Originally published on January 25, 2021 11:40 am
Over the last several years, parents from across the country have appeared on television and news outlets to raise money to develop treatments for their children with rare genetic disorders.
Many of these families, including one from Kansas City, Missouri, have pinned their hopes on one Texas researcher, Dr. Steven Gray of the University of Texas Southwestern Medical Center in Dallas, who claims to be on the verge of treating a number of rare conditions. They ve raised millions of dollars to fund his research, although breakthroughs haven t happened to the extent many had hoped.
While gene therapy holds great promise, the growing trend of family-funded research concerns some medical ethicists, who say that suggestions to parents that treatments may be imminent can raise thorny ethical issues.
Building the Pipeline: Passage Bio Launches Manufacturing Operations
Passage Bio President and CEO Bruce Goldsmith, PhD, details the company’s approach to manufacturing and its progress in developing its pipeline of gene therapies
December 23, 2020
Passage Bio plans to support lab operations for its gene therapy programs at a new lab at the Princeton West Innovation Campus. The lab will be the company’s second manufacturing facility, following the opening of a facility in Harmans, MD, at the campus of Catalent Cell & Gene Therapy.
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Gene therapy developers have been ringing out 2020 with a series of announcements about new manufacturing facilities, or at least production agreements with CDMOs. Thermo Fisher will build at its campus in Carlsbad, CA, while Taysha Gene Therapies disclosed plans in Durham, NC. Also, Passage Bio has announced two manufacturing developments at Catalent Cell & Gene Therapy’s Harmans/BWI campus in Harmans, MD and at the Princeton West Innova
RA Session II, founder, president and CEO of Taysha (Taysha Gene Therapies)
Taysha Gene Therapies hit the ground running when it launched this spring, cruising through a $95 million series B straight to a $100 million IPO in September. With its lead program now approved for human trials in Canada, the company is sketching out plans for what it hopes will become its prime commercial manufacturing site.
Taysha leased a 187,000-square-foot commercial-scale manufacturing facility in Durham, North Carolina, planning to invest $75 million to kit out the facility for preclinical, clinical and commercial gene therapy production. It’s also due to receive up to $9.4 million in state and local incentives for the project, Taysha said in a release.
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TSHA-101 to be first bicistronic vector evaluated in human clinical trials; TSHA-101 designed to deliver both HEXA and HEXB transgenes within a single AAV9 vector construct
TSHA-101 CTA is the second clinical trial clearance received, in addition to TSHA-118’s open investigational new drug application for CLN1
Interim data from Phase 1/2 trial anticipated in 2021
DALLAS, TX, USA I December 21, 2020 I Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that Queen’s University in Ontario, Canada, received Clinical Trial Application (CTA) approval from Health Canada for its investigator-sponsored Phase 1/2 trial exploring TSHA-101, Taysha’s investigational AAV9-based gene therapy, for the treatment of infantile GM2 gangliosidosis.
Taysha Gene Therapies Announces Queen s University s Receipt of Clinical Trial Application Approval from Health Canada for Phase 1/2 Clinical Trial of TSHA-101 for the Treatment of Infantile GM2 Gangliosidosis – IT Business Net itbusinessnet.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from itbusinessnet.com Daily Mail and Mail on Sunday newspapers.