Valrox (valoctocogene roxaparvovec) from San Rafael Calif-based Biomarin Pharmaceutical Inc. moved one step closer to entering the U.S. market, with the company reporting that that the FDA had accepted for priority review the BLA for its investigational AAV5 gene therapy for adults with hemophilia A.
Ultragenyx Pharmaceutical Inc.’s top-line win in January with DTX-301 gene therapy in ornithine transcarbamylase (OTC) deficiency seemed to presage even better things to come later this year, and analysts more recently hailed fourth-quarter earnings that showed satisfying progress with Crysvita (burosumab).
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.
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four gene therapies already approved and more than 900 in development, the FDA has finalized six guidances and issued a draft guidance to clarify the rules of the road for developing and manufacturing the treatments.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.
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.
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.
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