Page 11 - Gene Therapy Program News Today

AIDS@40: Stories of hope and heroes The people and the science devoted to stopping HIV Álex Cámara / Getty Images June 5, 2021, will mark 40 years since Los Angeles physician Dr. Michael Gottlieb and colleagues published the first medical account of what would eventually become known as AIDS. It was a short summary in the Centers for Disease Control and Prevention’s printed weekly newsletter of five cases of pneumonia in gay men, all of whom eventually died. The global story tha...

Date Time Discovery Labs Signs Foundational Lease with University of Pennsylvania Gene Therapy Program as Anchor Tenant KING OF PRUSSIA, Pa., (May , 2021) – The Discovery Labs has signed a foundational lease with the University of Pennsylvania Gene Therapy Program (GTP), which will use Discovery Labs’ suburban campus for a portion of its expanding research operations focused on the development of genetic medicines for rare and orphan diseases, as well as acquired and pandemic infectious di...

/PRNewswire/ -- The Discovery Labs has signed a foundational lease with the University of Pennsylvania Gene Therapy Program (GTP), which will use Discovery...

Published: May 19, 2021   Joined an alliance of life science companies dedicated to rare disease therapy development to support advocacy for policies that will improve processes for timely and cost-effective delivery of treatments   DALLAS--(BUSINESS WIRE)-- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system...

Published: May 13, 2021   Mary Newman, former SVP of Regulatory Affairs at Astellas Gene Therapies, joins Taysha with over 30 years of experience in regulatory affairs and research and development in the biotech industry Extensive experience deeply rooted in gene therapy at Astellas Gene Therapies, Audentes Therapeutics and BioMarin   DALLAS--(BUSINESS WIRE)-- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commer...

Message : Required fields Passage Bio collaborator University of Pennsylvania’s Gene Therapy Program to present poster regarding development of a novel mouse model for evaluating efficacy of adeno-associated virus gene therapy for metachromatic leukodystrophy (MLD) Passage Bio gene therapy candidate, PBML04, showed preliminary signs of efficacy in novel mouse model of MLD PHILADELPHIA, May 11, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines comp...

Published: May 10, 2021 Galafold® (migalastat) PerformanceReflects Continued Strong Adoption in All Key Global Regions; On-Track to Achieve Revenue Guidance of $300M-$315M 1Q21 Total Galafold Revenue of $66.4M Driven by Continued Global Growth Positive Pre-BLA Meeting Held with U.S. FDA for AT-GAA in Pompe Disease; Rolling BLA Submission On-Track for Completion in 2Q21with Global Submissions Expected Throughout 2021 New Data from Pompe and Fabry Gene Therapy Programs to be Presented at Ame...

Published: May 06, 2021 SOUTH SAN FRANCISCO, Calif., May 06, 2021 (GLOBE NEWSWIRE) -- Catalyst Biosciences, Inc.. (NASDAQ: CBIO) today announced its operating and financial results for the first quarter ended March 31, 2021 and provided a corporate update. “We made significant progress across our Protease Medicines platform, specifically in our complement and hemostasis programs. We are preparing to initiate an observational trial in patients who have diseases related to CFI deficiency in...

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Better Outfitted and Screened Viral Vectors Are Safer and More Effective Better Outfitted and Screened Viral Vectors Are Safer and More Effective Adeno-associated virus (AAV) vectors are the leading vectors for gene delivery. Two AAV-based gene therapies have been approved by the U.S. Food and Drug administration, and dozens more are in clinical trials. However, AAV vectors pose challenges including costly manufacture, high percentages of empty capsids, and safety issues associated with high-d...