Healthcare Conference week, attracting hundreds of industry leaders, investors and others to address the challenges and opportunities in this innovative field.

"As an industry, we are on the brink of achieving some extraordinary breakthroughs in cell and gene therapy for cancer and other diseases," said GenScript Biotech CEO Frank Zhang, PhD. "Four gene and cell therapies have recently been approved by the FDA, bringing new hope to patients, and this is only the beginning. Our vision is to make cancer a chronic or curable disease rather than a deadly one, and to transform the treatment of cancer, autoimmune and other diseases by leveraging the advantages of cell and gene therapy."

While significant advances are being made, the Forum also tackled some of the more pressing challenges, such as mitigating treatment side effects, improving treatment efficacy in solid tumors and scaling up manufacturing. Panelists from Kite Pharma, GE Healthcare Life Sciences, Ziopharm Oncology, Oxford Biomedica, Genethon, CARsgen Therapeutics, J&J Innovation Asia Pacific, the American Society of Gene & Cell Therapy, Loncar Investment, Lilly Asia Ventures, and many others participated in the event.

In the U.S. alone, the U.S. Food and Drug Administration is expected to approve 40-60 cell and gene therapies by 2030. During a panel discussion focused on regulatory issues, experts considered what regulators will need to do to keep up with the rapid pace of innovation, the new hospital-based regulatory pathway in China, how to ensure quality through the manufacturing process, and the challenges and opportunities that come with regulatory harmonization among different countries.

China continues to attract significant attention from industry and investors and is poised to grow even more. During his welcoming remarks, Zhang noted China's emergence as a global economic leader, with a projected $1.1 trillion spend on healthcare this year, as well as the growing disease burden in China. By 2030, an estimated 4.3 million Chinese will be diagnosed with one of the 14 major cancers, according to research from IMS Health. Panelists addressed issues such as the amount of capital required to achieve scale in China, and advantages of the market in China.

"The drug development business is changing rapidly and China is at the fore in a number of ways," Zhang said. "Biotech and pharma companies do not need or desire to have the infrastructure to scale their drugs through commercialization. With lower costs, China is a natural place for companies to contract out costly development and manufacturing to organizations that have the expertise and experience to collaborate with them through the entire discovery to development lifecycle."

For its part, GenScript has put significant resources into its Contract Development and Manufacturing Organization (CDMO) business to meet the increasing demand. In 2018, the company officially launched its biologics CDMO segment, and last year opened a new GMP compliant biologics research center. GenScript is also leading the way in cell therapy through its antibody discovery service and plasmid and virus production capabilities.

InDecember2017,LegendandJanssen, a Johnson & Johnson business,enteredintoaworldwidecollaborationandlicenseagreementtodevelop,manufactureandcommercializeLCARB38M(JNJ4528)inmultiplemyeloma. The companies are advancing this investigational B-cell maturation antigen (BCMA) targeted chimeric antigen receptor (CAR) T-cell therapy in previously treated patients with the disease. Clinical trials are ongoing in both the U.S. and China and the therapy has received orphan drug designation and breakthrough treatment designation from the FDA in the U.S. and PRIME designation from the European Medicines Agency.

GenScript is one of only a few Chinese companies to have a significant presence during JPM week. Based on the success of this year's Forum and the interest in cell and gene therapy, GenScript will host its second annual Forum with influential leaders on the topic again next year during JPM week at the San Francisco Grant Hyatt.

About the GenScript Biotech Global Forum

The GenScript Biotech Global Forum, with the theme "Cell & Gene Therapy and the Booming China Market," brings together industry, investors, government and academia to learn, collaborate and become inspired and engaged in the advancement of cell and gene therapy in China and across the globe. As the most exclusive annual gathering of global cell and gene therapy leaders during the JP Morgan week, the inaugural Forum in 2020 attracted more than 700 attendees gathered in San Francisco, representing leading biotech and pharma companies, cell and gene therapy innovators, top universities and government agencies. The forum aims to build a bridge between the cell therapy industry and capital community, accelerating mutual understanding and creating collaboration opportunities.

About GenScript Biotechnology

GenScript Biotech Corporation (Stock Code: 1548.HK) is a leading global biotechnology group and a pioneer in the field of gene synthesis. The companyoperates in four main categories: life sciences CRO services, biologics development and manufacturing, industrial biology and cell therapy.

Founded in 2002 and listed on the Hong Kong Stock Exchange in 2015, GenScript has established a global presence across Greater China, North America, the EU, and Asia Pacific. Today, over 100,000 customers from over 160 countries and regions around the world use GenScript's premier, convenient, and reliable products and services.

GenScript currently has more than 2,900 employees globally, 34 percent of whom hold master's and/or doctorate degrees. In addition, GenScript has a number of leading commercial products, more than 100 patents and 270 patent applications. As of June 2019, GenScript's products and services have been cited in 40,300 scientific papers worldwide.

GenScript is committed to being the most reliable biotech company in the world and to achieving our vision of making humankind and nature healthier through biotechnology.

For more information, please visit https://www.genscript.com/

Contact:

Corporate:Fiona CheCorporate Communications Manager, GenScript+86 -025-58897288-6321 Fiona.che@genscript.com

Media Susan ThomasPrincipal, Endpoint Communications(619) 540-9195susan@endpointcommunications.net

SOURCE Genscript Biotech Corporation

http://www.genscript.com

Link:
GenScript Biotech Global Forum Highlights Advances in Cell and Gene Therapy and Opportunities in China - PRNewswire

International companies recognise the appeal of the UK cell and gene therapy ecosystem and are sponsoring the majority of the UK commercial clinical trials which account for 77% of the total 127 ongoing trials.

London UK, 15th January 2020 - The Cell and Gene Therapy Catapult (CGT Catapult) today released their 2019 UK Advanced Therapy Medicinal Products (ATMPs) clinical trials database showing that the 127 ongoing trials represent a ~45% increase compared to 2018, and account for 12% of the total global cell and gene therapy clinical studies.

The NHS and UK ecosystem are providing the right platforms to allow innovative therapies to progress through to the clinic in ever increasing numbers. Cell and gene therapies require novel approaches, systems and infrastructure to enable the delivery of these life changing, living medicines to patients. The favourable UK environment is being recognised internationally with the majority of commercially sponsored trials being backed by international organisations.

Cell and gene therapies continue to progress towards large scale commercialisation to add to the therapies already approved for use. The database shows that 77% of UK cell and gene therapy trials are now sponsored by commercial organisations compared to only 25% in 2013. This number reflects the continuous investment that companies are making in cell and gene therapies, and the transformative effects that these therapies are proving to have on patients lives.

The main indication for cell and gene therapies clinical trials remains oncology (39%) followed by ophthalmology (13%) and haematology (12%).

Health Minister Baroness Blackwood said:

These extraordinary figures show the UKs life sciences sector is leading the world in getting cutting-edge treatments to NHS patients as quickly as possible. As our research capabilities continue to grow, our exceptional NHS as a whole will be able to reap the benefits of not only more innovative medicines for patients, but investment from companies seeking outstanding expertise.

Ian Campbell, Interim Executive Chair, Innovate UK commented:

The UK is a world leader in developing innovative therapies. Through continued support from government, we are building a network of excellence which brings together research and commercial organisations. With the Cell and Gene Therapy Catapult playing a key role in advancing the development of these therapies, this will not only bring concrete benefits to patients but will also allow the UK sector to compete globally.

Keith Thompson, Chief Executive Officer, Cell and Gene Therapy Catapult stated:

The total number of cell and gene therapy clinical trials in the UK has been increasing consistently by an average of 25% year on year since 2013. This has been enabled by the development of the UKs fantastic ecosystem to support the development and clinical adoption of cell and gene therapies. The infrastructure and initiatives that have been put in place, with strong backing by the government, including the Advanced Therapy Treatment Centres network, are giving companies the confidence to setup and run their innovative clinical studies here. The result, is that we are now seeing therapies moving from academic projects towards becoming commercial products that can be delivered at scale by the NHS.

The report and database can be downloaded at ct.catapult.org.uk/clinical-trials-database

About Cell and Gene Therapy Catapult:

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 200 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK. For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

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The UK and the NHS are providing the right environment and infrastructure to allow innovative cell and gene therapies to reach patients. -...

In 2019 a Novartis drug called Zolgensma got approved and made huge headlines. The headlines were less about the fact that this one time treatment can treat spinal muscular atrophy (a condition occurring in 1 out of 10,000 newborns and limiting their life to often mere two years) but about it being the most expensive drug ever.

In the United States the list price of Zolgensma stands over $2 million. The one time treatment is a so-called gene therapy that injects a virus aimed at fixing the failed or mutated gene responsible for motoric muscle functions. While this treatment is good news for many patients, it also fueled the debate on the ethics of high drug prices.

I would like to provide a few fresh, not fully reflected, and hopefully provoking thoughts for the new year on what policy makers should keep in mind when aiming to control drug prices.

So why dont we just cap the prices per drug?

Two million dollars per patient is definitely a crazy high amount. The reason why it cant be offered much cheaper right now is that there are (fortunately) not that many patients that actually need this treatment. The drug manufacturer has a very small target audience they can charge for this treatment of a rare disease. From that small pool of people they need to recoup their development costs for this drug, make up for totally failed projects, and make shareholders happy with a profit margin.

This sounds cruel So why dont we have stronger laws on capping such excesses? If we legislate a maximum cap to how much a drug per patient is allowed to cost, we will end up with pharmaceutical research aimed at treating and curing common diseases and patients with rare diseases will have a bleak future.

Why are there so often drugs that are similar to already existing drugs and dont focus on curing diseases we cant cure or treat yet?

Medical research is unfortunately a shotgun. When starting to develop a molecule or target a genetic defect you never really know where you end up. On average, only one of every 5,000-10,000 substances synthesized in research facilities will make it successfully through all stages of product development to become an approved drug. Many projects and even entire biotech companies fail to bring even one product to the commercial stage. Investing in life sciences requires a very healthy appetite for risk, and hence an incentive scheme that rewards those able to create value with their inventions is required. By the time a medical drug reaches the regular patient, an average of 12.5 years will have elapsed since the first discovery of the new active substance. The total investments needed to get to one active substance that can be accessed by a patient is around two billion Euros.

Why is delinkage is a bad, very bad, idea?

There are big proponents within the World Health Organization and some southern European governments to de-link the research and production of medical drugs. Supporters of this so-called delinkage support publicly funded R&D of drugs and private companies merely charging for their production costs plus a modest markup. This semi-planned pharma economy should frighten all those who are still waiting for medical innovations to cure their or their loved ones from medical conditions. Medical innovation (or to be more precise the lack of it) in planned economies such as the Soviet Union is a solid benchmark of why this is a bad idea. Research funding has been politicized in such systems and only funneled into pet projects of apparatchiks or (in the best case) to very common diseases the majority of voters are interested in.

Side note: The Soviet Union put in a lot of effort to develop cutting-edge doping drugs for their Olympic athletes but neglected researching actual cures. An example is the story of a Russian scientist who smuggled in a drug with the help of Mayo Clinic practitioners to treat his daughters tuberculous meningitis was even sentenced to death for being in contact with an American scientist.

So how can things stop getting more expensive?

Critics of the innovative pharmaceutical industry often make the point that pharma has an incentive to not fully cure patients as they only make money with sick patients. The same argument can be made about hospitals and outpatient doctors. There are historic capitation examples in which doctors only get paid per healthy patient month (I was told by someone very smart that this used to be the case in ancient Chinese villages). These concepts obviously work much better in a super-low-tech health system or for the reimbursement of providers only. Adding pharmaceutical research into this equation makes things definitely hard; but not impossible.

So would it be possible to have fully integrated healthcare companies that are providers, payers, and pharma industry at the same time?

A rather crazy thought experiment: I just googledvertical integration pharmaceuticals and insuranceand surprisingly couldnt find much. My healthcare management textbook told me a decade ago that integrating (or collaborating) with pharma would be called a diagonal collaboration as pharma and med tech are often not even seen as a proper part of the health system. A vertically integrated healthcare company owning drug patents and having patent cross license agreements with similar companies would have a higher incentive to provide its patients/insurance population with all drugs it has access to. Given the growing importance of health data both in treating patients (patient value chain) but also finding cures could be massively leveraged in such an integrated organization. The main profits would be generated through insurance premiums and drug sales to markets the insurance is not active in or to competitors.

This could be an interesting market response to realigning incentives in private healthcare and healthcare in general. The biggest roadblocks and reasons why this has not happened yet are:

Will the next big thing come out of a garage lab?

I recently watched the very entertaining Netflix documentary seriesUnnatural Selectionthat shows both the significant advances of gene therapy by established pharma companies and some outlaw biohacker activists that try to democratize biotech research through their garage labs. While most of these biohackers seem to be overly optimistic of their chances to develop effective cures and genetic improvements, one should not reject the possibility of disruption within the biotech industry.

The big consumer-reaching advances of the computer and software industry happened eventually in Californian and New Mexican garages and not in the labs of IBM or GE. Gene editing techniques such as CRISPR bring a lot of hope to patients and families struggling with genetic diseases. And first advances with this technology in agriculture and animal health suggest that treatments can be developed in relatively small and inexpensive labs and research environments.

So theres a possibility that the competition density in the biotech industry will intensify and market entry barriers be lowered due to gene editing technologies. At the same time regulatory barriers and approval requirements need to adopt to these technologies, allow them, and take into account that too high barriers will keep competition low and hence prices for new treatments high.

Quick and easy one to save Billions in 2020:Scrap value added and all sales taxes from prescription medicine. In some countries this would lead to a reduction of drug prices by 25%! It is also more than sad that some developing countries still levy tariffs on imported innovative medicines.

Please feel free to chip in to this debate. It is important to keep an open mind and find new ways to fund innovative medicine. At the same time it is important to stick to the realities of medical research and innovation.

A less futuristic but more public policy oriented paper on The Consumer Case for Intellectual Property authored by me can be foundhere.

Fred Roeder has been working in the field of grassroots activism for over eight years. He is a Health Economist from Germany and has worked in healthcare reform and market access in North America, Europe, and several former Soviet Republics. One of his passions is to analyze how disruptive industries and technologies allow consumers more choice at a lower cost. Among many op-eds and media appearances, he has been published in the Frankfurter Allgemeine Zeitung, Wirtschaftswoche, Die Welt, the BBC, SunTV, ABC Portland News, Montreal Gazette, Handelsblatt, Huffington Post Germany, CityAM. LAgefi, and The Guardian. Follow him on Twitter @FredCyrusRoeder

A version of this article was originally published on Consumer Choice Centers website as For the new year: Some fresh thoughts on how to tackle high drug prices and has been republished here with permission.

Excerpt from:
Viewpoint: Pricey gene therapies fuel debate over drug pricesand why there is no easy fix - Genetic Literacy Project

Unusually, the agreement means that both companies could be set for a windfall due to the structure of the co-development and co-commercialization partnership.

Adaptimmune Therapeutics could receive a total payment of $897m (806m) for its part in the deal, which includes an upfront payment of $50m, $7.5m per year for research and the rest locked away in milestone payments.

Astellas has the opportunity to make back some of this down payment, but only if Adaptimmune takes forward a drug candidate that the former company opts against developing at which point, Astellas could receive up to $552.5m in milestone payments.

The two companies are targeting the development of stem-cell derived allogeneic T-cell therapies to patients with cancer, potentially developing up to three targets. In particular, Adaptimmune will utilize its target identification and validate capabilities to generate target-specific T-cell receptors (TCRs), chimeric antigen receptors (CARs), and HLA-independent TCRS.

Astellas will contribute its donor cell and gene editing platform that it acquired through its buyout of Universal Cells. In addition, the Japanese drugmaker will provide the funds for research up until completion of a Phase I trial for each candidate.

After which point, both companies will have the option to progress with co-development and co-commercialization of the candidate, or to allow independent development of the candidate.

Astellas will also reserve the right to develop two targets independently.

Helen Tayton-Martin, chief business officer at Adaptimmune, spoke to us about the potential benefit of developing allogeneic treatments.

She explained, The first single advantage is that the products are already available frozen, and can be available almost immediately to be thawed and administered when they need them, rather than going through the cycle of apheresis, product production, shipping and re-infusion.

A second advantage is the greater consistency of the products we will manufacture, as [theyre] not dependent on individual patient cells, Tayton-Martin added.

When asked about the problems current commercial CAR-T treatments have faced in meeting specification, Tayton-Martin stated that she is confident that the companys integrated manufacturing capability will not face similar challenges to products already on the market.

Regardless of how far the potential product candidates progress, Tayton-Martin confirmed that the company now has the capital to fund it through to Q1 2021.

For Astellas, the deal marks another effort to develop its rapidly growing pipeline of potential advanced therapeutic candidates. The deal prior to this came only last week, in the form of a $665m deal for CAR-T specialist Xyphos.

Last year saw similar activity for Astellas, with a $3bn deal for gene therapy specialist Audentes announced as the year came to a close, adding on previous smaller deals to build its portfolio in the cell and gene space.

More:
Astellas and Adaptimmune partner on T-cell therapies - BioPharma-Reporter.com

The ball has dropped on a new year and a new decade, as we move from the 2010s into the 2020s. The last 10 years have seen incredible advances in science and technology, including a dramatic reduction in the cost of genetic sequencing, the first successful uses of gene therapy in humans, and the existence of gravitational waves. But what about thenextdecade? What previously impossible things will humans achieve? TheWyss Institute for Biologically Inspired Engineering at Harvard Universityasked its faculty members across a wide range of scientific disciplines what they predict will be the most impactful developments in their fields between now and the year 2030.

George Church Synthetic Biology

By 2030, we hopefully will see human clinical trials being run on transplanted organs from highly edited pigs and proteins from recoded genomes. Whole-genome sequencing may become a high-quality, equitably priced alternative to expensive gene therapies for rare diseases. Imaging will move in for close-ups (5 nm resolution) and every pixel will tell its story (DNA, RNA, protein, and lineage). Finally, we hope to see synthetic biology impact carbon sequestration via virus-resistant plants and algae, and cold-resistant elephants reverting arctic ecosystems to highly photosynthetic grasslands.

Jim Collins Synthetic Biology

Synthetic biology is well-positioned to help advance medicine over the next decade via the development of next-generation diagnostics and gene and cell therapies. The field also has tremendous potential to enhance basic research in molecular biology, by enabling the creation of novel tools to probe and analyze the complex functions of biomolecular components and systems in living cells.

Mike Levin Developmental Biology

The biggest knowledge gap, and frontier of opportunity, is taming the biological software that underlies embryogenesis and regeneration. Understanding the bioelectricity, biomechanics, and transcriptional circuits that allow cells to cooperate toward large-scale goals is the key to regenerative medicine, birth defects, cancer reprogramming, aging, synthetic bioengineering, and even new AI. Being able to exploit the decision-making, memory, and intelligence of cell swarms will result in transformative applications at the intersections of deep ideas in cognitive science, cybernetics, developmental biology, and computer science.

I anticipate the development of genetic and synthetic technologies to combat climate change and, concurrent with those developments, global discussions at all levels regarding their safe, equitable, and effective application.

Ting Wu, Synthetic Biology

Pam Silver Synthetic Biology

The engineering of biology will play a key role in the ability of the earth to support 10 billion people by implementing safe, faster, and more predictable biological systems. To feed the world and mitigate climate change, advances in synthetic biology will include increased utilization of sunlight together with mitigation of environmental contamination. The ability to respond quickly to epidemics and design better therapies will be a key advance for the field. And, as we move the needle on solving the problems on Earth, synthetic biology will also play a role in enabling and implementing the future of space exploration.

Eugene Goldfield Therapeutic Robotics

Therapeutic robotic systems in the next 10 years will no longer be considered robots. Their parts and control systems will be molecularly based, and will have capabilities akin to an immune system. The boundary between living and synthetic will continue to blur over future decades, requiring even greater care in the domain of ethics.

Don Ingber Bioinspired Therapeutics and Diagnostics

The most exciting developments in the field of bioinspired therapeutics and diagnostics will be a new paradigm for drug development that combines several unique innovations into a system that is faster, cheaper, and reduces harm to animals and humans in preclinical and clinical trials. I am especially excited about our increasing ability to analyze clinically relevant human physiological and pathophysiological responsesin vitro; high-throughput, phenotype-based screening of model organisms; novel molecular dynamics simulation capabilities; and the expanding application of deep learning technologies to solving specific clinical problems.

Samir Mitragotri Drug Delivery

The next decade in drug delivery will highlight the role of cells as drugs and carriers. Unlike drugs of the past, cells are unique in that they are living entities and have the ability to navigate through the body and reach destinations that most traditional drugs cannot. Strategies to deliver these living therapeutics will require novel approaches, and will create opportunities to use cells as carriers for targeting drugs to hard-to-reach tissues. Of particular interest are drugs that exploit or control the immune system for the treatment of cancer, autoimmune diseases, and allergies, among others. Strategies based on immune cells and immunological intervention will play a major role in drug delivery research and technology in the next 10 years.

David Walt Diagnostics

In the next 10 years, we will begin to realize the promised rewards of personalized medicine and personalized health, moving toward a system where we monitor individuals for key biomarkers and compare those results to their own measurements at an earlier time, rather than relying on population averages that dont reflect the wide biological variations that exist between people.

Dave Mooney Immunomaterials

I expect the next 10 years will lead to the demonstration, in human patients, that immunomaterials can dramatically alter the progression of various diseases. Immunomaterials will allow physicians to concentrate immune cells where they are needed in the body, regulate their activity, and disperse them when their job is done. The materials themselves will dissolve and degrade to leave nothing foreign in the body after treatment, but will create an immune memory that prevents the return of the disease.

William Shih Molecular Robotics

A major ongoing development in biomolecular science is encoding large numbers of single-molecule measurements into DNA records that can be read out later using high-throughput DNA sequencing. However, even future sequencing technologies will lack the bandwidth for sampling more than a small fraction of these records. Molecular robots built out of DNA, on the other hand, will be able to count and classify large sets of DNA records, and then summarize the results into brief DNA reports that can then be read out by DNA sequencing or other means. Thus, molecular robots will greatly increase the effective bandwidth of DNA-recording applications.

Dave Weitz Materials Science

I think that materials-by-design will become closer to a reality. We will learn how to formulate new structures on many different-length scales using a variety of fabrication methods complemented by computer-assisted design and assembly. And both the structure and functionality of the materials will be determined and controlled it will be the equivalent of precision medicine, but for materials design and synthesis.

Lou Awad Rehabilitative Medicine

To date, very few medical interventions have been designed to completely restore the pre-injury movement patterns of patients with neuromotor injury the current rehabilitation paradigm aims to rapidly attain independent walking, but patients often become independent by compensating for their injury rather than fully recovering the fast, economical, and stable gait of healthy human walking. The last decade has seen such remarkable advances in movement diagnostics,neurostimulationinterventions, and wearable robotics that the next decade is poised toachieve true restoration rather than mere compensation.

I predict that major advances in assembling cells and tissues will emerge that will allow us to print living organs for clinical use.

Chris Chen, 3D Organ Engineering

Elliot Chaikof Regenerative Medicine

Regenerative medicine employs repair, reconstruction, and replacement as strategies to treat patients with a diseased or damaged organ or tissue. Over the next decade, our capacity to repair may be dramatically enhanced through the discovery of agents that reverse the epigenetic clock, or eliminate or rejuvenate senescent cells. Reconstructive surgery will benefit from genetically reengineered off-the-shelf universal donor cells and engineered whole organs that can be used for any patient. Finally, I believe that the barrier for tissue replacement will be breached through the use of human-pig chimeras, initially to generate universally compatible human red blood cells for transfusions and, subsequently, whole organs for transplantation.

Kit Parker 3D Organ Engineering

As we start implanting organs into animals, and eventually patients, one of the most important things we will realize is the knowledge gaps we have in basic anatomy and physiology. All that we know about the functional anatomy of organs might not be enough to sufficiently mimic what we think is Natures anatomy and, unfortunately, there are hardly any classically trained anatomists or physiologists in the world anymore. To successfully engineer implantable organs, we need to develop the scientific talent to do the old-school physiology experiments that havent been done in 50 years or more to understand what we are building or need to build.

The rest is here:
Harvard Wyss researchers on the next decade in... - ScienceBlog.com

Editors Note: Endpoints News is reporting live from #JPM20 after kicking things off with an action-packed event, which you can replay here. What follows is a stream of tidbits we have collected while wandering around Union Square in San Francisco. Check back in throughout the week for updates by John Carroll and Jason Mast.

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SAN FRANCISCO A year ago Takeda CEO Christophe Weber and R&D chief Andy Plump arrived at JP Morgan right on the heels of closing their big Shire buyout. Now theyre back after shaking up the portfolio, boosting R&D spending by about 50% to $4.5 billion and adjusting the pipeline a task which isnt quite finished yet.

In an interview on Tuesday, Plump told me that Takeda is preparing the latest in a long string of spinouts after setting aside a package of psych drugs that would be better suited to the hands of some specialists. Like a lot of the major R&D outfits, its not one of their core fields of expertise.

These Takeda drugs including therapies for depression and schizophrenia are very interesting but still difficult, involving 3 clinical-stage programs and a handful of preclinical efforts, which Plump is quick to concede offer plenty of challenges to developers.

Spinouts are something that Takeda is good at. Theyve been doing these deals for several years now in the US, Europe and Asia creating up to 25 companies where they typically hand off drugs to entrepreneurs and retain a chunk of equity of around 25% to 30% of the biotech involved.

Their decisions on what to keep and what to deal out, adds Plump and Weber, have nothing to do with projected revenue.

The key aspect is innovation, says Weber. Are they innovative therapies? And its not because you shouldnt do it on projected revenue so much as you cant do it that way.

You cant do it on NPV, adds Plump, because then NPV becomes the driver of the decision and the NPV is always wrong.

The two Takeda execs spent considerable time and effort in revamping Takeda into a global, top 15 player with scale.

We were lacking scale to sustain our R&D investment, says the CEO. The Shire buyout gave us scale that allows us to compete with anyone. John Carroll

#JPM20: Catalent CEO John Chiminski isnt going anywhere important without his lucky boots

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In my one-on-one with Catalent CEO John Chiminski I was drawn to the cowboy boots he was wearing not exactly the kind of standard Brooks Brothers attire you often see at JP Morgan and asked him how many miles he had put on the boots as he traveled the world managing a global CDMO.

Heres part of the response, and its priceless:

In advance of the IPOour owners, Blackstone, went to the investment bankers and they said, Hey, before Chiminski goes out on the road show, hes got to shave his beard and he cant wear the boots. So I told the investors and I would just say, I used a couple of hand gestures and I said, look, you might as well shave my head, because Ill have about that much confidence. And I will tell you right now, if I didnt show up with my boots, which is a little harder nowadays cause Ive got arthritic hips, if I didnt show up with my boots, Id had some investors that are selling. So these boots have about a million miles and 165 million shares of Catalent were sold with these babies

Boots arent going anywhere guys. John Carroll

#JPM20: Considered New Pfizer yet? The CEO says hes whipping it into shape and getting more focused in R&D

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SAN FRANCISCO Albert Bourla isnt a fan of the Old Pfizer. Or of the analysts who are ignoring the New Pfizer.

Bourla, giving his second JP Morgan presentation as Pfizer CEO after over 25 years at the US drug-making giant, continued the rebrand he had been tapped to lead: Positioning Pfizer as a company that had been bloated for years but was now set for a streamlined future.

Phase II success rates the industry is at 30%. Pfizer for many years was at 15%, Bourla told JP Morgan analyst Christopher Scott on stage. We were taking an approach of very little rigor.

Bourla said Pfizer would further pare down on non-R&D expenses, continuing a trend they began by spinning off their Upjohn division and combining their consumer health business into a joint venture with GlaxoSmithKline. The GSK venture will move to an IPO within 3 or 4 years, he said, echoing previous comments from the British pharma.

Within research, Bourla said, they would focus on 6 areas, rather than the 12 they had long developed. Asked which parts of the pipeline were exciting but overlooked, Bourla dove for a minute through the R&D assets he thought Wall Street had ignored, including their vaccines, gene therapies, and inflammation drugs.

We do have five different JAKS that we are starting in more than 10 different indications, and only one of them I have seen minor projections for, he said. I can go on and on.

Bourla said they would focus on licensing small add-ons to the pipeline in those 6 areas and wouldnt move toward a larger merger or acquisition. It was a discouraging comment for a JP Morgan crowd watching for the next major deal, but also one Pfizer has made in the past months before acquiring Array in an $11.4 billion deal.

We never say never, Bourla said. Jason Mast

#JPM20: Consummate dealmaker Vivek Ramaswamy bags a record regional upfront in latest deal this time focused on Japan

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SAN FRANCISCO At the start of my panel on dealmaking on Monday I joked that consummate negotiator Vivek Ramaswamy was probably making a deal as we spoke, texting terms on the mobile.

Today he tells me I wasnt far off the mark. A deal was cooking right off stage.

On Day Three of JP Morgan Ramaswamy has completed what his team is calling the largest ever regional licensing deal for a derm product prior to Phase III data and one of the largest upfront payments ever for a Japanese licensing deal prior to Phase III data across any indication.

And this one was signed late Tuesday night after they put the finishing touches to the contract.

I almost laughed when you said that, Ramaswamy tells me about the panel comment. If I had not put my phone on flight mode that would have been true.

His dermatology play Dermavant has licensed out exclusive rights to develop, register and market tapinarof in Japan to Japan Tobacco, which is passing on the license to its subsidiary, Torii Pharmaceutical. The Japanese company is paying $60 million and up to $53 million in development milestones for tapinarof, in development for psoriasis and atopic dermatitis.

Dermavant CEO Todd Zavodnick is clearly stoked about the numbers for his drug, and the shot of working on this with JTs company.

Its not just a licensing deal, he says, its the right partner.

Ramaswamy bought the drug from GSK for up to $324 million. The pharma giant had been revamping its pipeline as R&D chief Hal Barron took aim at a comeback. The nonsteroidal anti-inflammatory topical cream which activates the aryl hydrocarbon receptor hasnt been a high-profile agent. But researchers have highlighted promising mid-stage data to underscore its potential. Now its in Phase III for psoriasis, with plans to tee up atopic dermatitis.

Not surprisingly, any mention of AD immediately spark comparisons to the marketing juggernaut Regeneron and Sanofi have created for Dupixent. But Dermavants drug is a topical, and Zavodnick says that in AD, patients typically get an injectable and a topical. So he believes that patients will start with the topical and then move to the combination, leaving plenty of room for his drug in the market.

Ramaswamy also notes that its rare to see two significant dermatology deals at one JP Morgan. Eli Lilly got the party started with their $1.1 billion Dermira buyout, which gave them lebrikizumab. Dermavants deal makes for a nice book end to that package of news.

Says Ramaswamy: Dermatology is back. John Carroll

#JPM20 Two biotech legends talk about gene therapys past and future

SAN FRANCISCO I had a chance on Tuesday to moderate a discussion on gene therapy R&D at the huge WuXi conference at the Hilton with Fraziers Tachi Yamada ex-CSO at GSK and Takeda and James Wilson from Penn, two big figures in the field who started a new biotech a little under a year ago to focus on monogenic diseases of the CNS. I did a bow to both of them for everything theyve pioneered in the last 2 decades, which spurred Yamada to give Wilson credit for the science work. As for Wilson, he got the biggest response from the audience with a remark that Tachi is always just called by his first name, making him the Beyonce of biotech. John Carroll

#JPM20: Biogen CEO Michel Vounatsos offers a smile of confidence as he presents aducanamab

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SAN FRANCISCO Biogen isnt softening its rhetoric.

Two months after research chief Al Sandrock all-but dared the FDA not to approve its Alzheimers drug, CEO Michel Vounatsos told an overflowing ballroom at JP Morgan that the success of a drug that operates like aducanumab is a dream.

Some of you were at CTAD and saw the data, Vountsos said, referring to the controversial subgroup analysis they presented their aducanumab trial at the Clinical Trials for Alzheimers Disease conference. Who in the room doesnt know someone who has been demented?

That analysis, coming months after the company first announced the drug had failed, relied on believing how late changes called protocol amendments to one of two identical trials made one succeed and the other fail.

Without all the protocol amendments, I would not be here, today, standing with a smile of confidence on the way forward, Vounatsos said, not quite smiling.

Still, Biogen has heard the criticism from investors, most notably Bairds Brian Skorney, that their big ambitions have left them with few assets in their pipeline likely to raise revenue soon. Vounatsos walked through the other parts of their pipeline, including a lupus drug, an ALS drug, a new MS drug and, of course, another new Alzheimers drug with a different approach: Tau. Jason Mast

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#JPM20: Gilead launches new T cell engineering player with help from familiar players at Vida and Westlake

SAN FRANCISCO After making its mark in cancer cell therapies with the Kite buyout, Gilead has allied itself with a pair of venture investors to hunt for new therapies that can address the flip side of that therapeutic coin. And theyre chipping into the launch round while adding more cash and milestones for a research collaboration to get things underway.

The new biotech theyre showing off around JP Morgan this week is Kyverna Therapeutics, which is being gifted with some cutting-edge T cell engineering tech as well as a $25 million Series A to fund work on the discovery of new drugs for autoimmune disease braking the immune system instead of organizing an attack. Gilead signed off on a collaboration that starts with a $17.5 million upfront and a full slate of research and commercial milestones that can go up to $570 million.

The startup brings together some high-profile figures and technology.

Dominic Borie, a Genentech vet and the former head of external research for Horizon Therapeutics, is heading the new venture as CEO. While at Stanford, Borie was credited with playing a part in validating JAK inhibition for rheumatoid arthritis a breakthrough tech that a bevy of drug developers would like to leapfrog with new and better drugs. Jeffrey Greve, formerly at Delinia ahead of their Celgene buyout, is CSO.

Theyre being backed by Fred Cohen at Vida Ventures and Beth Seidenberg, who founded Westlake Village BioPartners in LA after jumping from Kleiner Perkins, where she specialized in biotech for years. Cohen kickstarted Vida along with a group of experienced venture players, with a big role for Arie Belldegrun, who sold Kite to Gilead and retains some A list contacts at the big biotech.

The upstart will be using synNotch technology from Kite, which picked it up in Gileads acquisition of Wendell Lims Cell Design Labs a couple of years ago, which Belldegrun had backed as well, sitting on the board with Seidenberg.

These are the relationships that breed trust and companies. John Carroll

#JPM20: BioMarin chief Bienaime is building a gene therapy pipeline, and the manufacturing needed to control markets

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SAN FRANCISCO JJ Bienaimand his team arent just running as fast as possible to stay in the lead in the race to the first marketing OK for a gene therapy for hemophilia A. Theyre building a pipeline, and the manufacturing capacity needed to dominate their markets.

In a chat just ahead of the firing gun at JP Morgan, the BioMarin CEO told me that the biotech had obtained official sanctions in the US and UK to launch a Phase I/II study of BMN 307 this quarter for phenylketonuria (PKU). Now they will set out to see if a single dose of their gene therapy can restore natural Phe metabolism, normalize plasma Phe levels and enable a normal diet in patients with PKU.

Next up in the gene therapy portfolio, he tells me, is HAE.

Whats more, he adds, BioMarin has completed work at a facility in Novato that doubles their gene therapy capacity jumping from 5,000 to 10,000 patients a year. That could handle the hemophilia A market and the rest of the markets to come for some time. It also gets them around the bottleneck of gene therapy production that has been holding back some of the new players in the field.

Its not our intent to turn into a pure gene therapy company, but it will play an important role in BioMarin, says Bienaim.

For now, the story around gene therapy at BioMarin is centered on a simple theme: BioMarin has the first shot at an upcoming approval in hemophilia A, but a waning level of response leaves a few analysts wondering whether Sangamo, in particular, can catch up with a better therapy. The reasoning is built around the assumption that patients would prefer to wait for a better gene therapy, as they can only be dosed with an AAV product once.

Bienaim, though, doesnt believe thats true. He points to new work on next-gen gene therapies skirting AAV that can be repeatedly dosed, and that opens the door to a re-up down the road if their product stops working. Patients will take a prospective cure, he says, with a 4-year update coming up that he believes will show a continued protection against bleeds.

One other point: The CEO believes that given the high cost of treating hemophilia patients now, payers will accept a drug that costs $2 million to $3 million. (Not that that is the price they expect to charge. That decision comes later.)

But will they want some assurances that it will work long enough to make it less expensive in the long run? John Carroll

#JPM20: Vertex lays out post-CF ambitions as Gilead demurs on acquisition

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SAN FRANCISCO Vertex laid out a vision for a post-CF and a post-Jeffrey Leiden future at their investor presentation Monday morning. It looks a lot like the past.

That future, the outgoing CEO Leiden said, will center on other fields like cystic fibrosis: Specific niches where few life-altering drugs exist, including sickle cell and a rare lung disease. Those are also fields where Vertex could stand to charge the high prices theyre becoming known for.

You will never see a me-too drug from us, Leiden said, a day after Alexis Borisy sparked widespread buzz with a new biotech aimed at lowering drug prices through me-too drugs. We think transformative medicines are of the highest value for patients.

This year was all but destined to mark a new chapter for Vertex. Nearly 20 years after starting their CF program, the company introduced their capstone last year in Trikafta, a drug that covers 90% of patients. Leiden celebrated the news by leaving the CEO spot to become executive chairman.

Leiden may or may not have been referring to Borisys startups with his comments, but the reference is fitting. Vertexs drugs have made cystic fibrosis a livable disease, but it comes at a price tag over $300,000 for Trikafta payers like the NHS have balked at.

With the Vertex changes and Gilead sitting on both a wealth of cash and a shaky pipeline, rumors swirled the big California biotech could pursue a massive buyout. But early Monday morning, Gilead CEO Daniel ODay threw cold water on that, telling investors that the company was looking to expand but any changes would be bolt-on acquisitions.

In what was billed as a fireside chat, incoming Vertex CEO Reshma Kewalramani detailed the pipeline she would lead. Those assets include marketing an expanded indication for Trikafta (its not yet approved for children under 12), a drug for alpha-1 antitrypsin deficiency a lung disorder similar to cystic fibrosis their gene therapy program for sickle cell and beta thalassamia, and the potential cell therapy cure for diabetes they acquired with the $950 million Semma buyout.

The water-cooler has also buzzed with talk that Vertex could acquire CRISPR Therapeutics, their partner on the gene therapy program.

Kewalramani didnt address that. Instead, she offered a target for Vertexs most recent acquisition: bringing the cell therapy into the clinic.

Weve set an ambitious goal for ourselves with Semma, Kewalramani said. Jason Mast

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#JPM20: 'The NPV is always wrong.' Takeda preps another spinout this time on psych - Endpoints News

BURLINGTON LabCorphas introduced a suite of cell and gene therapy development services fromCovance, its drug development business, to help biopharmaceutical companies develop cell and gene therapies for cancer and other diseases.

With approximately one-third of biopharmas pipeline focused on precision medicines, it becomes even more critical to find innovative ways to help our customers speed these transformational therapies to the patients who need them, said Paul Kirchgraber, M.D., chief executive officer of Covance.

The coordinated services cover the full range of therapy development: pre-clinical, clinical and post-approval.

Pre-clinical services include pharmacology and safety assessment, biodistribution testing and services, and vector and cell characterization and qualification.

Clinical services include clinical trials with focused expertise in oncology, rare diseases and specific therapeutic areas; bioanalysis testing and services; biomarkers, including companion diagnostics; central laboratory services; and regulatory and strategic product development consultation, including clinical development and commercialization strategy.

Post-approval services include long-term follow-up studies, real-world evidence, pharmacovigilance, post-marketing commitments support, regulatory consulting and commercialization.

Cell and gene therapies, categorized as advanced therapy medicinal products (ATMPs), are more complex and specialized than traditional drugs. They typically involve correcting or replacing genetic information to restore the correct function of cells or genes for many conditions, including cancer and rare genetic diseases.

ATMPs have the potential to offer a durable, life-changing therapeutic response, possibly with a single administration, for patients who may have few or no alternative treatment options, according to the Alliance for Regenerative Medicine in Washington.

About 950 companies worldwide are developing ATMPs, and 1,052 clinical trials were under way by the end of the third quarter of 2018, according to the Alliance. Most of those trials 650 were for oncology indications.

Covance has more than 20 years of experience in supporting advanced therapies. The company said its coordinated approach to services uniquely positions it to partner with sponsor organizations to address complexity, reduce cost and risk, and accelerate timelines of their novel therapies.

In the last four years, Covance has conducted more than 300 preclinical studies and more than 40 clinical trials for cell and gene therapies globally, said Steve Anderson, Ph.D., chief scientific officer at Covance.

Covance is uniquely positioned to provide scientific and technical expertise to support the rapid growth in development of cell and gene therapies and the introduction of new regulatory pathways for these products, Anderson said. Our knowledge and experience from discovery through post-approval are significant, as demonstrated by our support of recent approvals in both cell and gene-based therapies.

Kirchgraber said Covance has made ongoing investments in people, processes and technologies targeting cell and gene therapies. The company has also grown its capabilities and capacity by forming strategic partnerships and making key acquisitions, such as the purchase in early 2019 of MI Bioresearch, a contract research organization specializing in nonclinical oncology testing, with a focus on immunotherapies and adoptive T-cell therapeutic approaches, he said.

LabCorp was established in 1969 as Biomedical Laboratories, a small pathology lab located in a hospital basement in Burlington, N.C. Over the last 50 years it has become a dominant life sciences company through mergers, acquisitions and organic growth.

LabCorp provides diagnostic, drug development and technology-enabled solutions for more than 120 million patient encounters per year. The company typically processes tests on more than 2.5 million patient specimens per week and supports clinical trial activity in about 100 countries through its central laboratory business, generating more drug safety and efficacy data than any other company.

LabCorp employs about 3,600 people in Alamance County, where it is the largest private employer, and about 7,300 across North Carolina. Its global workforce includes nearly 61,000 employees.

The company reported revenue of more than $11 billion in 2018. It announced today that it willannounce its 2019 financial resultson February 13. Its shares are traded on the New York Stock Exchange.

(C) N.C. Biotech Center

Continued here:
LabCorp, Covance launch new effort focusing on gene therapies - WRAL Tech Wire

Days after shaking hands with German regulators over the launch and coverage of its beta-thalassemia gene therapy, bluebird bio has wooed a Celgene exec to lead its European operations.

Nicola Heffron, a biopharma vet with stints across Eli Lilly, GSK and Shire, jumps from a brief tenure overseeing marketing for Celgenes myeloid portfolio in Summit, NJ. She will now be based in Zug, Switzerland.

Shes replacing Andrew Obenshain as he joins CEO Nick Leschly and the leadership team in Boston, according to Bloomberg, which first reported the news. Obenshains new title is chief of wings.

On Monday bluebird announced that Germany will be the first country to commercially offer Zynteglo, their procedure encoding A-T87Q-globin gene in CD34+ cells extracted from patients. Under their value-based payment scheme, the $1.8 million price is divided into five installments. After an initial payment is made at the time of infusion, the payers wait and see and only pay if the patients continue to be transfusion-free.

Multiple statutory health insurances have signed onto the plan, bluebird said, and University Hospital of Heidelberg will host the first qualified treatment center.

The biotech has been busy sorting out manufacturing specs and talking to individual countries since the EU issued an historic OK last June. Its sanctioned for a specific group of beta-thalassemia patients those who are 12 years and older, transfusion dependent, do not have a 0/0 genotype and for whom hematopoietic stem cell transplantation is appropriate but a donor is not available.

For patients with TDT, lifelong chronic blood transfusions are required in order to survive, bluebird chief commercial officer Alison Finger emphasized in a statement. Their one-time infusion promises to do away with the transfusions for good.

A rolling BLA submission to the FDA has begun, bluebird added.

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Celgene exec jumps to head bluebird bio ops in Europe, where its $1.8M gene therapy Zynteglo is now available - Endpoints News

There are currently two approved therapies for spinal muscular atrophy (SMA), Biogens Spinraza and Novartis gene therapy Zolgensma. Spinraza is priced at $750,000 for the first year and $375,000 each year afterwards. Novartis Zolgensma, which is believed to be a one-shot cure, is priced at $2.1 million. That makes it the biopharmaceutical industrys most expensive one-time treatment.

Roche is expecting the U.S. Food and Drug Administration (FDA) to approve its own SMA therapy, risdiplam, by May 24. And the company has suggested that it plans to undercut both Biogen and Novartis on price in order to make up for being third-to-market.

SMA is a rare, autosomal recessive neuromuscular disease characterized by the degeneration of alpha motor neurons in the spinal cord. This results in progressive muscle weakness and paralysis. According to the Orphanet Journal of Rare Diseases, the estimated incidence is 1 in 6,000 to 1 in 10,000 live births.

The FDA approved Zolgensma in May 2019 to treat children less than two years old with SMA that have bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma is only approved for SMA Type 1. SMA type I, the severest form, is almost always fatal by two years of age, with a 50% mortality rate by seven months and a 90% mortality rate by 12 months.

A 2009 study found that with nutritional and respiratory care, a greater percentage of those patients were living beyond two years of age. Patients with Types II and III typically live into adulthood and could potentially have a normal life expectancy, although with a great deal of healthcare services. Spinraza is approved for all types.

William Anderson, chief executive officer of Roche Pharmaceuticals, has not reported an actual projected price for risdiplam, but said it will take the same strategy it did in pricing its hemophilia A drug Hemlibra in 2017 when it took on Novo Nordisk and Takeda.

With Hemlibra, we priced at about half of bypassing agent, Anderson said in an interview at the JP Morgan Healthcare Conference in San Francisco. We aim to underwhelm with our price.

It is likely that Zolgensma, because of it being a cure rather than a treatment, will be the therapy of choice for SMA. Still, analysts project risdiplam could hit $1 billion in annual sales, and Biogens Spinraza is already raking in about $2 billion per year. Spinraza and risdiplam will likely be seen as the treatments for older patients. And risdiplam has an advantage over Spinrazait is an oral medication, whereas Spinraza requires a spinal infusion every four months.

Zolgensma is also being evaluated in older patients, but the trial has been halted because of safety concerns, although Novartis expects the FDA to approve restarting the trial.

Novartis is also working on an oral drug for SMA called branaplam. In December, its president of the Novartis Institutes for BioMedical Research, Jay Bradner, suggested the company wasnt as enthusiastic about the drug, with Bradner saying Novartis did not see a big opportunity for oral therapy there, or we would develop this molecule further.

Since then, Novartis backpedaled a bit, saying the trial of branaplam would continue and a decision on moving forward would be based on the data readout later this year.

In November 2019, Roches risdiplam met its primary endpoint in the pivotal Part 2 of SUNFISH, which evaluated risdiplam in patients aged 2-25 years with Type 2 or 3 SMA. The primary endpoint was change from baseline in the Motor Function Measure 32 (MFM-32) scale after one year compared to placebo.

Risdiplam is an investigational survival motor neuron-2 (SMN2) splicing modifier. It is designed to increase and sustain SMN protein levels in the central nervous system and peripheral tissues.

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Roche Plans to Undercut SMA Therapy Price to Compete with Biogen and Novartis - BioSpace

A recent report added by Market Study Report, LLC, on Hemophilia Gene Therapy Market provides a detailed analysis on the industry size, revenue forecasts and geographical landscape pertaining to this business space. Additionally, the report highlights primary obstacles and latest growth trends accepted by key players that form a part of the competitive spectrum of this business.

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Global Hemophilia Gene Therapy Market Growth, Size, Analysis, Outlook by 2020 Trends, Opportunities and Forecast to 2025 - Technology Magazine

Infographic by Dan Helmici

By Riley Holmes 1/14/20 11:43pm

A Chinese court sentenced He Jiankui (Ph.D. 10), who revealed that he had genetically-edited twin girls last year, to three years in prison on Dec. 30, 2019. The questions surrounding his PhD advisor, Rice University bioengineering professor Michael Deems involvement in the Hes experiments, remain unanswered. In November 2018, Rice began a full investigation into Deems role in the research.

According to the New York Times, He plead guilty to forging documentation from ethics committees approving the study, which he used to recruit participants. Additionally, Chinese media outlets revealed his work on a previously undisclosed third child.

Since Rices Nov. 2018 statement, no more public updates on the internal investigation of Deem have been given. The Office of Public Affairs declined to comment for this article.

In the original statement, Rice stated they had been unaware of the project.

This work as described in press reports violates scientific conduct guidelines and is inconsistent with ethical norms of the scientific community and Rice University, the statement read.

Meanwhile, Stanford Universitys investigation cleared three researchers associated with He in Spring 2019 after concluding they did not encourage or directly participate in the project, according to the New York Times coverage.

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Dr. Christopher Scott, Chair of Medical Ethics and Health Policy at Baylor College of Medicine, studies ethical, legal, social and policy implications of biotechnologies similar to the ones He used. Scott formerly taught a required research ethics course to NIH grant recipients, and also mentioned that he discussed Hes project with an ethics class at Rice last fall.

The thing that is troubling about the China case is that its not a China ethics problem, it is an international problem, Scott said. So, the question from an ethics point of view is what are the professional and ethical obligations of those folks, who have either direct knowledge of intent or knowledge that the experiment was conducted, to report this fellow?

A Chinese scientist associated with the project claimed Deem was more than just a bystander, according to an article posted on STAT news in Jan. 2019. The studys manuscript lists Deem as an author. After He announced his work at a conference in Hong Kong in Nov. 2018, Deem told the Associated Press he had met the twins parents. According to the Houston Chronicle, however, he attempted to remove his name from the paper after it was sent to journals such as Nature. The study was ultimately rejected and never published. Deem had served as Hes advisor when he completed his PhD at Rice between 2007 and 2010.

Deem did not respond to requests for comment at the time of publication. Two other bioengineering faculty members also declined to comment.

According to Scott, another issue arises with the language of the consent paperwork which described the gene-therapy experiment as a vaccination against H.I.V. The Thresher was not able to obtain a copy of the consent paperwork referenced by Scott.

When it comes to genetic editing, Scott said the unknowns and risks of these experiments differ from administering a new drug to a patient or implanting a heart device.

These are genetic effects that are felt, carried for life, and also carried in heritable ways to generations, Scott said, There has to be a commitment to understanding these sorts of things, how you follow these genetically-edited kids through adulthood, and later their children, and their childrens children - a lot of unknowns.

Two other Chinese scientists connected to Hes work were also given prison sentences, according to coverage by the New York Times. If the project had resulted in a participants death, Hes sentence might have exceeded 10 years, according to the Times.

Even though this was an uncomfortable and unfortunate event, it's really one of those teaching moments, Scott said You have to ask the question institutionally, what can be done to up the level of ethical foresight in teaching universities and research universities? That's a hard question to answer.

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Rice Ph.D graduate known as 'CRISPR Baby' scientist sentenced to three years in prison - The Rice Thresher

Global Gene Therapy Market research report provides an effective industry outlook, future trends and dynamics for market growth rate, market size and key players of the industrys information with forecast. Furthermore, report includes a brief on marketing research methodology as well as opportunities offered by the market.

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Table of Content:1 Introduction of Gene Therapy Market1.1 Study Coverage1.2 Product Introduction1.3 Study Objectives1.4 Years Considered2 Executive Summary3 Breakdown Data by Manufacturers3.1 Sales by Manufacturers3.2 Revenue by Manufacturers3.3 Manufacturing Base Distribution, Product Types3.4 Manufacturers Mergers & Acquisitions, Expansion Plans4 Breakdown Data by Type4.1 Overview4.2 Breakdown Data by Type5 Breakdown Data by Application5.1 Overview5.2 Breakdown Data by Application6 Gene Therapy Market, By Geography6.1 North America6.2 Europe6.3 Asia Pacific6.4 Central & South America6.5 Middle East and Africa6.6 Rest of the World7 Company Profiles7.1 Company Details7.2 Company Business Overview7.3 Sales, Revenue and Gross Margin7.4 Products Offered7.5 Recent Development8 Future Forecast8.1 Overview8.2 Market Forecast by Regions8.3 Sales Forecast by Regions8.4 Revenue Forecast by Regions8.5 Forecast by Type8.6 Forecast by Application9 Market Opportunities, Challenges, Risks and Influences Factors Analysis9.1 Market Opportunities and Drivers9.2 Market Challenges9.3 Market Risks/Restraints10 Appendix10.1 Research Methodology

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Report: Cell and Gene Therapy Consumables Market Industry Key Players, Size, Trends, Opportunities, Growth- Analysis to 2027 - Market Research News...

Horizon Discovery Group plc (LSE: HZD) ("Horizon", "the Company" or "the Group"), a global leader in the application of gene editing and gene modulation technologies, today announced that it will provide access to a novel base editing technology licensed from Rutgers, The State University of New Jersey, for exclusive use in therapeutic, diagnostic and services applications. This technology is incorporated into Horizons next-generation gene editing platform to enable the development of novel therapeutics that rely on engineering patients cells either directly in the body (gene therapy), or externally before transplanting back into the patient (cell therapy). This platform will also expand the Companys research tools and service provisions.

The Company formed an exclusive partnership with Rutgers in January 2019 to further develop the novel base editing technology invented by Dr. Shengkan Jin, associate professor of pharmacology, and co-inventor Dr. Juan C. Collantes, post-doctoral research fellow at Rutgers Robert Wood Johnson Medical School, and has since been funding research in base editing at the University while undertaking its own evaluation and proof-of-concept studies. Horizon has a number of internal programs designed to accelerate the clinical uptake of this technology and is now seeking 35 partners to assess and shape the development of its Pin-point base editing platform.

Horizon will offer partners access to a novel system that could be used to progress more effective multi-gene knockout cell therapy programs through clinical development with an improved safety profile. Partners will also gain access to the Companys expertise in genome engineering of different cell types, access to early technical data, and influence over the direction of future development.

Base editing is a novel technology for engineering DNA in cells, which the potential to correct certain errors or mutations in the DNA, or inactivate disease-causing genes. Compared with currently available gene editing methodologies such as conventional CRISPR/Cas9, which creates "cuts" in the gene that can lead to adverse or negative effects, this new technology allows for accurate gene editing while reducing unintended genomic changes that could lead to deleterious effects in patients.

Dr. Jonathan Frampton, Corporate Development Partner, Horizon Discovery, said: "The technology could have a significant impact in enabling cell therapies to be progressed through clinical trials and towards commercialization. Horizon is pleased to offer an effective and precise base editing technology and, alongside Rutgers, aims to make base editing available to all appropriate cell and gene therapy companies as well as research departments. Partnering with leading organizations will help us to drive innovation and deliver the best therapy for the patient."

Dr. Shengkan 'Victor' Jin of Rutgers University stated: "The cytidine deaminase version of the technology alone could potentially be used for developing cell therapies such as gene modified cells for sickle cell anemia and beta thalassemia, HIV resistant cells for AIDS, over-the-shelf CAR-T cells for cancer, and MHC-compatible allogenic stem cells for transplantation. Other applications could include use as gene therapies for inherited genetic diseases including antitrypsin deficiency and Duchenne muscular dystrophy. In addition, we intend to take full advantage of the unique modularity and versatility features of Pin-point platform and develop efficient gene inactivation agents for potential treatment of many devastating diseases where the leading causal contributing factors are well defined. At the top of this disease list are Alzheimers disease, amyotrophic lateral sclerosis, and familial hypercholesterinemia."

Dr. S. David Kimball, Senior Vice President for Research and Economic Development at Rutgers University, added: "The gene editing technology developed by Rutgers has the potential to revolutionize how scientists think about their search for better options and outcomes in the treatment of disease. It has the potential to solve some of the most persistent global health challenges. This partnership with Horizon Discovery is paving the way to deliver biotherapies for precision medicine and diagnostics and improve human health. I am proud that Rutgers, together with Horizon, is among the frontrunners in the field of gene editing."

View source version on businesswire.com: https://www.businesswire.com/news/home/20200114005331/en/

Contacts

Zyme Communications (Trade and Regional Media) Lorna CuddonTel: +44 (0)7811 996 942Email: lorna.cuddon@zymecommunications.com

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Horizon Discovery to Provide Access to Novel Base Editing Technology - Yahoo Finance

SAN FRANCISCO andPFFFIKON, Switzerlandand LONDON, Jan. 13, 2020 /PRNewswire/ --DiNAQOR AG, a global gene therapy platform company, today announced a research collaboration and exclusive license agreement with UCL to develop novel gene therapies for the treatment of monogenic cardiomyopathies, diseases of the heart muscle that can lead to heart failure in children and adults.

The collaboration will focus on the development of therapies targeting several genes using DiNAQOR's cardiac modular technology platform. Under the terms of the agreement, DiNAQOR and UCL will collaborate to advance DiNAQOR's two discovery cardiac gene therapy programs into clinical development. DiNAQOR will obtain exclusive worldwide rights to any therapies developed as part of the collaboration. If a product is successfully commercialized from the collaboration, UCL will be entitled to royalties on sales.

The license agreement was carried out by UCL's technology commercialisation company, UCL Business Ltd (UCLB), part of UCL Innovation & Enterprise. Based on the research of Professor Thomas Voit at the UCL GOSH Institute of Child Health,the agreement aims to utilise UCL's leading expertise in gene therapy.

"Early stage partnerships are critical to expedite innovation in gene therapy research and development. UCL is one of the world's leading research centers and is ideally suited to help us expand our pipeline of gene therapies for cardiomyopathies," said Dr. Valeria Ricotti, Chief Medical Officer at DiNAQOR.

"This partnership represents an exceptional opportunity to accelerate development of potentially life-saving gene therapies for patients suffering from heart failure. We are excited to collaborate with the DiNAQOR team to help find a cure for monogenic cardiomyopathies," said Dr. Perry Elliott, Professor of Cardiovascular Medicine at The UCL Great Ormond Street Institute of Child Health (GOS ICH).

About Monogenic Cardiomyopathies

Cardiomyopathy is a disease of the heart muscle that can lead to heart failure. Approximately 50% of all cardiomyopathies are caused by a single-gene or monogenic defect. There are 1.7 million people in the European Union and the United States currently affected by a monogenic cardiomyopathy, 300,000 of these individuals have a defect in the MYBPC3 gene. There is currently no cure for patients living with genetic cardiomyopathies.

About DiNAQOR

Founded in 2019, DiNAQOR AG is a global gene therapy platform company focused on advancing novel solutions for patients suffering from heart disease. The company's lead preclinical program, DiNA-001is focused on the treatment of MYBPC3-linkedcardiomyopathy. DiNAQOR is headquartered in Pfffikon, Switzerland, with additional presence in London, England and Boston, Massachusetts (US). For more information visit http://www.dinaqor.com.

About UCL London's Global University

UCL is a diverse community with the freedom to challenge and think differently.

Our community of more than 41,500 students from 150 countries and over 12,500 staff pursues academic excellence, breaks boundaries and makes a positive impact on real world problems.

We are consistently ranked among the top 10 universities in the world and are one of only a handful of institutions rated as having the strongest academic reputation and the broadest research impact.

We have a progressive and integrated approach to our teaching and research championing innovation, creativity and cross-disciplinary working. We teach our students how to think, not what to think, and see them as partners, collaborators and contributors.

For almost 200 years, we are proud to have opened higher education to students from a wide range of backgrounds and to change the way we create and share knowledge.

We were the first in England to welcome women to university education and that courageous attitude and disruptive spirit is still alive today. We are UCL.

http://www.ucl.ac.uk| Follow @uclnews on Twitter | Watch our YouTube channel | Listen to UCL podcasts on SoundCloud | Find out what's on at UCL Minds | #MadeAtUCL

About UCL Business Ltd (UCLB)

UCL Business Ltd (UCLB), part of UCL Innovation and Enterprise, is a leading technology commercialisation company that supports research and innovations arising from UCL, one of the UK's top research-led universities. UCLB has a successful track record and a strong reputation for identifying and protecting promising new technologies and innovations from UCL academics. UCLB has a strong track record in commercialising medical technologies and provides technology transfer services to UCL's associated hospitals; University College London Hospitals, Moorfields Eye Hospital, Great Ormond Street Hospital for Children and the Royal Free London Hospital. It invests directly in development projects to maximise the potential of the research and manages the commercialisation process of technologies from laboratory to market.

Contacts

DiNAQOR:KWM CommunicationsKellie WalshT: 1-914-315-6072E: kwalsh@kwmcommunications.com

UCL:UCL Media Relations Dr. Rebecca CaygillT: +44 (0)20 3108 3846 / +44 (0)7733 307 596 E: r.caygill@ucl.ac.uk

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DiNAQOR AG Announces Research Collaboration and License Agreement with UCL to Investigate Gene Therapies for Heart Failure - P&T Community

XconomyBoston

JP Morgans annual healthcare conference convenes in San Francisco this week and Generation Bio CEO Geoff McDonough is betting gene therapy will be among the discussion topics. While such therapies now offer ways to address some rare diseases that previously had no treatment, they also have limitations.

Generation Bio is among the new wave of companies trying to avoid or overcome the challenges facing the current slate of gene therapies. To support its efforts, the Cambridge, MA-based company has raised an additional $110 million for its most advanced programs while also aiming for something more.

It gets us to the clinic, although its almost certain well take the company public ahead of that, McDonough says.

The Series C financing includes crossover investors, firms that back both private and public companies and whose involvement is viewed as a sign a company is preparing to go public. T. Rowe Price led Generation Bios latest round, joined by Farallon, and Wellington Management Company. Also participating in the round were earlier investors Atlas Venture, Fidelity, Invus, Casdin, Deerfield, Foresite Capital, and an entity associated with SVB Leerink. The latest round comes two years after Generation Bio closed a $100 million Series B round.

Currently available gene therapies (and some that are still in development) use an engineered virus to deliver their genetic payloads. But the virus can be a problem. Some people have preexisting antibodies to adeno-associated virus (AAV), the virus used in many gene therapies. And if patients dont already have those antibodies, they can develop them after being dosed with the treatment. That means patients cant receive another dose if they need it, and so far, the durability of gene therapies is not yet known.

Generation Bio avoids viral delivery altogether. The companys gene therapies use closed-ended DNA, or ceDNA, packaged inside a lipid nanoparticle. McDonough says it works just like AAV and it has the added advantage of being easier and less expensive to manufacture compared to AAV-based gene therapies. He adds that if patients need another dose, antibodies wont be a problem. But the company still needs to show that in clinical trials.

Generation Bios most advanced programs are for the blood disorder hemophilia A and phenylketonuria (PKU), an inherited metabolic disorder. There are other companies developing gene therapies for both diseases. Pfizer (NYSE: PFE) has taken over development of SB-525, a hemophilia A gene therapy candidate initially developed by its partner, Sangamo Therapeutics (NASDAQ: SGMO). BioMarin Pharmaceutical (NASDAQ: BMRN) on Monday announced that US and UK regulators cleared the company to begin clinical testing of BMN 307, its experimental gene therapy for PKU. Both experimental treatments use AAV to deliver the therapies to cells.

Besides supporting its hemophilia A and PKU clinical research, McDonough says the new financing will enable his company to look at other rare metabolic disorders such as Wilson and Gaucher disease. He adds that the company will also explore how its technology can be used to deliver therapies to more tissues in the body, such as skeletal tissue and the retina. By being able to reach more tissues, the goal is to expand the scope of gene therapy beyond rare diseases and bring it to the masses, he explains.

McDonough says Generation Bio has been careful not to work with other companies because its technology was still in development. But now that the technology is more mature, the biotech will explore research alliances with larger pharmaceuticalfirms.

I think we will do a partnership this year, he says. The question is in what domain and with which partner.

Photo by Jeremy Bishop on Unsplash

Frank Vinluan is an Xconomy editor based in Research Triangle Park. You can reach him at fvinluan [[at]] xconomy.com.

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Generation Bio Pockets $110M for Next Wave of Gene Therapy, IPO Plans - Xconomy

NEW YORK and GAINESVILLE, Fla., Jan. 13, 2020 /PRNewswire/ --The Muscular Dystrophy Association (MDA) and AavantiBio, a biotechnology company developing a gene-targeting therapy for Friedreich's ataxia (FA), today announced the award of MDA Venture Philanthropy (MVP) funding totaling $1,076,232 to advance AavantiBio's phase 2 clinical trial of a gene-replacement therapy for the disease.

MVP is MDA's drug development program, which is exclusively focused on funding the discovery and clinical application of treatments and cures for neuromuscular disorders. MVP evaluates and makes targeted investments in for-profit and not-for-profit companies and academics developing therapeutics.

FA is a rare, hereditary disease that causes damage to parts of the spinal cord and brain, and there is currently no cure. AavantiBio was founded with the aim of developing an effective treatment for the disease and improving the lives of FA patients.

"With the approval of several first-time gene-targeting therapies for neuromuscular diseases over the past several years, including the first-ever gene-replacement therapy for spinal muscular atrophy, our community should have much to hope for as more and more therapies continue to be developed," says Lynn O'Connor Vos, MDA's president and CEO. "MDA is thrilled to be a part of the quest to help further develop the first gene-targeting therapy for the treatment of Friedreich's ataxia. By partnering with AavantiBio, together we can address the unmet need faced by patients who live with this genetic disease, for which there are still no treatments and no cures."

Co-founders Manuela Corti, PT, PhD, assistant professor of Pediatrics at the University of Florida, Gainesville, and Barry Byrne, MD, PhD, associate chair of Pediatrics and director of the Powell Gene Therapy Center at UF, started working with the FA community five years ago and are thrilled about their new partnership with MDA.

"This is a great opportunity for AavantiBio, and we're thankful to the MDA for their generous contribution," Dr. Corti says. "We hope to strengthen our collaboration as we work together on this project."

Dr. Corti and Dr. Byrne aim to include both adults and children who have FA in their clinical trials, paving the way for new solutions.

"We're delighted to take the next step in fulfilling our commitment to patients and families living with FA based on this investment from MDA," Dr. Byrne says. "We're looking forward to initiating screening for the first clinical study and a pivotal study in FA before the end of the year. The endorsement and investment from the MDA will be key to our programmatic growth."

MDA's investment will help accelerate AavantiBio's mission and begin production of the clinical gene vector for its therapy program. Clinical trials are expected to begin in 2020.

Dr. Corti was previously awarded an MDA research grant to develop and test a gene-replacement therapy in a mouse model of FA. With the current funding, AavantiBio will sponsor a study at the University of Florida led by Sub Subramony, MD, professor of Neurology. The clinical trial will assess changes in neurological and cardiac function in patients with FA treated with both intravenous (systemic) and intrathecal (into-the-spine) injections of the company's gene-replacement therapy for the mutated FXN gene.

About Friedreich's ataxia

FA is a mitochondrial disease. Mutations in the frataxin gene (FXN) lead to decreased production of the frataxin protein, resulting in diminished energy production in cells, including those of the nervous system and heart. FA's major neurological symptoms include muscle weakness and ataxia, or a loss of balance and coordination. FA mostly affects the spinal cord and the peripheral nervesthat connect the spinal cord to the body's muscles and sensory organs, but it can also affect the cerebellum (causing ataxia) and heart. The prevalence of FA has been estimated at 1 in every 50,000 individuals worldwide. Symptoms typically begin between the ages of 5 and 15 years, and the rate of progression varies from person to person. There currently are no effective cures or treatments for FA.

About AavantiBio

Founded in 2017 and based inGainesville, Fla., AavantiBio is a biotechnology companyfounded on the vision of creating the first effective treatment for FA. The company's gene-replacement therapy approach uses an adeno-associated virus (AAV) vector to deliver a functional copy of the FXN gene to a patient's cells.

The co-founders bring more than 30 years of research experience in the field of neuromuscular disease, including their current work focusing on FA. Dr. Corti has more than 10 years of research experience in gene therapy approaches for the treatment of Duchenne muscular dystrophy and Pompe disease. Dr. Byrne has made significant contributions to theunderstanding and treatment of Pompe disease. He was previously awarded nearly $2 million in MDA funding to conduct foundational research in developing and testing AAV vectors in animal models of muscular dystrophy, and he was the first to show that AAV vectors are able to effectively express therapeutic genes in striated muscle cells.

About the Muscular Dystrophy Association

MDA is committed to transforming the lives of people affected by muscular dystrophy, ALS, and related neuromuscular diseases. We do this through innovations in science and innovations in care. As the largest source of funding for neuromuscular disease research outside of the federal government, MDA has committed more than $1 billion since our inception to accelerate the discovery of therapies and cures. Research we have supported is directly linked to approved, life-changing therapies across multiple neuromuscular diseases. We support the largest network of multidisciplinary clinics providing best-in-class care at more than 150 of the nation's top medical institutions, and each year thousands of children and young adults learn vital life skills and gain independence at MDA Summer Camp and through recreational programs. For more information visitmda.org.

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MDA Awards Venture Philanthropy Funding of More Than $1M to AavantiBio to Develop Gene-Targeting Therapy for Friedreich's Ataxia - P&T Community

CAMBRIDGE, Mass., Jan. 13, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced that it has appointed John C. Martin, Ph.D., to its Board of Directors, effective today. Dr. Martin brings decades of executive leadership to Sareptas board, having played an instrumental role in building one of the worlds foremost biotechnology companies. During his 20-year tenure as chief executive officer of Gilead Sciences, he oversaw the growth of the company and development of its scientific portfolio into 24 marketed products.

Johns business record is unparalleled, and he brings expertise and insight gleaned from leading the long-term, transformational growth of one of the industrys premier companies that will add to the strength of the Sarepta board. We are incredibly fortunate to welcome him to the companys board of directors, saidM. Kathleen Behrens, Ph.D., Chairperson of Sareptas Board of Directors.

Having introduced curative therapies, Johns vision, acumen, and wisdom are unique in our industry. As Sarepta prepares to deliver one-time therapies for rare diseases, Johns strategic guidance as a member of the companys board will be invaluable as we work to change the model for how rare diseases are treated, said Doug Ingram, Sareptas president and chief executive officer.

Dr. Martin served as the Executive Chairman and Chairman of Gilead Sciences, Inc., from March 2016 through March 2019, after having served as Chairman and Chief Executive Officer from June 2008 through March 2016 and President and Chief Executive Officer from 1996 through May 2008. He joined Gilead in 1990 as Vice President, Research and Development. Prior to Gilead, Dr. Martin held several leadership positions at Bristol-Myers Squibb and Syntex Corporation. Martin currently serves on the Board of Directors at The Scripps Research Institute and Kronos Bio. Additionally, he served on the Centers for Disease Control/Health Resources and Services Administrations Advisory Committee on HIV and STD Prevention and Treatment and was a member of the Presidential Advisory Council on HIV/AIDS. In 2008, Dr. Martin was inducted into the National Academy of Engineering and, in 2019, he received the National Academy of Sciences Award for Chemistry in Service to Society.

Dr. Martin holds a Ph.D. in organic chemistry from the University of Chicago, an MBA from Golden Gate University and a B.S. degree in chemical engineering from Purdue University.

Heidrick & Struggles led the search process for Sarepta.

AboutSarepta TherapeuticsSarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for Limb-girdle muscular dystrophy diseases (LGMD), Charcot-Marie-Tooth (CMT), MPS IIIA and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases. For more information, please visit http://www.sarepta.com.

Forward-Looking StatementsThis press release contains forward-looking statements. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the expectation that Dr. Martins expertise and insight will add to the strength of the Sarepta board and that Dr. Martins strategic guidance will be invaluable as Sarepta prepares to deliver one-time therapies for rare diseases; and Sareptas mission to profoundly improve and extend the lives of patients with rare genetic-based diseases.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: Sarepta may not be able to execute on its business plans, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its products to U.S. and ex-U.S. markets for various reasons including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2018 and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Companys business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review Sarepta's 2018 Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Internet Posting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc. Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics Appoints Renowned Biotech Executive John C. Martin to its Board of Directors - GlobeNewswire