With four gene therapies already approved and more than 900 in development, the FDA has finalized six guidances and issued a draft guidance to clarify the rules of the road for developing and manufacturing the treatments.
According to Janet Lambert, CEO of the Alliance for Regenerative Medicine (ARM), in her delivery of the international advocacy group’s state of the industry briefing at Biotech Showcase in San Francisco, 2019 proved to be a significant year of growth for the regenerative medicine sector.
Emendo Biotherapeutics Inc. CEO David Baram told BioWorld his firm’s allele-specific gene-editing approach offers such an advantage over previous methods that “we decided to take the challenge of curing diseases that require the highest precision possible,” and the New York-based firm bears an impressive list of partners. “Doors opened immediately and collaborations formed very fast,” sometimes “even faster than we could digest,” he said.
In a move that Sarepta Therapeutics Inc.’s president and CEO, Doug Ingram, called “transformational” for the company and “the largest licensing transaction in cell or gene therapy history,” Sarepta granted Roche Holding AG exclusive commercial rights outside the U.S. for SRP-9001, its gene therapy for Duchenne muscular dystrophy (DMD).
DUBLIN – Compared to other indication areas, gene therapy for cardiac disease is still at the “caveman” stage, according to Dinaqor AG CEO and Chairman Johannes Holzmeister. The Pfäffikon, Switzerland-based firm aims to drag it into the modern era by applying a novel locoregional delivery system to its adeno-associated virus 9 (AAV9) vector technology.
BEIJING – Gene therapy startup Hui-Gene Therapeutics Ltd. Co., of Shanghai, said it secured more than ¥100 million (US$14 million) in a series A financing round to develop a safer gene therapy to treat genetic diseases caused by single-base mutations.
The mantra for a while has been that it's not good enough to get your drug approved, you've got to get it payed for, too. For gene therapy treatments, the payment is more complex, and you can add manufacturing and supply chain logistics to the list of challenges.
As Wall Street ponders how pricing and reimbursement for gene therapies might shake out, companies in the field march intrepidly on, among them Orchard Therapeutics Inc., which early this month rolled out heartening results from an ongoing proof-of-concept trial evaluating the safety and efficacy of OTL-203 for mucopolysaccharidosis type I (MPS-I), a space that's heating up.
DUBLIN – Gyroscope Therapeutics Ltd. raised £50.4 million (US$60.5 million) to further development of GT-005, its clinical-stage gene therapy for treating dry age-related macular degeneration (AMD), and to advance a second-generation of the subretinal delivery system it gained through its recent merger with Orbit Biomedical Ltd.
RSS
