Archive for the ‘Gene Therapy Research’ Category

Gene therapy developers targeting the US market have a clearer idea of what it will take to win approval thanks to new guidance documents issued by the FDA.

The US regulator set out its expectations for developers in six documents published last week. There are final guidance documents on gene therapies for hemophilia, retinal disorders and rare diseases.

In addition, there are final guidance documents on chemistry, manufacturing and controls (CMC), observational studies and on the assessment of gene therapies that use retroviral vectors.

Image: iStock/Piotrekswat

There is also draft guidance explaining how the FDA will interpret sameness when deciding whether to award orphan drug status.

The agency said that when assessing two gene therapies targeting the same disease it will consider both the gene itself and the viral vector.

To date only four gene therapies that introduce new genetic material into a patients cell have been approved by the FDA.

However, many more gene therapies are on the way according to the agency which said it anticipates more approvals in the coming years based on the 900 investigational new drug (IND) it has received.

FDA Commissioner Stephen Hahn said, The growth of innovative research and product development in the field of gene therapy is exciting to us as physicians, scientists and regulators.

These therapies, once only conceptual, are rapidly becoming a therapeutic reality for an increasing number of patients with a wide range of diseases, including rare genetic disorders and autoimmune diseases.

This was echoed by Peter Marks, director of the FDAs Center for Biologics Evaluation and Research, who stressed the importance of effective gene therapy regulations.

As the regulators of these novel therapies, we know that the framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field and further enable innovators to safely develop effective therapies for many diseases with unmet medical needs.

Marks cited the rapid pace of scientific development as a challenge for the agency, explaining that assessing the safety, efficacy and long-term impact of gene therapies is highly complex.

Read more here:
US FDA predicts gene therapy surge - Bioprocess Insider - BioProcess Insider

With more than 900 investigational new drug (IND) applications for ongoing clinical studies related to gene therapies, and with the number of advanced therapy medicinal products at clinical stage worldwide exceeding 1,000, the US Food and Drug Administration (FDA) this week released a number of policies.

The policies, addressed to developers and manufacturers, include six final guidance documents on gene therapy manufacturing and clinical development of products, following up to respective draft guidance documents released in 2018, and a draft guidance related to orphan drug designations for therapeutic candidates.

Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review, commented Peter Marks, director of the FDAs Center for Biologics Evaluation and Research, adding The framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field.

Regarding the draft guidanceInterpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations, the agency explained that it focuses on how the FDA will evaluate differences between gene therapy products when they are intended to treat the same disease.

The final guidance titled Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) aims to inform sponsors on how to provide sufficient CMC information, in order to assure product safety, identity, quality, purity, and strength (including potency) of the investigational product and to be able to claim market authorization from the regulatory body.

Addressed to developers and manufacturers of retroviral vector-based human gene therapy products, the second document titled Testing of Retroviral Vector-Based Gene Therapy Products for Replication Competent Retrovirus (RCR) during Product Manufacture and Patient Follow-up determines testing for RCR during manufacture, as well as the regulations for follow-up monitoring of patients who have received such treatments.

Titled Long-Term Follow-Up After Administration of Human Gene Therapy Products, the third document includes recommendations by the FDA regarding the design of long-term follow-up studies for the collection of data on delayed adverse events.

Specifically, the FDA suggests that, as a result of long-term exposure to an investigational gene therapy, patients may be at increased risk of undesirable and unpredictable outcomes, and therefore they may be monitored for an extended period of time past the active follow-up period. The document outlines several factors based on which a risk assessment should be performed to determine the necessity of long-term monitoring for each product.

Another guidance of the FDA is focused on Human Gene Therapy for Hemophilia, and it provides recommendations regarding the clinical trial design for such therapies, as well as addressing discrepancies between Hemophilia A and B coagulation factors activity assays.

Focusing on Human Gene Therapy for Retinal Disorders, the fourth FDA guidance includes recommendations related to product development, preclinical testing, and clinical trial design for such gene therapy products.

Finally, the guidance on Human Gene Therapy for Rare Diseases, with suggestions on the clinical design for such products, is needed, according to the FDA, due to the limited study population size and potential feasibility and safety issues. Moreover, the FDA cites issues related to the interpretability of bioactivity/efficacy outcomes that may be unique to rare diseases or to the nature of the product.

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FDA guidance on gene therapies development and manufacturing - BioPharma-Reporter.com

Abstract / Synopsis:

Research is finding key patient benefits to gene therapy as a promising treatment strategy for Leber's hereditary optic neuropathy (LHON).

This article was reviewed by Patrick Yu-Wai-Man, FRCOphth, FRCPath, BMedSci, MBBS, PhD

Data from two clinical studies of Lebers hereditary optic neuropathy (LHON) showed substantial visual improvements in patients with both disease durations of less than six months and between six months and one year. The improvements resulted from a unilateral injection of a gene therapy vector (GS010) and remarkably, the viral vector seemed to be carried over to the untreated eye.

The mechanism of action for these unexpected results need to be clarified with further experimental work.

Related: Research targets precision dosing for gene, cell therapy

LHON is the most common cause of mitochondrial blindness with a minimal prevalence of one in 30,000 individuals in the population. It causes blindness mostly in young adult men with a peak age of onset in the third decade of life. It is invariably a bilateral disorder in which the fellow eye becomes affected within three to six months after disease onset in the first eye.

Both eyes are affected simultaneously in about 25% of patients, according to Patrick Yu-Wai-Man, FRCOphth, FRCPath, BMedSci, MBBS, PhD, an academic neuro-ophthalmologist with faculty positions at the University of Cambridge, Moorfields Eye Hospital, and the UCL Institute of Ophthalmology, in the UK.

Three primary mutations within the mitochondrial genome cause about 90% of cases worldwide, namely, m.3460G>A, m.11778G>A and m.14484T>C, with m.11778G>A being the most common mutation by far, accounting for over 70% of those affected with LHON. Unfortunately, most affected patients remain legally blind with vision worse than 1.3 logarithm of the minimum angle of resolution (logMAR) or 3/60 in Snellen equivalent.

Given the poor prognosis, there is an urgent clinical need to identify effective treatments for this blinding optic nerve disease.

Related: LHON gene therapy: Deciphering phase III data

TreatmentGene therapy is obviously a very attractive treatment option, because the underlying pathophysiology is due to insufficient amount of the wild-type protein, Dr. Yu-Wai-Man said. Therefore, if the defective gene is replaced, we should be able to rescue the retinal ganglion cells, preserving function and improving the visual prognosis.

He described the principles of allotopic gene expression that involves inserting the mitochondrial gene of interest, in this case MTND4, into the nuclear genome with a modified viral vector. The wild-type protein produced has a specific mitochondrial targeting sequence that directs it to be imported into the mitochondrial compartment.

The use of an intravitreal injection is a big advantage for this treatment approach as it is a relatively straightforward procedure that provides direct access to the inner retina. Previous preclinical work indicates that allotopic expression is able to rescue the retinal ganglion cells from the deleterious effects of the m.11778G>A mutation.

Related: Gene therapy offering hope for retinal, corneal patients

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Studies target unilateral gene therapy injection - Ophthalmology Times

Investment Thesis

Objective: Wealth-building of an always fully-invested portfolio via repeated near-term (weeks or months) capital gains from careful, diversified, odds-on issue selection and timely price opportunity capture.

The stocks compared here are Sarepta Therapeutics, Inc. (NASDAQ:SRPT) and Arrowhead Pharmaceuticals Inc. (NASDAQ:ARWR). SRPT was inadvertently omitted from the Biotech Developer review recently published. Rather than picture it by itself, ARWR is provided here as a best-ranked alternative.

These market pros have insights you and I can't have because their everyday job is to satisfy investment organizations running billion-dollar portfolios who want to adjust their holdings in multi-million-dollar trade transactions. These market-makers [MMs] have to round up sellers when their clients want to buy, and buyers when they want to sell. That's hard to do when most investors will hold off to get better prices, whether they are buying or selling.

And when lots of money is involved in each trade, the players get pretty careful about what they want to do and when they will do it. But the big-money types work hard to be on top of developments, following some issues intently, anticipating what is likely to be happening to stock prices in the near future. Depending on what they know, or think they know, and what they think others believe is likely to happen, they may take surprising postures. Often their holding-period horizons are months, not years. Or even less.

So, the MMs have to respond when a big-money house says "sell a bunch of this and buy a lot of that, and do it in the next 15 minutes, or you can forget about keeping us as a good repeat-order client".

The MMs will round up any of their other clients who they know have holdings in the stocks or appetites (at a price) to initiate, expand or contract holdings in the issues involved. It's rare when a "cross" can be made with enough "other side of the trade" exists at acceptable prices to "fill" the trade order without having to put some of their own firm's capital at risk in order to balance buying demand with selling supply.

As market-makers, they will provide the balancing position when they can set up a hedge deal to protect the market risk involved in their "facilitation" of the trade's being completed. If they can't, then the trade order gets killed, not filled, to wait for a time when the market is more accommodating.

But what it takes to buy that market risk price-change protection for the MM tells what the players on both sides of the "insurance" market believe can happen to the stock's price during the limited lives of the derivative contracts for options, futures, swaps, or other highly leveraged involved instruments used in the hedge.

That's where we find the balances between forecast upside price gain prospects and price drawdown exposures today and can compare them with what has been seen day by day over the past couple of decades. That is what supports the opening statements above about +15% to +30% gains in specific stocks. Those are fact-based histories of all prior real-market experiences from forecasts made before the fact, not just blown smoke over some after-the-fact single illustration of convenient history.

"Sarepta Therapeutics, Inc. focuses on the discovery and development of RNA-based therapeutics, gene therapy, and other genetic medicine approaches for the treatment of rare diseases. The company offers EXONDYS 51, a disease-modifying therapy for Duchenne muscular dystrophy (DMD). Its products pipeline include Golodirsen, a product candidate that binds to exon 53 of dystrophin pre-mRNA, which results in exclusion or skipping of exon during mRNA processing in patients with genetic mutations; and Casimersen, a product candidate that uses phosphorodiamidate morpholino oligomer [PMO] chemistry and exon-skipping technology to skip exon 45 of the DMD gene. In addition, the company's pipeline comprises SRP-5051, a peptide conjugated PMO that binds to exon 51 of dystrophin pre-mRNA. It has collaboration agreements with Nationwide Children's Hospital to advance micro-dystrophin gene therapy program under the research and license option agreement; Galgt2, a gene therapy program for the treatment of DMD; and Neutrophin 3, a gene therapy program to treat Charcot-Marie-Tooth neuropathies. The company also has a license agreement with Lysogene to develop LYS-SAF302, a gene therapy for mucopolysaccharidosis IIIA; a license and option agreement with Lacerta to develop treatments for CNS-targeted and lysosomal storage diseases; and research collaboration and option agreement with Genethon to develop micro-dystrophin gene therapy products. In addition, it has a research agreement with Duke University to advance gene editing CRISPR/Cas9 technology for restoring dystrophin expression; a collaboration agreement with Summit (Oxford) Ltd. to commercialize products in Summit's utrophin modulator pipeline; a strategic collaboration with Paragon Bioservices; and a strategic collaboration with CENTOGENE for the identification of patients with DMD in the Middle East and North Africa region. Sarepta Therapeutics, Inc. was founded in 1980 and is headquartered in Cambridge, Massachusetts."

Source: Yahoo Finance

SRPT's and ARWR's recent daily price ranges over the past 6 months are shown in Figures 1 and 2, along with measures of their current forecast price up-to-down balances. Also shown are the odds of long position gains being earned in the couple of months subsequent to points in time in the past 5 years when MMs had the same kind of outlook they have today.

Figure 1

source: Author

Figure 2

source: Author

As a contrast, here is what MM forecasts for the "market-index" ETF of SPDR S&P 500 Trust (SPY) looks like at this time:

Figure 3

source: Author

How effective the MMs have been in forecasting for these stocks is a matter of market records, when conditions of uncertainty similar to today's are examined. That was done in the row of data between the graphics of Figures 1-3. For ease of comparison, they are repeated and slightly expanded in Figure 4.

Figure 4

source: Author

As explained in the prior Biotech Developer revue featuring ARWR, the SRPT Range Index [RI] of 23 produced 108 of 124 net gain %Payoffs under TERMD of +30.1%.

A comparison of the +30.1% payoffs with the present forecast of +23.1% suggests an exceptional profit achievement with a degree of credibility for the current outlook of 1.30, as indicated at column [N] of Figure 4.

So much for the "good side" of a buy proposition; what about the "bad side"?

As we condition the credibility of the upside price change forecast by comparison with actual experience, so too do we look to see how bad the downside might get. But with concern only during those "long" holding periods when committed capital would be at risk under the TERMD discipline. All other periods are irrelevant, shocking as they may be.

Figure 1's data row tells what the worst case price drawdowns have been (an average of them) during all of each actual exposure period when they were to be held. What matters is how bad a fear of loss may get induced any time, not just whether or not it existed at the end of the holding. Investors will have varied reactions to the exposures, so there is no way to evaluate potential risk impact by historic outcomes. But some useful guidance may be provided by having knowledge of the maximum degree of intensity possibly becoming present.

One logically-simplified way to address the combination of stock price risk and reward is to weight each part by its probability and combine the two. The "Win Odds" of profitable position odds here for SRPT of 108 out of 124, or 87 out of 100 offer such a probability. One minus those odds, or 100 - 87 provides the loss probability weight. Thus 0.87 times +30.1% plus 0.13 times -8.7% produces a weighted net payoff of +25.1%.

To make this style of evaluation more comparable between varied investment opportunity situations, an integration of the likely holding periods used in the calculation is helpful. For SRPT, the average number of market days required by all 129 positions of the sample was only 40 out of the maximum 63 possible, because of the high proportion of upside target prices reached.

A standard evaluation measure used in many capital planning decision situations is the expected net payoff stated in "basis points" of 1/100ths 1%, per day of capital involvement. On a 365-day calendar year +19 bp/day when sustained for a year doubles the original capital, or a CAGR of +100%. When a smaller-count of 252 market days makes up a relevant year, the fewer days are each proportionally more powerful, so only 14 bp/mkt day does the 100% equivalent.

Comparison is the essence of evaluation. If the investing objective is to make capital as productive of future spend-able amounts as possible, using an odds-weighted bp/d yardstick can be helpful.

To that end, Figure 4 includes the relevant MM forecasts and their prior outcomes for ARWR and the market-index proxy of the SPDR S&P 500 Index ETF (SPY). Also, the average of some 2,700 current-day MM price-range forecast issues, and a ranked set of the day's likely 20 best of those near-term wealth-building stocks under TERMD portfolio discipline.

All of these comparisons in Figure 4 have the same basic data as included in the row of Figure 1 for SRPT. That is expanded by the columns [O] through [R] to provide for odds-weighted bp/day price-prospect evaluation comparisons.

Competition from the market-index alternative SPY at this point in time is rather limited because of an unenthusiastic upside target outlook of only +5.5% at a CAGR of only +9% and an Odds-Weighted net prospect [Q] of +0.8%. That is better, though, than the overall population of 2,711 where MM forecasts are a modest net decline (-2.2%).

Sarepta Therapeutics, Inc. and Arrowhead Pharmaceuticals Inc. both offer outstanding prospects for capital gains with strong odds for achievement in short periods of holding. SRPT has the larger potentials, but ARWR appears historically to have quicker achievement prospects. Payoff potentials in basis points per day are exceptional. For further information, please check my blog here on Seeking Alpha.

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

Additional disclosure: Disclaimer: Peter Way and generations of the Way Family are long-term providers of perspective information, earlier helping professional investors and now individual investors, discriminate between wealth-building opportunities in individual stocks and ETFs. We do not manage money for others outside of the family but do provide pro bono consulting for a limited number of not-for-profit organizations.We firmly believe investors need to maintain skin in their game by actively initiating commitment choices of capital and time investments in their personal portfolios. So, our information presents for D-I-Y investor guidance what the arguably best-informed professional investors are thinking. Their insights, revealed through their own self-protective hedging actions, tell what they believe is most likely to happen to the prices of specific issues in coming weeks and months. Evidences of how such prior forecasts have worked out are routinely provided in the SA blog of my name.

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Sarepta Therapeutics, Inc. And Arrowhead Pharmaceuticals Now Top-Ranked Biotech Stock Buys - Seeking Alpha

Microsoft announced a new five-year, $40 million artificial-intelligence initiative geared toward global health challenges and research.

Dubbed AI for Health, the program will work to equip academia and non-profit research organizations for their medical research efforts, including the development of diagnostics, treatments and preventive measures. It also aims to address global health crises and healthcare disparities.

"We know that putting this powerful technology into the hands of experts tackling this problem can accelerate new solutions and improve access for underserved populations, Microsoft President Brad Smith said in a statement.

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The tech giant hopes to tackle the uneven distribution of data science expertise as well: Microsoft estimates that less than 5% of the worlds AI professionals work in healthcare and non-profit organizations.

The new program will build upon the companys previous collaborations, like individual efforts focused on sudden infant death syndrome, leprosy and diabetic retinopathy, as well as its work on developing a secure system for sharing biomedical data.

AI for Healths first set of grantees include the Novartis Foundation, Fred Hutchinson Cancer Research Center, Seattle Children's Research Institute, PATH, Intelligent Retinal Imaging Systems and the international development organization BRAC.

RELATED: Novartis to put AI on every employee's desk through Microsoft partnership

"Countries like Bangladesh, where BRAC was founded, have made enormous strides in health equity in the last three decades. Unfortunately, at least half the world's population still lacks access to essential health services," said BRACs executive director, Asif Saleh. "Across our outreach areas in Asia and Africa, we see massive potential in using advanced data analytics and AI to bridge the gap between 'health for some' and 'health for all,' and we welcome Microsoft's commitment in making this happen."

Last year, Microsoft signed on to a wide-ranging partnership with Novartis to help put AI tools on the desk of each of the drugmakers research associates. That five-year project will also establish a joint innovation lab tasked with upgrading the Big Pharmas R&D, clinical trials and manufacturing efforts.

It will also take on research projects in macular degeneration and irreversible blindness, cell and gene therapy manufacturing efficiency and expediting the design of new drug compounds. Meanwhile, Microsofts new work with the companys foundation will focus on limiting the spread of disease.

"Leprosy is one of the oldest diseases known to humans, but today an estimated 2 to 3 million people are still living with the disease," said Ann Aerts, head of the Novartis Foundation. "Around the world, we are working to accelerate efforts to eliminate leprosy by focusing on interventions that aim to interrupt transmission. The use of AI is transformative and a game-changer in how we can accelerate progress and scale our work to reach the people in need."

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Microsoft launches 5-year, $40M AI initiative in global health - FierceBiotech

The Discovery Labs and Deerfield Management Company have formed The Center for Breakthrough Medicines, a contract development and manufacturing organization (CDMO). The CDMO is occupying over 40 percent (680,000 sq. ft.) of The Discovery Labs' 1.6 million square foot biotech, healthcare and life sciences campus in King of Prussia, PA.The CDMO provides preclinical through commercial manufacturing of cell and gene therapies and component raw materials. It offers process development, plasmid DNA, viral vectors, cell banking, cell processing, and support testing capabilities all under one roof. The company said the $1.1 billion facility will provide instant capacity as the largest known single source for accelerating the delivery and affordability of lifesaving and life-changing therapies from the bench to the patient's bedside.The company has initiated a substantial hiring effort, expecting to hire over 2,000 team members within the next 30 months.In addition to developing the world's largest single-point cell and gene therapy manufacturing facility, The Discovery Labs is establishing THE COLONY which will provide custom built discovery labs, breakthrough funding, sponsored research agreements, housing and relocation for the world's leading iconic experts in cell and gene therapy.THE COLONY will seek to work hand in hand with scientists from both academic and pharmaceutical institutions to unlock and expedite groundbreaking therapies.Marco A. Chacn, founder of Paragon Bioservices and chairman of The Discovery Labs said, "Musicians, artists, members of religious communities and great thinkers throughout time have formed colonies where freedom of thought and expression combined with unlimited dreams and potential have resulted in the world's greatest accomplishments. The United States of America is a perfect example. The goal of THE COLONY is to unshackle the potential of the world's greatest scientific minds."Audrey Greenberg, board member and executive managing director, The Discovery Labs, said, "The Center for Breakthrough Medicines will be serving companies from the earliest stages through commercialization. Its exceptional scale and offering will quickly relieve the production bottleneck for advanced therapies by reducing the time, complexity, and cost of commercializing vitally needed gene and cell therapies.The addition of this end-to-end manufacturing capability is expected to significantly enhance the offerings of The Discovery Labs in an area that has become one of the largest life sciences hubs in the world. Renovations are underway to construct a total of 86 plasmid, viral vector production, universal cell processing, CGMP testing, process development and cell banking suites. The viral vector and cell processing suites will be fully compliant with both U.S. Food and Drug Administration and European Medicines Agency standards. All suites will offer the flexibility to meet client-specific workflows and will be able to adapt quickly to meet demand. The company is in the process of reserving capacity now for late 2020."Today brilliant scientists are advancing an unprecedented number of gene and cell therapy drug candidates. The real tragedy, however, is a scarcity of manufacturing know-how, which is complex and expensive," said Alex Karnal, partner and managing director, Deerfield Management and a board member of the Discovery Labs. "With its visionary business model, it is hoped that The Center for Breakthrough Medicines will help realize the promise of cell and gene therapies in time to treat the many patients who need them."The Discovery Labs provides a central campus where the world's greatest scientists can collaborate on new therapeutic discoveries to eradicate diseases affecting small and large segments of the global population. The Center for Breakthrough Medicines will work with these leaders, life sciences companies, large pharmaceutical companies, and academic and government institutions.This new manufacturing capability is a transformational addition to The Discovery Labs market offering and dovetails with The Discovery Labs biotech incubator, Unite IQ. Unite IQ offers immediate space to emerging life sciences companies and scientists giving them the ability to grow from startup to enterprise company on one campus. The incubator and accelerator space at Unite IQ provides a comprehensive home for startups with every resource needed to initiate business operations. Unite IQ tenants are expected to utilize the discovery, development, testing, and manufacturing capabilities of the Center for Breakthrough Medicines with seamless forward integration of processes and analytics, and seamless tech transfer from research lab to large scale production.

Continued here:
Cell & Gene Therapy CDMO Begins Operations - Contract Pharma

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Dr. Mahendran Mahadevan, a professor in the Department of Obstetrics and Gynecology at the University of Arkansas for Medical Sciences, willspeakfrom 3-4 p.m. Monday, Feb.3, in Room D2 of the Food Science Building, 2650 N. Young Ave. His presentation, "Food, Health, and Disease,"is open to everyone.

Mahadevan's presentation will focus on how different types of food and beverages plays a role in human body's health defense systems (Angiogenesis, stem cells/regeneration, microbiome, DNA protection, and immunity). This basic biological knowledge related to foods will be useful for better understanding about the effects of foods on the prevention and management of human diseases.

Mahadevan's research interests include: 1) roles of genetics, obesity, nutrition, food supplements, nutraceuticals, physical activity and other environmental/life style factors on prevention/public health and maternal, fetal, and child health; 2) tissue banking; 3) embryo and stem cell culture/expansion (particularly culture medium/conditions); and 4) gene therapy and stem cell gene therapy particularly in cancer and genetic diseases.

Mahadevan received his Veterinary medicine degree in 1975 from University of Ceylon Peradeniya, Sri Lanka and his doctoral degree in Reproductive biology from Monash University, Australia in 1982. His academic experience includes faculty positions in the Department of Physiology, School of medicine at the University of Ceylon and in the Department of Obstetrics and Gynecology at the University of Arkansas for Medical Sciences.

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UAMS Professor to Present Relationships Among Food, Health, and Disease in Food Science Seminar - University of Arkansas Newswire

DUBLIN, Jan. 31, 2020 /PRNewswire/ -- The "Global Healthcare Market Outlook, 2020" report has been added to ResearchAndMarkets.com's offering.

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Amid rising global trade tensions and sluggish global economic outlook for 2020, the global healthcare market is expected to cross the $2 trillion mark in 2020.

Healthcare will be among the top two priorities for voters in the 2020 presidential election in the US. In the European region, looming BREXIT indecision is likely to have a strong impact on Europe's biggest digital health market (UK). Globally, 2020 will be a reality check for long-pending national healthcare policies and regulatory reforms that must re-invigorate future strategies.

The new vision for healthcare for 2020 and beyond will not just focus on access, quality, and affordability but also on predictive, preventive, and outcome-based care models promoting social and financial inclusion. Social Determinants of Health (SDOH) will emerge has a big theme across progressive health systems to proactively engage the right patients and improve health outcomes to help healthcare organizations meet quality standards. In 2020, consumer-driven models of healthcare will gain more market traction, as they stand to better bridge the gap of what consumers want and what healthcare can deliver.

Continued steps will be taken by retail (Walmart, Costco, Amazon, Ali Health), and consumer tech (Google, Apple, Microsoft, and so on) companies globally; to make further headway (intrude) into vetted healthcare space. In 2020, the convergence of Artificial Intelligence (AI), Blockchain, and the Internet of Things (IoT) will further catalyze the space of innovation adoption and related applications in the healthcare realm. For example, while Blockchain will improve data liquidity to empower AI and analytics vendors/applications to digest a large amount of data, AI can manage Blockchain systems more efficiently than humans.

Research Scope

Every year, the team of futurists, analysts, and consultants at the publisher's Transformational Healthcare Group come together to render a comprehensive analysis to predict the themes, technologies, and global forces that will define the next 12 to 18 months (future) for the healthcare industry.

As a part of this research deliverable, the publisher provides bold perspectives and predictions for the global healthcare market in 2020. The sectors covered include pharmaceuticals and biotech, in-vitro diagnostics, medical technologies, medical imaging, and healthcare IT. The analysis captures sectoral and regional trends and provides predictions for the upcoming year. The study provides guidance on where to find the greatest opportunities for expansion.

Predictions for the global healthcare market in 2020 include:

Key Issues Addressed

Key Topics Covered

1. Executive Summary

2. Revisiting 2019 Predictions

3. Global Healthcare Market Outlook for 2020

4. Key 2020 Healthcare Market Predictions

5. Regional Predictions 2020

6. Sector Outlook 2020

7. Key Conclusions

For more information about this report visit https://www.researchandmarkets.com/r/o8dr1g

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

Media Contact:

Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

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View original content:http://www.prnewswire.com/news-releases/global-healthcare-market-study-2020-perspectives--predictions-for-the-pharmaceuticals--biotech-in-vitro-diagnostics-medical-technologies-medical-imaging-and-healthcare-it-sectors-300996869.html

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Global Healthcare Market Study 2020: Perspectives & Predictions for the Pharmaceuticals & Biotech, In-Vitro Diagnostics, Medical Technologies,...

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A collective analysis of Sulfur Palletized Plant And Granulator Market offering an exhaustive study based on current trends influencing this vertical across various geographies has been provided in the report. Also, this research study estimates this space to accrue considerable income during the projected period, with the help of a plethora of driving forces that will boost the industry trends during the forecast duration. Snippets of these influences, in tandem with countless other dynamics relating to the Sulfur Palletized Plant And Granulator Market, like the risks that are predominant across this industry along with the growth prospects existing in Sulfur Palletized Plant And Granulator Market, have also been charted out in the report.

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Gene Therapy Market Segments and Key Trends 2019-2029 - Melanian News

TMRR in its latest research report states that the global market size of Gene Therapy and Antisense Drugs market was $XX million in 2018 with XX CAGR from 2014 to 2018, and is expected to reach $XX million by the end of 2029 with a CAGR of XX% from 2019 to 2029.

Global Gene Therapy and Antisense Drugs Market Report 2019 Market Size, Share, Price, Trend and Forecast is an intuitive and exhaustive study on the current and future prospects of the global Gene Therapy and Antisense Drugs industry. The key insights are elucidated as under:

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There are 4 key segments covered in this report: machine segment, product type segment, end use segment and regional segment.

Competitive landscape of Gene Therapy and Antisense Drugs market has tier 1, tier 2 and tier 3 players and provides a dashboard view of their strategies and intensity mapping.

Segmentation

On the basis of therapeutic area, the gene therapy and antisense drugs market is segmented into cancer, anemia, rheumatoid arthritis, cardiovascular diseases, HIV/AIDS, cystic fibrosis, diabetes mellitus and obesity, and renal diseases.

By gene transfer method, ex vivo gene transfer and in vivo gene transfer are the segments of the market. The former involves the transfer of cloned genes into cells, i.e., cells are altered outside the body before being implanted into the patient, whereas the latter involves the transfer of cloned genes directly into the patients tissues. The outcome of in vivo gene transfer technology mainly depends on the general efficacy of gene transfer and expression.

Global Gene Therapy and Antisense Drugs Market: Regional Outlook

The global gene therapy and antisense drugs market is segmented into North America, Asia Pacific, Europe, and Rest of the World. Amongst these, North America holds the leading position in the market followed by Europe. The increasing incidence of cancer and other fatal diseases, unhealthy lifestyle practices such as excessive smoking and excessive consumption of high fat content food, and increasing research efforts for treatment against cancer are the major factors driving the gene therapy and antisense drugs market in these regions.

Asia Pacific is expected to emerge as a significant market for gene therapy and antisense drugs. The high population density including a large geriatric population, expeditiously increasing demand for technologically advanced therapeutics, and increasing government support for improved healthcare infrastructure in the region is driving the growth of this regional market. Furthermore, favorable reimbursement policies and tax benefits on newer therapies will further fuel the growth of the Asia Pacific gene therapy and antisense drugs market.

Major Companies Mentioned in Report

Some of the leading companies operating in the global gene therapy and antisense drugs market are GenVec Inc., Avigen Inc., Genome Therapeutics Corp., Tekmira Pharmaceuticals Corporation, Isis Pharmaceuticals, Cell Genesys Inc., and others. These companies are profiled for their key business attributes in the report.

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For regional segment, the following regions in the Gene Therapy and Antisense Drugs market have been covered

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Gene Therapy and Antisense Drugs Market size and Key Trends in terms of volume and value2018 2028 - Dagoretti News

Cannabis, and what it might be able to do for human health, inspires me. And I think it can do the same for other scientists and scientists to be.

Newlegal frameworksandemerging clinical researchhave opened the door to research on cannabis and the compounds it makes. In just a few years, weve gone from having a rudimentary understanding of the plants compounds to discoveringcrucial connectionsamong cannabinoids and human physiology. Our broader understanding of these plant compounds is boosting the STEM job market.

Weve only begun to scratch the surface of clinical research for the medical and health applications of cannabis. This exploration cant happen without collaboration between organic chemists, biologists, botanists, agronomy experts, medical doctors, pharmacologists, and experts in clinical trials, drug formulation, and therapeutic product design.

The potential for new and innovative medications and wellness products derived from cannabis could be huge. Promising applications include reducing or relieving pain, quelling nausea, and easing seizures or the symptoms of Parkinsons disease. Researchers are investigating myriad other conditions that may respond well to cannabinoids.

Theres no question that the cannabis industry needs scientists. But it looks as though scientists need the cannabis industry as well.

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'We've only begun to scratch the surface': How the cannabis boom opens the door to new medicines and wellness products - Genetic Literacy Project

NEW YORK, Jan. 30, 2020 /PRNewswire/ --

High incidence of cancer & other target diseases is a major factor driving the growth of the gene therapy market

Read the full report: https://www.reportlinker.com/p05843076/?utm_source=PRN

The global gene therapy market is projected to reach USD 13.0 billion by 2024 from USD 3.8 billion in 2019, at a CAGR of 27.8% during the forecast period. The high incidence of cancer and other target diseases, availability of reimbursement, and the launch of new products are the major factors driving the growth of this market. In addition, the strong product pipeline of market players is expected to offer significant growth opportunities in the coming years. However, the high cost of treatment is expected to hamper market growth to a certain extent in the coming years.

Neurological diseases segment accounted for the largest share of the gene therapy market, by indication, in 2018Based on indication, the market is segmented into neurological diseases, cancer, hepatological diseases, Duchenne muscular dystrophy, and other indications.The neurological diseases segment accounted for the largest share of the market in 2018.

This can be attributed to the increasing number of gene therapy products being approved for the treatment of neurological diseases and the high market penetration of oligonucleotide-based gene therapies.

Viral vectors segment to register the highest growth in the gene therapy market during the forecast periodThe gene therapy market, by vector, has been segmented into viral and non-viral vectors.In 2018, the non-viral vectors segment accounted for the largest share of this market.

However, the viral vectors segment is estimated to grow at the highest CAGR during the forecast period, primarily due to the increasing demand for CAR T-based gene therapies and the rising incidence of cancer.

North America will continue to dominate the gene therapy market during the forecast periodGeographically, the market is segmented into North America, Europe, the Asia Pacific, and the Rest of the World.In 2018, North America accounted for the largest share of the gene therapy market, followed by Europe.

Factors such as the rising prevalence of chronic diseases, high healthcare expenditure, presence of advanced healthcare infrastructure, favorable reimbursement scenario, and the presence of major market players in the region are driving market growth in North America.

The primary interviews conducted for this report can be categorized as follows: By Company Type: Tier 1 - 32%, Tier 2 - 44%, and Tier 3 - 24% By Designation: C-level - 30%, D-level - 34%, and Others - 36% By Region: North America - 50%, Europe - 32%, Asia Pacific - 10%, and Rest of the World - 8%

List of companies profiled in the report Amgen, Inc. (US) Biogen (US) Novartis AG (Switzerland) Gilead Sciences, Inc. (US) Spark Therapeutics, Inc. (US) MolMed S.p.A. (Italy) Orchard Therapeutics plc. (UK) SiBiono GeneTech Co., Ltd. (China) Alnylam Pharmaceuticals, Inc. (US) Human Stem Cells Institute (Russia) AnGes, Inc. (Japan) Dynavax Technologies (US) Jazz Pharmaceuticals, Inc. (Ireland) Akcea Therapeutics (US) bluebird bio, Inc. (US) uniQure NV (Netherlands) AGTC (US) Mustang Bio (US) Cellectis (France) Poseida Therapeutics, Inc. (US) Sangamo Therapeutics (US)

Research Coverage:This report provides a detailed picture of the global gene therapy market.It aims at estimating the size and future growth potential of the market across different segments (by vector, indication, delivery method, and region).

The report also includes an in-depth competitive analysis of the key market players, along with their company profiles, recent developments, and key market strategies.

Key Benefits of Buying the Report:This report will help market leaders/new entrants by providing them with the closest approximations of the revenue numbers for the overall gene therapy market and its subsegments.It will also help stakeholders better understand the competitive landscape and gain more insights to position their business better and make suitable go-to-market strategies.

Also, this report will enable stakeholders to understand the pulse of the market and provide them with information on the key market drivers, challenges, and opportunities.

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The global gene therapy market is projected to reach USD 13.0 billion by 2024 from USD 3.8 billion in 2019, at a CAGR of 27.8% - PRNewswire

Taken together, cell and gene therapy could be called Philadelphias signature industry, and soon it may have its signature project.

Courtesy of The Discovery Labs

A rendering of the Center for Breakthrough Medicines, a cell and gene therapy manufacturing facility at The Discovery Labs in King of Prussia, Pa.

On Jan. 23, The Discovery Labs announced it will soon build a $1.1B, 680K SF contract development and manufacturing facility within its 1.6M SF campus in King of Prussia, as part of a joint venture with New York-based Deerfield Management. This Center for Breakthrough Medicines would take up the majority of the 1M SF former GlaxoSmithKline lab and manufacturing complex at 411 Swedeland Road that is part of Discovery Labs.

It will employ 2,000 people and become easily the largest contract development and manufacturing organizationin the world.

Though industry consensus isthat cell and gene therapy needs more manufacturing capability to keep growing and to speed up crucial development timelines, multiple sources told Bisnow that the Discovery Labs plan for CBM might be too ambitious in scale, price and timeline.

An unprecedented price tag for investors

Currently, the title for the largest cell and gene therapy manufacturing facility is held by Lonza, whichopened a 300K SF building in the Houston suburb of Pearland, Texas, in 2018. Lonza says itemploys over 200 full-time staffers at the facility. In the Philadelphia region, the only CDMO of any size is Wuxi AppTecs 287K SFcomplexat the Philadelphia Navy Yard. The new facility in King of Prussia has much larger ambitions.

[CBM] would service the world, Discovery Labs Executive Managing Director Audrey Greenberg said. Whats great about the King of Prussia location and the region, not just from the perspective of talent, is were very close to Europe on the East Coast. So well be able to supply viral vectors and plasmids to much of the U.S., Europe and possibly even Asia Pacific, South America and the Middle East.

Discovery Labs co-founder Brian ONeill, who also owns development firm MLP Ventures, is putting up initial funding for CBM, with some help from Deerfield and additional investors to join in later, Greenberg said. If ONeill needs to raise even half of the $1.1B price tag from outside investors, he may have a hard time finding anybody willing to put up the requisite cash for an operation that wont be developing its own intellectual property, sources said.

Both Greenberg and Jim Daly, a project director for biotech architecture and design firm CRB specializing in cell and gene therapy, believe that the pool of potential investors for the fledgling industry is deep and actively searching for deals.

I am amazed at the amount of capital that is behind so many companies, some of which didnt exist a year ago, Daly said. [But] thats a pretty rich number to try to get folks to step up for.

Courtesy of DIGSAU

A rendering of Iovance Biotherapeutics' lab, office and cell therapy manufacturing facility at the Philadelphia Navy Yard.

Raising funds is the hardest part about bringing a manufacturing facility online, whether a CDMO or a companys own build-to-suit,Gattuso Development Partners President John Gattuso said. Gattuso is developing a136K SF build-to-suit lab and manufacturing facility in the Navy Yard for Iovance Biotherapeutics. Until that is ready, Iovance contracts its production to Wuxi.

Amicus Therapeutics, which recently took space at uCity Square in University City, contracts with Wuxi, Daly said. Spark Therapeutics, the darling of Philadelphias gene therapy scene for getting multiple therapies FDA-approved, contracts out its manufacturing as well.

The University of Pennsylvania and Childrens Hospital of Philadelphia, the academic birthplace of Phillys gene and cell therapy startups, both have their own production needs met on campus, Daly said.

Before a company's therapy gets Food and Drug Administration approval, it relies on venture capital investment and grants to fund its research and development. Having a facility that can service many of them fills a gap in the market, Gattuso said, but a 680K SF facility may be considered too large todepend on non-revenue producers.

If you just think about how to finance a large facility filled with nothing but early, noncredit companies, its hard to imagine," Gattuso said.

Meeting the specific needs of a new, niche industry

Gene and cell therapy have material needs that require specialized manufacturing at every stage, from early research to commercial production for patients. Currently, no single contract facility has the manufacturing capability to meet those needs from bench to bedside, Greenberg said.

To research and develop new gene therapies, labs need plasmids (simple, circular strands of DNA) and viral vectors (benign containers for introducing new genes in a body) for every stage. Once a therapy is developed and can be used in clinical trials and eventually distributed to patients, then a company needs to effectively reproduce the finished product. All of these processes need to be done in highly sterilized clean room environments that are more complex and expensive than even most lab environments.

At full build-out, CBM is set to contain 36 suites for cell therapy processing, 10 for the production of plasmids, 20 for the production of viral vectors, and 20 for testing, process development and cell banking (or the storage of live cells needed for future batches). The suites will have a modular design so they can adapt to a changing client base and future advancements in production.

Courtesy of The Discovery Labs

A partial rendering of The Discovery Labs, MLP Ventures' planned biotech and life sciences coworking community in the Philadelphia suburb of King of Prussia.

Cell and gene therapy are both at such an early stageuntil some of those advancements come to pass, it is prohibitively expensive for a company to scale up to the point where it can fill and afford its own dedicated facility. Gene therapy can be produced in batches that can be only so large before the cell lines break down, and cell therapy is still dependent on autologous treatment, which means that each iteration only can serve one patient.

Those challenges and costs associated with project size are exactly what CBM is hoping to address, but multiple sources agreed that if a company has the resources, it will virtually always prefer to have its own facility to better control both the manufacturing process and its intellectual property.

Once you get to a certain scale, we see a strong desire on the part of certain firms to really control their own environment and be in their own building, Gattuso said. Its not just having the facility available, its about who controls that facility and how the IP is handled inside the facility.

Greenberg estimates there is five times more demand than supply for manufacturing in the gene and cell therapy industries nationwide, and that in three years that could explode to 5,000 times more demand than supply. Multiple sources in the industry agree that there is a substantial bottleneck in procuring the plasmids and viral vectors needed for even the early phases of research, but beyond that it is an unsettled issue.

The uncertain next steps

Though more than 200 "investigational new drug" permits have been issued in the past couple of years by the FDA, those only fast-track the clinical testing of methods for treating diseases and conditions so rare that few, if any, alternatives exist for sufferers.

Since two gene therapies and one cell therapy were fully approved by the FDA in 2017, only three more have gained approval since, Dark Horse Consulting founder Anthony Davies said at the Phacilitate conference in Miami on Jan. 22,according to BioProcess International. The first approved cell therapy, made by Novartis, has been halted by the FDA for manufacturing issues for over a year, Davies said.

Courtesy of The Discovery Labs

An interior rendering of gene and cell therapy hub The Discovery Labs in King of Prussia, Pa.

That frustration doesn't dampen CBM's prospects, as even therapies that will eventually fail need manufacturing capability. But if startups stuck in limbo between their earliest phases and full commercialization are in CBM's sweet spot, then a breakthrough that opens the floodgates could lead to those same companies instead opting for their own space, Daly said.

Part of the reason were seeing the bottleneck is in the supply side, and that would suggest that this is only a temporary phenomenon," Daly said. "As the Iovances of the world start to build out and manufacture their own plasmids and vectors, that demand will wane. That sides not super complicated or difficult; its more about what theyre prioritizing now.

Discovery Labs is in the design phase for CBM as of the end of January, but expects to begin gutting and renovating the former GSK facility within the next three months and bringing its first suites online by the end of this year. Greenberg estimates the first phase to open will amount to 50K to 75K SF of clean room space and about 100K SF for testing and development, which will require about 175 to 200 employees to staff.

Even working with a pre-existing facility that has many of the specialized needs for gene and cell therapy manufacturing, CBM's timeline of less than a year stretches credibility, multiple sources toldBisnow.Meeting the high standards for sterilization and IP protection is an exacting process that requires construction labor more specialized than for the average office building, though Gattuso said Philadelphia is maybe the best market in the country for finding that specific labor.

Staffing the building once operational will be an even taller task; sources estimate that training and getting clean room certification for technicians could take months on its own. The Discovery Labs hopes to address that by opening up its own training school, the Science University of Experiential Learning, within its grounds, but that won't be ready for that first wave of employees CBM will need by year's end.

Because of how much success Penn and CHOP have had in producing talent for gene and cell therapy production, the Philadelphia region makes sense as a place to try building something unprecedented. But despite the area's pride and optimism in its burgeoning industry's growth potential, CBM may be too much, too soon.

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The Biggest Gene And Cell Therapy Manufacturer Is Getting Built In King Of Prussia. Is It Too Ambitious? - Bisnow

A novel base editing technology invented at Rutgers, The State University of New Jersey with the potential to be used for the creation of new cell and gene therapies will be made available to researchers worldwide through an exclusive partnership with the Horizon Discovery Group.

The technology invented by Shengkan Victor Jin, associate professor of pharmacology, and co-inventor Juan C. Collantes, post-doctoral research fellow, at Rutgers Robert Wood Johnson Medical School can be potentially used for developing cell therapies for sickle cell anemia and beta thalassemia, HIV resistant cells for AIDS, off-the-shelf CAR-T cells for cancer, and MHC-compatible allogenic stem cells for transplantation. It could also be used as gene therapies for inherited genetic diseases such as antitrypsin deficiency and Duchenne muscular dystrophy.

The gene editing technology developed by our researchers has the potential to revolutionize how scientists think about their search for better options and outcomes in the treatment of disease worldwide, said S. David Kimball, PhD, Senior Vice President for Research and Economic Development at Rutgers University. Just as important is our ability, through this significant partnership with Horizon Discovery Group, to share our discoveries and inventions with the scientific community around the world who are equally committed to improving human health.

In January 2019, Rutgers University formed an exclusive partnership with Horizon to further the development of the proprietary base editing technology invented by Jin and Collantes. Since the initial partnership, Horizon, a global leader in the application of gene editing and gene modulation technologies, has been funding research in base editing at Jins laboratory. The company has now exercised its option to exclusively license the technology for commercialization of all therapeutic applications. This partnership places Rutgers among the front runners in the field of gene editing.

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, stated Dr. Jonathan Frampton, Corporate Development Partner, Horizon Discovery.

Horizon has a number of internal programs designed to accelerate the clinical uptake of this technology and is now seeking partners to assess and shape the development of its Pin-point base editing platform. The company will offer partners access to a novel system that could be used to advance more effective multi-gene knockout cell therapy programs, with an improved safety profile, through clinical development. 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.

We intend to take full advantage of the unique modular and versatile features of the 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, said Jin.

Base editing is a novel technology for engineering DNA in cells, with 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.

# # #

About Rutgers, The State University of New Jersey

Rutgers, The State University of New Jersey, is a leading national research university and the state of New Jerseys preeminent, comprehensive public institution of higher education. Established in 1766, the university is the eighth oldest higher education institution in the United States. More than 70,000 students and 23,400 full- and part-time faculty and staff learn, work, and serve the public at Rutgers locations across New Jersey and around the world.www.rutgers.edu

As the premier public research university in the state, Rutgers is dedicated to teaching that meets the highest standards of excellence, to conducting cutting-edge research that breaks new ground and aids the states economy, businesses, and industries, and to providing services, solutions, and clinical care that help individuals and the local, national, and global communities where they live.research.rutgers.edu

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Rutgers partners with Horizon Discovery Group | - University Business

Pfizer aims to be the third big pharma with a significant presence in gene therapy. Its plans to initiate this year three Phase 3 trials targeting mutation-driven blood and muscular diseases would make it a large player in this cutting-edge area of medicine.

The difference between Pfizer and its Swiss rivals Novartis and Roche is that its treatments for muscular dystrophy and hemophilia do not look like they will be the first to market. With hopes that gene therapy could be a one-and-done treatment, arriving second could put Pfizer at a disadvantage if eager patients rush for curative therapies.

Having spun of its off-patent drugs business, the pharma is now trying to talk up the "new Pfizer." Its gene therapies are among seven pipeline projects that it cited Tuesday during its year-end earnings call as critical to its strategy of becoming a more innovation-focused company.

Company executives weren't, however, asked to answer how Pfizer views the emerging gene therapy competition. BioMarin Pharmaceutical looks set to get to the market earlier in hemophilia A than Pfizer, while Uniqure in hemophilia B and Sarepta Therapeutics in Duchenne muscular dystrophy appear ahead.

Pfizer's hemophilia A project, the Sangamo Therapeutics-originated SB-525, is up against BioMarin's valrox, which has been submitted to the Food and Drug Administration for an approval decision later this year.

In hemophilia B, fidanacogene elaparvovec, licensed from Roche subsidiary Spark Therapeutics, is in a neck-and-neck race with UniQure's etranacogene dezaparvovec in Phase 3 testing. Duchenne research, meanwhile, is led by Sarepta, which is launching a Phase 3 trial of its drug this year, putting Pfizer's at a disadvantage.

Other than announcing its intent to launch Phase 3 trials in hemophilia A and Duchenne, Pfizer didn't provide much more detail about these clinical programs. Mikael Dolsten, Pfizer's chief scientific officer, said more could be revealed about the DMD program at an upcoming research & development day.

Progress on that project had been delayed after one patient was hospitalized with kidney complications, but Dolsten said trial investigators had dosed additional patients. The Phase 2 will wrap up this spring, and the new data and longer follow-up will help guide a Phase 3 trial design, the company said.

Dolsten also described the hemophilia A project as having a 'best-in-class profile," even though BioMarin's valrox has impressed hematologists with its ability to increase expression of a key blood-clotting protein.

In addition, he said the company hopes it can bring one new gene therapy into its pipeline per year.

Building its drug development portfolio is one reason why the company has chosen not to buy back shares, said CEO Albert Bourla.

He pointed to the company's need in the past to buy back shares to support their valuation because of revenue declines, but now he said the company is in a different strategic position.

"The company is going to have a best-in-class revenue growth story," he said. "We can use the capital to invest in good Phase 2, Phase 3 assets to grow our pipeline."

Original post:
Pfizer lays out gene therapy aspirations - BioPharma Dive

Company signs option agreement with The Rockefeller University to access intellectual property covering compounds targeting key regeneration pathway

Decibel Therapeutics, a development-stage biotechnology company developing novel therapeutics for hearing loss and balance disorders, today announced a new strategic research focus on regenerative medicine approaches for the inner ear. The company is also announcing a collaboration and option agreement that gives Decibel exclusive access to novel compounds targeting proteins in a critical regenerative pathway.

Decibels research focus on regeneration will be powered by the companys research and translation platform. The company has built one of the most sophisticated single cell genomics and bioinformatics platforms in the industry to identify and validate targets. Decibel has also developed unique insights into regulatory pathways and inner ear delivery mechanisms that together enable precise control over gene expression in the inner ear and differentiate its AAV-based gene therapy programs.

"Our deep understanding of the biology of the inner ear and our advanced technological capabilities come together to create a powerful platform for regenerative medicine therapies for hearing and balance disorders," said Laurence Reid, Ph.D., acting CEO of Decibel. "We see an exciting opportunity to leverage this platform to address a broad range of hearing and balance disorders that severely compromise quality of life for hundreds of millions of people around the world."

The first program in Decibels regeneration portfolio aims to restore balance function using an AAV-based gene therapy (DB-201), which utilizes a cell-specific promoter to selectively deliver a regeneration-promoting gene to target cells. In collaboration with Regeneron Pharmaceuticals, Decibel will initially evaluate DB-201 as a treatment for bilateral vestibulopathy, a debilitating condition that significantly impairs balance, mobility, and stability of vision. Ultimately, this program may have applicability in a broad range of age-related balance disorders. There are currently no approved medicines to restore balance. Decibel expects to initiate IND-enabling experiments for this program in the first half of 2020.

Decibel is also pursuing novel targets for the regeneration of critical cells in both the vestibule and cochlea of the inner ear; these targets may be addressable by gene therapy or other therapeutic modalities. As a key component of that program, Decibel today announced an exclusive worldwide option agreement with The Rockefeller University, which has discovered a novel series of small-molecule LATS inhibitors. LATS kinases are a core component of the Hippo signaling pathway, which plays a key role in regulating both tissue regeneration and the proliferation of cells in the inner ear that are crucial to hearing and balance. The agreement gives Decibel an exclusive option to license this series of compounds across all therapeutic areas.

The agreement also establishes a research collaboration between Decibel and A. James Hudspeth, M.D., Ph.D., the F.M. Kirby Professor at The Rockefeller University and the director of the F.M. Kirby Center for Sensory Neuroscience. Dr. Hudspeth is a world-renowned neuroscientist, a member of the National Academy of Sciences and the American Academy of Arts and Sciences, and a Howard Hughes Medical Institute investigator. Dr. Hudspeth has been the recipient of numerous prestigious awards, including the 2018 Kavli Prize in Neuroscience.

"Rockefeller scientists are at the leading edge of discovery, and we are excited to see the work of Dr. Hudspeth move forward in partnership with Decibel," said Jeanne Farrell, Ph.D., associate vice president for technology advancement at The Rockefeller University. "The ambitious pursuit of harnessing the power of regenerative medicine to create a new option for patients with hearing loss could transform how we address this unmet medical need in the future."

In parallel with its new research focus on regenerative strategies, Decibel will continue to advance key priority preclinical and clinical programs. DB-020, the companys clinical-stage candidate designed to prevent hearing damage in people receiving cisplatin chemotherapy, is in an ongoing Phase 1b trial. Decibel will also continue to progress DB-OTO, a gene therapy for the treatment of genetic congenital deafness, which is being developed in partnership with Regeneron Pharmaceuticals. The DB-OTO program aims to restore hearing to people born with profound hearing loss due to a mutation in the otoferlin gene and is expected to progress to clinical trials in 2021.

Story continues

To support the new research focus, Decibel is restructuring its employee base and discontinuing some early-stage discovery programs.

About Decibel Therapeutics, Inc.Decibel Therapeutics, a development-stage biotechnology company, has established the worlds first comprehensive drug discovery, development, and translational research platform for hearing loss and balance disorders. Decibel is advancing a portfolio of discovery-stage programs aimed at restoring hearing and balance function to further our vision of a world in which the benefits and joys of hearing are available to all. Decibels lead therapeutic candidate, DB-020, is being investigated for the prevention of ototoxicity associated with cisplatin chemotherapy. For more information about Decibel Therapeutics, please visit decibeltx.com or follow @DecibelTx.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200129005162/en/

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Matthew Corcoran, Ten Bridge Communicationsmcorcoran@tenbridgecommunications.com (617) 866-7350

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Decibel Therapeutics Announces Strategic Research Focus on Regenerative Medicine for the Inner Ear - Yahoo Finance

In a second research report published this year so far, investigators found that the U.S. Food and Drug Administration (FDA) is approving drugs faster than ever. Unfortunately, it appears that the agency is also approving those drugs on less data and weaker evidence.

The first studypublishedin the journal SSRN was by researchers at Harvard University, the University of Texas at Dallas, and the Massachusetts Institute of Technology (MIT).It questioned if the FDA and other regulatory agencies worldwide dont rush certain approvals, particularly at the end of the year in a kind of desk-clearing activity.

The report notes, In the United States, the number of December drug approvals is roughly 80% larger than in any other month. Similar approval spikes occur at the end of each calendar month. Additionally, approvals spike before holidays, such as before Thanksgiving in the United States and the Chinese New Year in China (but not vice versa).

And more troubling is that there appears to be a correlation with more problems with these drugs. Lauren Cohen, a professor of finance and entrepreneurial management at Harvard Business School and one of the authors, told the Wall Street Journal, We see about twice as many adverse effects.

The second study appeared in the journal JAMA Network and was conducted by researchers with Harvard Medical School. The lead author, Jonathan Darrow, a lawyer with the medical schools Program on Regulation, Therapeutics and Law, told NPR, There has been a gradual erosion of the evidence thats required for FDA approval. He points out that patients and physicians should not expect that new drugs will be dramatically better than older ones.

The study notes that about half of recent drug approvals were built on a single pivotal clinical trial. Typically, two pivotal, Phase III trials were the norm. In addition, the study says that surrogate measures, which are utilized as stand-ins for presumed patient benefits, has grown. For example, in oncology drugs, what most patients would want are improvements in survival after receiving treatment. But some cancer trials use a surrogate measure of tumor shrinkage. Ideally, both would be taken into consideration.

Darrow and his research associates studied FDA approvals, changes in the law and regulations, and how the industry funds agency reviews from 1983 through 2018. They found that the average number of new drug approvals annually grew from 34 in the 1990s to 41 in the 2010s. In the 2000s, it dropped to 25 a year. But now they are increasing. For example, in 2019, the FDA approved 48 new molecular entities and new therapeutic biological products. That doesnt include vaccines, allergenic products, blood and blood products, plasma derivatives, cellular and gene therapy products, or the numerous new indications approved for existing therapies.

Darrow, with Jerry Avorn and Aaron S. Kesselheim, both with the Division of Pharmacoepidemiology & Pharmacoeconomics at Brigham & Womens Hospital, found that faster approvals were related to legislative and regulatory modifications that started in the 1980s. Although there are probably several reasons for those changes beginning in that period, much of it is likely related to the beginning of the HIV epidemic and demands from patient populations and advocates to fund more research and get therapiesany therapiesto market faster.

Just some of those regulations include: the 21st Century Cures Act (2016), which authorized funds for the Precision Medicine Initiative and Cancer Moonshot; the Biologics Price Competition and Innovation Act (BPCIA, 2010), creating an abbreviated pathway for follow-on biologic products; Breakthrough Therapy designation (2012), for drugs that showed substantial improvement over existing therapies; the Hatch-Waxman Act (1984), which created an Abbreviated New Drug Application pathway for drugs approved after 1962; and the Pediatric Research Equity Act (2003), which required results from pediatric assessments to be submitted as part of New Drug Applications (NDAs).

Congress also passed the Prescription Drug User Fee Act in 1993, and that first year, FDA collected $29 million in fees. In 2018, the agency brought in $908 million in PDUFA fees. Or, as the study notes, industry fees were responsible for about 80% of the money spent on FDA employee salaries for drug reviews.

There is some concern about the incentives that this created within the FDA, Darrow told NPR. And whether it has created a culture in the FDA where the primary client is no longer viewed as the patient, but as the industry.

Another factor that is related, is the concept of me-too drugs. These are basically drugs that are very structurally similar to approved drugs, with only minor differences. Thats not necessarily a bad thing, because they need to be at least as good as the drugs already on the market, and generally need to be betteralthough not necessarily by much. Which does mean a number of companies spend time on developing drugs that are only incrementally better than others on the market.

Joshua Sharfstein, former FDA Principal Deputy Commissioner, told NPR that there are more changes needed to ensure drugs are worthwhile for patients. Some of them are really great, and some of them [are] not so great. And a lot of them are very expensive.

Sharfstein is currently a professor at the Johns Hopkins Bloomberg School of Public Health. He wrote an editorial in JAMA that accompanied the newer study. In it, he suggests its time to reevaluate the FDAs expedited approval programs to determine which ones are working and which ones are increasing healthcare costs.

Weve kind of reached a point where it makes sense to pause and see whether we can do things better, Sharfstein said. And I think we can.

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The FDA Is Approving Drugs Faster, But That May Not Be A Good Thing - PharmaLive

Innovative treatments have turned the tables somewhat in the battle against cancer but one stumbling block appears to be pricing. CAR-T cell therapy can cost more than $1.5 million including the $475,000 cost of the drug (in this case, Kymriah from Novartis).

We are seeing that price is becoming a barrier because if you can imagine, hundreds of thousands of people all suddenly becoming eligible for CAR-T, there isnt enough money in the healthcare system to pay for that, said Paul Wotton, CEO of Obsidian Therapeutics, based in Cambridge, Massachusetts.

In a recent interview in San Francisco, Wotton and another biopharma executive pointed to a solution to the price conundrum: an off-the-shelf approach.

To clarify, to date the approved CAR-T therapies involve cells from the patients own body that are re-engineered and placed back into the body in order to overpower and kill the cancerous growth. While revolutionary, this is not only a cumbrous process but also expensive.

Now, some companies are going the route of using donor cells to develop their own treatment regimens. These are known as allogeneic therapies.

Wugen Therapeutics is one such company that is taking an allogeneic approach to CAR-T therapy. Based in St. Louis Missouri with technology developed out of Washington University, the company targets T-cell malignancies in leukemia and lymphoma, said Ayman Kabakibi, vice president of research and development. In a recent interview, he explained that so far CAR-T therapies have focused on B-cell malignancies because it is less difficult to target B-cell malignancies with a T-cell.

The reason is that if you go back to the basics of CAR-T you are trying to put a receptor (CAR) on the normal T-cell to allow it to recognize another antigen or marker on the tumor cell and when that interaction happens, two things happen the T-cell will proliferate and make more daughter cells and thats why we call it a living drug, Kabakibi explained. And the other thing that happens is that it secretes toxins that kill the tumor cells. Well, you can do that more easily for B cells because the antigens are not shared between the two.

But at Wugen, the company is using re-engineeerd normal T-cells to target cancerous T-cells and certain adjustments need to be made so that the cells dont end up killing each other.

So what we do is that we take the T-cells and we remove the antigen that we are targeting on the cancer cell using CRISPR CAS technology, he said. We take T-cells from a normal person. We eliminate that marker and then we put another gene to make it stress the receptor that it will recognize.

Why not take T-cells from the patients own body?

One reason is that in many cases by the time patients land on CAR-T therapy, they may have already undergone several rounds of other treatments that may have compromised the health of a normal T-cell.

Because the patient has had so many chemotherapies the chances of getting a healthy T-cell are actually pretty low, he said. There are 20-30 percent of patients that go for CAR-T therapy and they get into the clinic and get their blood taken out and then it takes six weeks to generate autologous CAR-T therapy but they cant do it because theres not enough starting material, not enough healthy cells.

This approach of using donor T-cells gets around this problem but Kabakibi believes that it also solves the problem of the skyrocketing cost of CAR-T therapies.

You dont get the economies of scale [with CAR-T using autologous approach,] he said. Thats why you have to be able to generate hundreds of doses and not from the patient, but from a normal patient. You need an off-the-shelf, allogenic version. You take the cells from a normal person and generate a hundred vials that could be shipped overnight to a patient and then that person can be treated in a few days.

Wotton, the CEO of Obsidian Therapeutics, echoed Kabakibi.

You have to find a way to drive down that cost of goods. The way science is going to address that I think is to have off-the-shelf approaches where you can grow billions of cells from a single cell, engineer it to be able to get it to all of us to treat whatever it is we are suffering from, Wotton said. It is much better to build that scale of manufacturing rather than on a patient by patient basis.

Obsidian Therapeutics has developed what it calls destabilizing domain technology to be able to more precisely dose cell and gene therapies.

We are pioneering controllable cell and gene therapies using technology that came out of Stanford University, Wotton said. So in a nutshell, if you give somebody gene therapy today, to produce a particular protein in the body you actually have no idea how much of that protein is going to express. And the same in the case of a CAR-T cell to patients, you actually dont know how many of those cells are going to survive, how quickly they are going to proliferate so being able to dose cell and gene therapies in the same way you can dose a small molecule would be extremely useful.

Obsidianhas a partnership with Celgene through which Celgene can in-license global rights to cell therapy product candidates developed by Obsidian that incorporate destabilizing domain-regulated interleukin 12 or CD40L to fight cancer. Obdisial is using an allogeneic approach.

Our goal in life is to turn cell therapy into the first line of treatment instead of the last line of treatment, Wotton said. The only way you are going to be able to do that is to provide a cell therapeutic that is off-the-shelf, readily available and easy for patients to access, which is actually the approach that we are trying to take.

However, he added that there has to be a balance between cost and value when it comes to novel treatment.

Kabakibi of Wugen Therapeutics agreed.

If you are generating a 100 vials of CAR-T cells, is the price going to drop by 100 percent? Unlikely, he said. I think some therapies will never be as inexpensive as a pill.

Photo: champc, Getty Images

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Makers of novel cell/gene therapy say off-the-shelf approach only way to solve pricing woes - MedCity News

Stroke is the third leading cause of death and disability in the United States. More than 87 percent are ischemic strokes, caused by obstruction of one or more cerebral arteries. With limited progress in developing treatments, there is a critical need for neuroprotective agents to effectively treat stroke.A study from Florida Atlantic University's Schmidt College of Medicine holds promise for a new way to treat stroke using an already FDA-approved drug granulocyte colony-stimulating factor (GCSF). GCSF enhances blood cellular development and is currently used to treat neutropenia (low white blood cells) caused by chemotherapy and has successfully been used with very few side effects for patients who require bone marrow transplants to stimulate blood cell formation.

The study is the first to report on the neuroprotective effect of GCSF against autophagy and mitochondrial stress in vivo. The data support the hypothesis that GCSF is one of the few growth factors that can reduce infarction by decreasing endoplasmic reticulum (ER) and mitochondrial stress while improving behavioral performance.

Results showed that GCSF improved neurological deficits that occur in the first few days following cerebral ischemia and improved long-term behavioral outcomes while also stimulating a neural progenitor recovery response. Researchers tested behavioral performance on corner and locomotor tests, used as an indicator of brain injury.

Using a mouse model, researchers investigated the efficacy of GCSF beyond the typical four-hour thrombolytic therapy (tPA) clot-busting drug the gold standard to treat stroke for global ischemia. They examined the pro-survival mechanisms of GCSF against apoptosis resulting from autophagy, mitochondrial stress and ER stress.

"In recent years, many studies including ours have shown that as an endogenous growth factor and immune system modulator factor, GCSF is beneficial in models of neurological disorders such as stroke and traumatic brain injury," said Jang-Yen (John) Wu, Ph.D., corresponding author, distinguished professor of biomedical science in FAU's Schmidt College of Medicine, and a member of the FAU Brain Institute (I-BRAIN). "Although the anti-apoptotic activity of GCSF is reported in global cerebral ischemia, this mechanism has not been fully explored."

Researchers used a mechanism-based therapeutic approach for stroke first to examine the connection of mitochondrial, autophagy and ER stress inhibition in the protective action of GCSF and then to analyze relevant ER stress pathways in the bilateral common carotid artery occlusion (BCAO) model of stroke. They confirmed the neuroprotection of GCSF gene therapy in the BCAO mouse stroke model by a decrease of dynamin-related protein (DRP1), a marker of mitochondrial stress, in the frontal and middle brain of the GCSF treated group.

The initial dose of GCSF was administered 24 hours post-BCAO and then followed by a single application of the same dose for another three days for a total of four days of administration. Researchers examined behavior and used immunoblotting to analyze key proteins in ER stress, autophagy and mitochondrial stress-induced apoptosis. BCAO mice receiving GCSF protein showed significantly less asymmetric turning in the corner test than BCAO mice without GCSF. In the behavioral assays, GCSF elicited increased locomotor sensitization verified by greater activity in the locomotor activity test, demonstrating the neuroprotective properties of the drug.

"More than 15 million people worldwide suffer from stroke and our study provides new and important insights into GCSF induced protection as it relates to ER stress and mitochondrial stress activated apoptosis, " said Howard Prentice, Ph.D., corresponding author, a professor of biomedical sciences in FAU's Schmidt College of Medicine, and a member of FAU's I-BRAIN. "Future research will need to focus on uncovering the complete mechanisms by which GCSF retains the ER and mitochondrial homeostasis."

Wu and Prentice have been developing GCSF as a therapeutic method to replenish new brain cells because of its ability to preserve the central nervous system, suppress cell death and at the same time elicit neurogenesis as well as angiogenesis. GCSF works the same way for other neurological diseases such as Parkinson's disease due to its neuroprotective properties.ReferenceModi et al. (2020) Mode of action of granulocyte-colony stimulating factor (G-CSF) as a novel therapy for stroke in a mouse model. Journal of Biomedical Science. DOI: https://doi.org/10.1186/s12929-019-0597-7

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Repurposed Drug Proves Neuroprotective in Stroke Model - Technology Networks

A new study by University of Massachusetts scientists has described the molecular process in which synaptic connections in the brain are compromised in multiple sclerosis.

The research, which was published in Immunity, investigates how gene therapy could be used to preserve neural circuits and prevent sight loss.

In experiments in mice, researchers inhibited a protein commonly found in the synapses of patients with multiple sclerosis.

Dr Sebastian Werneberg, from the University of Massachusetts Medical School, highlighted that the protein C3 prompts microglia to attack synapses.

A gene therapy approach was used to deliver a natural inhibitor of C3 to synapses in the visual system while leaving the rest of the brain untouched.

As a result of this inhibition, we saw improved visual function in mice, Dr Werneberg said.

Researchers will next explore how the C3 protein is being produced in multiple sclerosis and other neurodegenerative diseases.

Image credit: Pixabay/PublicDomainPictures

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Protecting against vision loss in multiple sclerosis OT - AOP

SAN DIEGO, Jan. 28, 2020 (GLOBE NEWSWIRE) -- Otonomy, Inc.(OTIC), a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, today announced preclinical results from the companys gene therapy collaboration with Applied Genetic Technologies Corporation (AGTC) focused initially on treating GJB2 deficiency for congenital hearing loss, and preclinical results demonstrating the therapeutic potential of a class of compounds being evaluated for otoprotection against cisplatin-induced hearing loss (CIHL). These results were presented during the ongoing Association for Research in Otolaryngology (ARO) 43rd Annual MidWinter Meeting being held in San Jose, California.

Together with our strategic partner, AGTC, we are encouraged by these initial preclinical results that demonstrate our ability to express a gene of interest in the target cells relevant to the treatment of congenital hearing loss due to GJB2 deficiency," said David A. Weber, Ph.D., president and chief executive officer of Otonomy. Also, the preclinical results presented for our OTO-510 program highlight the therapeutic potential of a novel class of cisplatin-binding molecules for protection against CIHL and the higher potency of these agents versus other molecules currently in clinical development.

Preclinical Results for GJB2 Gene Therapy Collaboration

In October 2019, Otonomy and AGTC announced a strategic collaboration to co-develop and co-commercialize an AAV-based gene therapy to restore hearing in patients with sensorineural hearing loss caused by a mutation in the gap junction protein beta 2 gene (GJB2) -- the most common cause of congenital hearing loss. The joint presentation by Otonomy and AGTC at ARO provided initial demonstration that a gene of interest can be expressed in support cells of the cochlea, which are the relevant target cells for treating GJB2 deficiency, using novel and proprietary AAV capsids. Furthermore, these studies identified several capsids with favorable tropism and gene expression level in support cells compared to previously reported capsids used in the field. Importantly, none of the novel AAV capsids evaluated for further development exhibited signs of cellular toxicity.

Preclinical Results for OTO-510 Otoprotection Program

Cisplatin is a potent chemotherapeutic agent that is widely used to treat a variety of cancers in adults and children. Unfortunately, the administration of cisplatin is commonly associated with severe adverse effects including CIHL that is progressive, bilateral and irreversible. At ARO, Otonomy presented preclinical results demonstrating varying degrees of otoprotection against CIHL for several different classes of therapeutic agents. In particular, a novel proprietary class of agents that potently bind to cisplatin demonstrated greater otoprotection than anti-oxidant and anti-apoptotic molecules, and increased potency relative to other cisplatin-binding molecules currently in clinical development. These results highlight the therapeutic potential of Otonomys novel otoprotectant agents as the basis for the OTO-510 program for CIHL.

About Otonomy

Otonomy is a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology. The company pioneered the application of drug delivery technology to the ear in order to develop products that achieve sustained drug exposure from a single local administration. This approach is covered by a broad patent estate and is being utilized to develop a pipeline of products addressing important unmet medical needs including Mnires disease, hearing loss, and tinnitus. For additional information please visit http://www.otonomy.com.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements generally relate to future events or the future financial or operating performance of Otonomy. Forward-looking statements in this press release include, but are not limited to expectations regarding the potential benefits, development activity and advancement of preclinical programs; the potential benefits of and activity under the collaboration agreement between AGTC and Otonomy, including but not limited to development activity; and statements by Otonomys president and CEO. Otonomys expectations regarding these matters may not materialize, and actual results in future periods are subject to risks and uncertainties. Actual results may differ materially from those indicated by these forward-looking statements as a result of these risks and uncertainties, including but not limited to: Otonomys limited operating history and its expectation that it will incur significant losses for the foreseeable future; Otonomys ability to accurately forecast financial results; Otonomys ability to obtain additional financing; Otonomys dependence on the regulatory success and advancement of its product candidates; the uncertainties inherent in the clinical drug development process, including, without limitation, Otonomys ability to adequately demonstrate the safety and efficacy of its product candidates, the nonclinical and clinical results for its product candidates, which may not support further development, and challenges related to patient enrollment in clinical trials; Otonomys ability to obtain regulatory approval for its product candidates; the risks of the occurrence of any event, change or other circumstance that could give rise to the termination of the collaboration agreement between AGTC and Otonomy; the risks of the occurrence of any event, change or other circumstance that could impact Otonomys ability to repay or comply with the terms of the loan provided by Oxford Finance LLC; side effects or adverse events associated with Otonomys product candidates; Otonomys ability to successfully commercialize its product candidates, if approved; competition in the biopharmaceutical industry; Otonomys dependence on third parties to conduct nonclinical studies and clinical trials; Otonomys dependence on third parties for the manufacture of its product candidates; Otonomys dependence on a small number of suppliers for raw materials; Otonomys ability to protect its intellectual property related to its product candidates in the United States and throughout the world; expectations regarding potential therapy benefits, market size, opportunity and growth; Otonomys ability to manage operating expenses; implementation of Otonomys business model and strategic plans for its business, products and technology; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled "Risk Factors" in Otonomys Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (the "SEC") on November 5, 2019, and Otonomys future reports to be filed with the SEC. The forward-looking statements in this press release are based on information available to Otonomy as of the date hereof. Otonomy disclaims any obligation to update any forward-looking statements, except as required by law.

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Otonomy Presents Preclinical Results for GJB2 Gene Therapy Collaboration and Cisplatin Otoprotection Program - Yahoo Finance

Passage Bio Jim Wilsons self-described legacy company has wooed a seasoned biotech executive to steer the clinical entry of its first three gene therapy programs.

Bruce Goldsmith jumps to the helm of Passage after a brief CEO stint at Civetta, a cancer-focused startup he helped launch while a venture partner at Deerfield. He takes over from OrbiMed partner and interim chief Stephen Squinto, who will now lead the R&D team.

He joins as the biotech preps IND filings for three lead programs in rare, monogenic diseases of the central nervous system in 2020 the lysosomal storage disorders GM1 gangliasidosis and Krabbe disease, as well as frontotemporal dementia.

Bruce is ideally suited to lead Passage Bio as chief executive officer given his strong neuroscience background coupled with his robust healthcare and biotechnology industry experience, board chairman Tachi Yamada said in a statement.

Passage launched last February with $115 million from a marquee group of Series A investors including Frazier (where Yamada is a partner), OrbiMed, Versant Ventures, New Leaf Venture Partners, Vivo Capital and Lilly Asia Ventures. With an office just a 10-minute walk away from Wilsons lab at the University of Pennsylvania, the company was designed to apply the gene therapy pioneers 35-year experience into cross-correctional therapies for CNS.

According to what he calls the Jim Wilson 90/10 rule, Squinto previously told Endpoints News, AAV vectors can cover and transduce 90% of motor neuron cells but only 10% to 15% of other brain cells making it difficult to go after indications where broad transduction is needed. But it can still prove useful in disorders that result from mutations in enzymes that can be taken up by neighboring cells once secreted normally.

A close pact with Penns Gene Therapy Program and Orphan Disease Center gave Passage rights to five programs right out of the gate, with options to license seven more.

Its a very aggressive clinical development strategy across a multitude of programs, Squinto said as he closed a $110 million Series B in September. Were not gonna rely on any one program to drive the value of Passage, were gonna rely on what is a very very full pipeline of opportunities.

Goldsmith will now lead a team of about 25 to build on preclinical and IND-enabling data from Wilsons lab a company growing exercise he honed as COO of Lycera. There, he was also credited for a number of business development initiatives.

The transition into the clinic would also mean moving production from early facilities at Penn to Paragons GMP sites, and eventually to a customized suite slated for completion in the third quarter of this year.

Squinto, a rare disease expert who devoted much of his career to Alexion, will continue to help oversee all of that as a board director.

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Eyeing a trio of trial initiations, Jim Wilson's gene therapy startup woos Bruce Goldsmith from Deerfield as CEO - Endpoints News

BALTIMORE, Jan. 29, 2020 /PRNewswire/ --Green Park Collaborative (GPC) is partnering with the National Hemophilia Foundation (NHF) to develop a publicly available, patient-reported outcome measure that measures impact on mental health outlook associated with receiving gene therapy for hemophilia A and B.

This project follows work completed in coreHEM, where a multi-stakeholder group achieved consensus on a core outcome set for gene therapy in hemophilia. In coreHEM, a mental health outcome was included, and the proposed definition included elements of psychological and mental health status, and emotional functioning due to the transformational change of a potentially one-time treatment. Because gene therapy will serve as a durable treatment for hemophilia, changes in mental and emotional health are expected. No current measures exist to effectively evaluate this outcome.

"Developing a new instrument to measure the transformative potential of gene therapy on an individual's overall mental health, whether positive or negative, will further enable our understanding of the entire spectrum of functional and social impact of living with hemophilia," stated Mark Skinner, co-investigator of the coreHEM study.

"Mental health in the bleeding disorders community has always been vital to the individuals and families we serve," said Dawn Rotellini, Interim CEO at NHF. "As we move towards enhanced and innovative treatments in hemophilia it will become imperative to measure how the community responds and how NHF can support them. We are excited to continue our partnership with the Green Park Collaborative to better serve the bleeding disorders community."

About Green Park Collaborative (GPC)GPC is a major initiative of CMTP, an independent 501(c)(3) non-profit organization dedicated to improving the quality, relevance, and efficiency of clinical research. GPC is a multi-stakeholder forum for developing condition- and technology-specific study design recommendations to guide the creation of evidence needed to inform both clinical and payment decisions.

About the National Hemophilia Foundation (NHF)The National Hemophilia Foundation (NHF) is a 501(c)(3) non-profit organization dedicated to finding better treatments and cures for inheritable bleeding disorders and to preventing the complications of these disorders through education, advocacy and research. NHF's programs and initiatives are made possible through the generosity of individuals, corporations and foundations, as well as through a cooperative agreement with the Centers for Disease Control and Prevention (CDC).

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Green Park Collaborative and the National Hemophilia Foundation launch project to develop a PROM to measure mental health outlook - P&T Community

The Cell and Gene Therapy Catapult and the University of Hertfordshire have launched a new course specifically addressing the foreseeable skills gap in the production of cell and gene therapies as they progress towards manufacturing at scale.

Developed in collaboration between the two organisations, this three-day course will provide theoretical and practical training on the aseptic manufacturing of cell and gene therapies in line with European regulatory guidance for good manufacturing practice.

The cell and gene therapy industry in the UK currently supports over 3,000 jobs, a six-fold increase since 2012, and employment in the sector is set to more than double by 2024 as more therapies progress towards commercialisation.

Manufacturing and bioprocessing roles in particular have tripled in the past two years alone, with scientists operating in the 26 cell and gene therapy manufacturing facilities throughout the UK.

Cell and gene therapies are transformative and potentially curative medicines and it is vital that manufacturing processes are safe and efficient whilst preserving the effectiveness of these living medicines.

The new training programme is designed for staff working in cell and gene therapy manufacturing. Delegates will benefit from experience in state-of-the-art facilities and will be awarded a University of Hertfordshire accredited certificate upon successful completion of the training, contingent on assessments of their knowledge, understanding and practical skills.

Dr Salman Rahman, commercial lead in the School of Life & Medical Sciences at the University of Hertfordshire, said: Students on the course will be taught theoretical knowledge including principles and operational aspects necessary for aseptic manufacturing of cell and gene therapy products in line with European regulatory guidance, complimented by practical learning experiences in state-of-the-art clean room facilities.

Keith Thompson, CEO of the Cell and Gene Therapy Catapult added: There is an accelerating demand for skills in the UK cell and gene therapy industry as employment, manufacturing space and the number of ongoing clinical trials increases year on year.

We are delighted to collaborate with the University of Hertfordshire in developing this new initiative which will support professionals andBy providing training on GMP quality standards which are vital to the manufacture of cell and gene therapies, we are furthering our mission to fuel the continuing growth of the industry.

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Boost for cell and gene therapy manufacture - Business Weekly

Horizon Discovery is to 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.

The technology is incorporated into Horizons next-generation gene editing platform. It will 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).

The platform will also expand the companys research tools and service provisions.

Horizon 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 to 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 at Horizon Discovery, said: The technology could have a significant impact in enabling cell therapies to be progressed through clinical trials and towards commercialisation.

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 organisations will help us to drive innovation and deliver the best therapy for the patient.

Dr Shengkan Victor Jin of Rutgers University added: The cytidine deaminase version of the technology alone could potentially be used for developing cell therapies such as gene modified cells for sickle cell anaemia 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.

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Horizon and New Jersey university power up gene therapy technology - Business Weekly