Search Immortality Topics:

Page 11234..1020..»

Category Archives: Genetic Therapy

The Center for Breakthrough Medicines is Building the World’s Largest Cell and Gene Therapy Contract Development and Manufacturing Organization (CDMO)…

The Center for Breakthrough Medicines has Signed a Long-Term Lease for 680,000 square feet at The Discovery Labs King of Prussia and Plans to Hire 2,000 PhD Scientists, Manufacturing Experts, Lab Technicians and Support Staff

The New Facility Will Relieve the Cell and Gene Therapy Industry's Production Constraints, Providing Patients Better Access to Treatments

The Discovery Labs Will be Home to THE COLONY Creating an International Magnet for Scientists to Co-create with Members of The Philadelphia Life Sciences Community

KING OF PRUSSIA, Pa., Jan. 22, 2020 /PRNewswire/--The Discovery Labs and Deerfield Management Company have formed The Center for Breakthrough Medicines, a Contract Development and Manufacturing Organization (CDMO) and specialty investment company, to alleviate the critical lack of capacity that is preventing patients from accessing critically needed cell and gene therapies. The CDMO is occupying over 40 percent of The Discovery Labs' 1.6 million square foot biotech, healthcare and life sciences campus in King of Prussia, PA.

(PRNewsfoto/The Discovery Labs)

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 immense $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 targeting the best and brightest of the life sciences community including, experts in CGMP manufacturing. The Company expects to hire over 2,000 team members within the next 30 months.

The CDMO has retained Nucleus Careers, a cloud-based specialty life sciences human capital recruiting and retention management expert, to buildout the entire team. Nucleus has proprietary recruiting and retention software designed for large scale human capital buildouts of high growth companies.

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, Ph.D., Founder of Paragon Bioservices and Chairman of The Discovery Labs states, "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." Dr. Chacn went on to say, "the goal of THE COLONY is to unshackle the potential of the world's greatest scientific minds."

The ability for the industry's greatest scientists to cohabitate, collaborate, cooperate, and communicate via technology and in person will create an exponential therapeutic "X FACTOR." THE COLONY seeks to unlock institutional barriers prohibiting the world's greatest scientists from moving at a pace necessary in today's ever-changing therapeutic revolution. THE COLONY will partner with the institutions where the scientists currently work by providing equity, license fees, and revenue sharing.

Story continues

"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," noted Audrey Greenberg, Board Member and Executive Managing Director for The Discovery Labs.

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 of 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

The Emerging Field of Cell and Gene Therapy in Pennsylvania

The demand for clinical and commercial manufacturing capacity is acute and expected to remain that way. The current shortfall in manufacturing for cell and gene therapies is severely underserved with few approved products. There are currently approximately 1,100 advanced therapies in the pipeline pending FDA approval. This will greatly increase highly skilled manufacturing demand. Dr. Peter Marks, Director of the FDA Center for Biologics Evaluation and Research, states, "what keeps me up at night is will we be able to manufacture these on a scale that will allow us to bring the benefit of these therapies to patients?"He further added that "if we can help see cost of goods and ability to manufacture reproducibly improve, I think that'll be a big thing."All of this adds up to a supply constrained market that The Center for Breakthrough Medicines aims to help address.

With the potential to treat and even cure disabling, and deadly diseases, gene and cell therapies are ushering in a new era of medicine. These therapies may eventually be able to cure genetic conditions, such as cystic fibrosis, hemophilia A, and a range of cancers. The Philadelphia area has become the epicenter for the flourishing field of gene and cell therapy. Research from CBRE currently ranks the market among the top biotech clusters for medical research and health services. The cluster has become known worldwide as "Cellicon Valley"for its leadership in research and development of this rapidly evolving field. The Discovery Lab's suburban Philadelphia location offers a talent rich environment due to the area's preponderance of large pharmaceutical companies and the Philadelphia region's position boasting the top 10 universities and primary school systems in nation.

Over the past three years, multiple Philadelphia companies have received approvals for major breakthroughs in cell and gene therapy. In 2017, the U.S. FDA approved the first-ever CAR-T cell therapy, Novartis's Kymriah, which originated at the University of Pennsylvania. Shortly thereafter, the FDA gave landmark approval for the first-ever gene therapy to treat a genetic blindness condition to Spark Therapeutics, a start-up founded by researchers at Children's Hospital of Philadelphia. These discoveries and others in the pipeline are attracting billions of dollars of venture capital. The Greater Philadelphia Region set a recent record in venture capital financing.

The Discovery Labs Center for Breakthrough Medicines joins more than 25 healthcare, life sciences and tech-enabled companies that already call The Discovery Labs King of Prussia home.

Brian O'Neill, Founder of The Discovery Labs Center for Breakthrough Medicines, and Tony Khoury, Board Member of The Discovery Labs and Engineer at Project Pharma, will be speaking at the 2020 PhacilitateWorld Stem Cell Summit discussing The Future of Gene Therapy Manufacturing at 4 p.m. today at the Hyatt Regency in Miami, Florida.

Contact Audrey Greenberg at for more information about development services, manufacturing capacity, incubator space or leasing information at the property.

About The Discovery LabsPart of MLP Ventures, The Discovery Labs is a global provider of world-class cGMP manufacturing, turnkey laboratory solutions, critical materials and office space that support therapeutic products and services to the biotechnology and pharmaceutical industry so that groundbreaking medicines get to the patients that need them. The location in eastern King of Prussia is a prototype for a global rollout of The Discovery Labs, providing Big Pharma, emerging life sciences, consumer and technology companies flexible, end-to-end technical real estate and business infrastructure for the customer's entire lifecycle from discovery to delivery, including manufacturing capacity. It is the first fully integrated environment that merges technology and life sciences under one roof to drive innovation.

About Deerfield Management

Deerfield is a healthcare investment management firm committed to advancing healthcare through investment, information and philanthropy.

Media Contact:Tony DeFazio, DeFazio Communications(o) 484-534-3306 (c)

View original content to download multimedia:

SOURCE The Discovery Labs

View original post here:
The Center for Breakthrough Medicines is Building the World's Largest Cell and Gene Therapy Contract Development and Manufacturing Organization (CDMO)...

Posted in Genetic Therapy | Comments Off on The Center for Breakthrough Medicines is Building the World’s Largest Cell and Gene Therapy Contract Development and Manufacturing Organization (CDMO)…

Gene Therapy Recovers Vision in Mice Models of MS, Uncovers How… – Multiple Sclerosis News Today

Early research in animal models and human samples reveals how loss of communication between nerve cells contributes to the symptoms of multiple sclerosis (MS), and shows how gene therapy could be used to preserve such connections and protect againstvision loss.

Researchers say their work identifies a new approach for developing MS therapies that target nerve cell communication, rather than myelin loss, and could be applicable to other neurodegenerative disorders.

The study, Targeted Complement Inhibition at Synapses Prevents Microglial Synaptic Engulfment and Synapse Loss in Demyelinating Disease, was published in the journal Immunity.

MS is a neurological disease marked by inflammation and a self-attack of the immune system against a persons brain, spinal cord, and optic nerves.

This attack damages the protective fatty substance covering nerve fibers (axons), which are necessary for proper transmission of nerve signals called myelin. As the myelin sheath is lost (demyelination), the communication between nerve cells is damaged or even interrupted, and nerve cell death occurs, leading to a range of disease symptoms.

Some MS patients experience a version of the disease called progressive MS, in which symptoms continuously worsen over time while their central nervous system (brain and spinal cord) shrinks (atrophies), and the junctions at which nerve cell terminals meet to communicate with each other, called synapses, are lost.

The majority of MS medications work to inhibit the self-attacking immune responses and inflammatory demyelination, but the neurodegenerative aspects of the disease have been more difficult to stop, particularly for patients with progressive MS.

Most MS research and FDA-approved treatments focus on demyelination and axon death, Dorothy P. Schafer, PhD, professor at the University of Massachusetts Medical School, said in a press release.

Far less is known about what happens to the synaptic connections between neurons, which has proven to be a key aspect of neurodegeneration likely leading to cognitive decline in other diseases such as Alzheimers disease, Schafer said.

Using tissue samples from deceased MS patients, a primate model of MS, and mice models of demyelinating disease, Schafer and colleagues investigated how synapses change during MS.They specifically looked at synapses involved in transmitting visual information from the eye to the brain via the optic nerve.

According to the studys first author, Sebastian Werneburg, PhD, a postdoctoral researcher at Schafers lab, the visual system is an ideal model for investigating MS because its easy to access for therapeutic intervention, subtle changes can be readily detected, and the visional pathway is affected in almost half of all patients with the disease.

Most MS patients experience vision problems at some point, which result from damage to the optic nerve or from lack of coordination in the eye muscle. These problems can be the first indication of the disease.

Similar to other neurodegenerative diseases, researchers found a profound synaptic loss in patient samples as a consequence of immune cells called microgliaeating nerve cell connections.

Microglia are cells that serve as one of the first and main forms of immune defense in the central nervous system, acting to clear cellular debris and dead neurons via phagocytosis a process by which some cells engulf other cells or particles.

In mice, synapse loss occurred independently of local demyelination and neuronal degeneration, but coincided with a rise in a specific immune factor called C3. C3 is part of the complement system, and is normally not present in the brains of adults. It is produced and activated during demyelinating diseases, but it is not clear why.

As C3 was seen to bind to synapses in models of MS, researchers reasoned this complement protein might be involved with the ongoing destruction of synapses in mice with MS-like disease.

To test this hypothesis, they specifically neutralized C3 at synapses of the visual pathway using gene therapy in mice. The strategy basically worked by delivering genetic material to synapses that provided instructions for the production of a C3 inhibitor.

After injection of the therapy, the inhibitor successfully blocked C3, reduced microglia engulfment, and preserved nerve cell connections, which improved eyesight in mice.

As a result of this inhibition, we saw improved visional function in mice, Werneburg said.

Overall, based on the results, the team believes that C3 probably is sending a signal to microglia telling them to eliminate synapses.

The next step will be to determine how C3 turns active during MS and other neurodegenerative diseases.

Its possible that therapies targeting different circuits of the brain can be used to protect against synaptic damage in other neurodegenerative diseases such as Alzheimers, Schafer said.

Ana is a molecular biologist with a passion for discovery and communication. As a science writer she looks for connecting the public, in particular patient and healthcare communities, with clear and quality information about the latest medical advances. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in genetics, molecular biology, and infectious diseases

Total Posts: 1,053

Patrcia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

See original here:
Gene Therapy Recovers Vision in Mice Models of MS, Uncovers How... - Multiple Sclerosis News Today

Posted in Genetic Therapy | Comments Off on Gene Therapy Recovers Vision in Mice Models of MS, Uncovers How… – Multiple Sclerosis News Today

Otonomy Announces Multiple Presentations at Association for Research in Otolaryngology Annual MeetingPreclinical results to be presented for GJB2 gene…

SAN DIEGO, Jan. 23, 2020 (GLOBE NEWSWIRE) -- Otonomy, Inc.,(Nasdaq: OTIC), a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, today announced multiple presentations at the upcoming Association for Research in Otolaryngology (ARO) 43rd Annual MidWinter Meeting, to be held January 25-29, in San Jose, California. These presentations include initial preclinical results from the companys gene therapy collaboration with Applied Genetic Technologies Corporation (Nasdaq: AGTC) focused on GJB2 deficiency, the most common cause of congenital hearing loss, and demonstration of otoprotection for a class of compounds Otonomy is evaluating in preclinical studies for cisplatin-induced hearing loss (CIHL).

Our multiple presentations at ARO again this year highlight our broad pipeline in neurotology, with data to be presented in support of our programs addressing the treatment of both inherited and acquired forms of hearing loss as well as protection against cisplatin ototoxicity," said David A. Weber, Ph.D., president and chief executive officer of Otonomy. While our highest priority during 2020 is the successful completion of our three ongoing clinical trials, including the Phase 3 trial of OTIVIDEX in Mnires disease, we remain committed to the advancement of our multiple preclinical programs that address important unmet needs in neurotology.

All Otonomy presentations are during poster sessions occurring from 1 to 4 p.m. PST on the days indicated below.

Joint presentation with AGTC related to the GJB2 gene therapy program:

Presentations related to Otonomys OTO-510 program for CIHL:

Presentations related to Otonomy's other clinical and preclinical hearing loss programs:

Joint presentation of data with research collaborators at the University of Washington related to preclinical evaluations of an age-related hearing loss model:

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

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, timing of results of ongoing clinical trials, including the Phase 3 clinical trial for OTIVIDEX; expectations regarding 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.


Media Inquiries:Spectrum ScienceChlo-Anne RamseyVice

Investor Inquiries:Westwicke ICRRobert H. UhlManaging

Here is the original post:
Otonomy Announces Multiple Presentations at Association for Research in Otolaryngology Annual MeetingPreclinical results to be presented for GJB2 gene...

Posted in Genetic Therapy | Comments Off on Otonomy Announces Multiple Presentations at Association for Research in Otolaryngology Annual MeetingPreclinical results to be presented for GJB2 gene…

The year in new drugs: Looking back and ahead –

Gene therapy is a very hot area. Scientists can now fix defective genetic mutations that cause diseases. An initial focus is in rare diseases with urgent clinical needs, but the future holds the possibility for significant expansion.

Another promising area is the broadening of the scope of cell therapy, which is currently being used for blood cancers, into solid tumors. If this is successful, it could transform the field of personalized cancer care.

Finally, Biogen has a novel antibody drug for the treatment of Alzheimers. If the FDA approves this therapy, it could lead to the resurgence of Alzheimers drug development, which represents a huge unmet medical need.

How about at OMRF? What did 2019 deliver, and what has you excited for 2020?

The FDA approved Adakveo in November, a new drug for sickle cell disease based on discoveries made by OMRFs Dr. Rod McEver. In 2020, Im excited about Progentec, an OMRF spin-off company thats developing novel diagnostic and disease management solutions for autoimmune patients.

Also, we have a new startup called Ambocept thats developing novel cancer therapeutics. Finally, two drugs that began in OMRF labs are progressing in clinical trials: one to treat hearing loss, the other for people suffering from deadly brain cancers.

Paula Burkes, Business writer

(Story continued below...)

Excerpt from:
The year in new drugs: Looking back and ahead -

Posted in Genetic Therapy | Comments Off on The year in new drugs: Looking back and ahead –

Bioprocessing Summit Europe Returns March 24-26, 2020 in Barcelona with New Analytical and Formulation Sessions – b3c newswire

Conference to Feature New Case Studies on Cell/Gene Therapy CMC and Manufacturing, Upstream and Downstream Processing

BARCELONA, Spain, January 21, 2020 / B3C newswire / -- Cambridge Healthtech Institute (CHI) today announced the return of Bioprocessing Summit Europe --the fastest growing bioprocessing meeting on the continentwith the addition of approximately 30 new sessions on Analytical and Formulation topics.

Now in its third year, the March 24-26, 2020 conference is moving to The Crowne Plaza BarcelonaFira Center, Barcelona, Spain, to accommodate increased registrations, which have doubled since the meeting was launched in 2018.

Altogether, more than 130 presentations for senior-level scientists and technologists from industry, government and academia will focus on Upstream Processing, Downstream Processing, Cell Therapy CMC and Manufacturing, and Gene Therapy CMC and Manufacturing, as well as innovations in Analytical/Formulation areas.

These sessions will provide solutions to the challenges of producing complex biologics while maximizing quality, risk management and cost-efficiency. Experts from such organizations as Novartis, Pfizer, National Institute for Bioprocessing Research and Training (NIBRT), Roche, Merck, Sanofi-Aventis and Bristol-Myers Squibb will share scientific data, insights and case studies on technologies and processes.

Bioprocessing Summit Europe gives participants a rare opportunity to benchmark with their peers, and benefit from unpublished information that is inaccessible elsewhere, said Dan Barry, Senior Conference Director, CHI.

The new Analytical and Formulation stream will include two tracks:

Other conference highlights will include:

For more information, please see

About Cambridge Healthtech Institute Cambridge Healthtech Institute (CHI), a division of Cambridge Innovation Institute, is the preeminent life science network for leading researchers and business experts from top pharmaceutical, biotech, CROs, academia, and niche service providers. CHI is renowned for its vast conference portfolio held worldwide including PepTalk, Molecular Medicine Tri-Conference, SCOPE Summit, Bio-IT World Conference & Expo, PEGS Summit, Drug Discovery Chemistry, Biomarker World Congress, World Pharma Week, The Bioprocessing Summit, Next Generation Dx Summit, Immuno-Oncology Summit, and Discovery on Target. CHI's portfolio of products include Cambridge Healthtech Institute Conferences, Barnett International, Insight Pharma Reports,Bio-IT World,Clinical Research NewsandDiagnostics World.


Dawn Ringel, Ringel PR+1-781-449-8456This email address is being protected from spambots. You need JavaScript enabled to view it.

Keywords: Protein Aggregates; Proteins; Cell- and Tissue-Based Therapy; Cell Aggregation; Research; Genetic Therapy; Biological Products; Europe; Drug Compounding; Drug Formulation; Pharmaceutical Formulation; Biological Products

Published by B3C newswire and shared through Newronic

The rest is here:
Bioprocessing Summit Europe Returns March 24-26, 2020 in Barcelona with New Analytical and Formulation Sessions - b3c newswire

Posted in Genetic Therapy | Comments Off on Bioprocessing Summit Europe Returns March 24-26, 2020 in Barcelona with New Analytical and Formulation Sessions – b3c newswire

CNCE Hits Right Notes, BXRX Faces FDA In Feb., All Eyes On AGTC – Nasdaq

(RTTNews) - Today's Daily Dose brings you news about Akcea's promising trial results of antisense drug AKCEA-APOCIII-LRx, Applied Genetic's upcoming event, the near-term catalyst of Baudax Bio and Sierra Oncology's reverse stock split.

Read on...

Shares of Akcea Therapeutics Inc. (AKCA), a majority-owned affiliate of Ionis Pharmaceuticals Inc., and Ionis Pharmaceuticals Inc. (IONS) rose more than 10% on Wednesday, following positive topline results from the phase II study of the antisense drug AKCEA-APOCIII-LRx in the treatment of patients with hypertriglyceridemia who are at risk for or have established cardiovascular disease (CVD).

The study met the primary endpoint of significant triglyceride lowering and multiple secondary endpoints with a favorable safety and tolerability profile.

According to the Company, at the highest, once-monthly dose of 50 mg, more than 90% of patients achieved serum triglycerides of less than or equal to 150 mg/dL, compared to less than 5% of patients in the placebo group. The mean triglyceride level of patients at baseline was 285 mg/dL.

AKCA closed Wednesday's trading at $19.24, up 10.96%.

Applied Genetic Technologies Corp. (AGTC) is all set to announce interim six-month data from the dose-escalation groups, and an update of enrollment for its ongoing two parallel phase I/II studies of its gene therapy candidate for Achromatopsia, dubbed CNGB3 and CNGA3, on January 23, 2020.

Achromatopsia is a condition characterized by partial or total absence of color vision. People with complete achromatopsia cannot perceive any colors; they see only black, white, and shades of gray. Incomplete achromatopsia is a milder form of the condition that allows some color discrimination (Source: Genetics Home Reference, NIH).

On January 9, 2020, the Company had reported positive interim six-month data from its ongoing phase I/II clinical trial of AAV gene therapy product candidate expressing the RPGR protein for the treatment of X-linked retinitis pigmentosa. The news sent the stock up as much as over 130% to $9.50 that day.

AGTC touched a new 52-week high of $10.42 in intraday trading on Wednesday, before closing at $9.86, up 19.52%.

The FDA decision on Baudax Bio Inc.'s (BXRX) New Drug Application for intravenous (IV) meloxicam for the management of moderate to severe pain is set for February 20, 2020.

The intravenous Meloxicam has been twice rejected by the FDA - in May 2018 and in March 2019. The Company had appealed the 2019 Complete Response Letter.

The assignment of the decision date of February 20, 2020, follows the FDA's acceptance of Baudax's latest complete response package relating to its 2019 complete response letter (CRL) and appeal filing, seeking approval for IV meloxicam.

Baudax Bio is a spinoff from Recro Pharma Inc (REPH) and began trading on the NASDAQ Capital Market on November 22, 2019.

BXRX closed Wednesday's trading at $8.53, up 21.16%.

Concert Pharmaceuticals Inc. (CNCE) has received a Notice of Allowance from the U.S. Patent and Trademark Office for patent related to CTP-543, the Company's investigational treatment for alopecia areata.

The patent, which is expected to expire in 2037, covers pharmaceutical compositions of CTP-543 and methods of treating alopecia areata with CTP-543.

Last month, the Company successfully completed a phase II trial of CTP-543 in alopecia areata. Alopecia areata is an autoimmune disease in which the immune system attacks hair follicles, resulting in partial or complete loss of hair on the scalp and body.

Concert Pharma intends to conduct an end of phase II meeting with the FDA and advance CTP-543 into phase III testing in 2020.

Concert has been locked in a patent battle with Incyte Corp. (INCY) related to CTP-543. Concert's CTP-543 is deuterated Ruxolitinib.

Ruxolitinib, a drug that selectively inhibits Janus kinases 1 and 2 (JAK1 and JAK2), is marketed by Incyte in the U.S. as Jakafi and by Novartis as Jakavi outside the U.S for the treatment of patients with myelofibrosis and polycythemia vera.

In April 2017, Incyte filed an inter parties review, or IPR, petition with the PTAB, of the U.S. PTO, challenging the validity of U.S. Patent No. 9,249,149, which claims deuterium-modified versions of Ruxolitinib, including CTP-543, are a novel composition of matter. Last April, the Patent Trial and Appeal Board (PTAB) ruled that the claims of the '149 patent are not patentable. Concert appealed the ruling of PTAB last June.

Amid all these appeals and counter appeals, CTP-543 being issued a new patent is good news for Concert Pharma.

CNCE closed Wednesday's trading at $13.09, up 33.03%.

Sierra Oncology Inc. (SRRA) has implemented a 1-for-40 reverse stock split that became effective Wednesday at 4:30 p.m. Eastern Time.

The Company's common stock will trade on a split-adjusted basis at the market open on January 23, 2020.

SRRA closed Wednesday's trading at $0.34, down 11.14%.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

View post:
CNCE Hits Right Notes, BXRX Faces FDA In Feb., All Eyes On AGTC - Nasdaq

Posted in Genetic Therapy | Comments Off on CNCE Hits Right Notes, BXRX Faces FDA In Feb., All Eyes On AGTC – Nasdaq