The European Commission approved two therapies for progressive, genetic diseases: Biogen Inc.’s Friedreich’s ataxia drug, Skyclarys (omaveloxolone), and Crispr Therapeutics AG’s CRISPR/Cas9 gene therapy for sickle cell disease and transfusion-dependent beta-thalassemia, Casgevy (exagamglogene autotemcel, exa-cel).
Regenxbio Inc. plans to file a BLA this year seeking accelerated approval for gene therapy candidate RGX-121 to treat young children with mucopolysaccharidosis type II, also known as Hunter syndrome, based on positive data from the phase I/II/III Campsiite trial, which not only hit the biomarker endpoint but also indicated potential systemic benefits.
With Adverum Biotechnologies Inc.’s preliminary safety and efficacy data made public from the ongoing Luna phase II trial testing gene therapy ixoberogene soroparvovec (ixo-vec) in wet age-related macular degeneration (AMD), Wall Street promptly began stacking the results against those of competitors. CEO Laurent Fischer pointed out that ixo-vec boasts the “highest rate of injection-free patients of any study of any program at any dose.”
A disease-modifying gene therapy for Sanfilippo syndrome type A has demonstrated reductions in heparin sulfate within cerebrospinal fluid, as well as increases in the cognitive function of young patients, arming Ultragenyx Pharmaceutical Inc. with data needed to support an accelerated BLA with the U.S. FDA.
Sangamo Therapeutics Inc.’s gene therapy for Fabry disease, isaralgagene civaparvovec (ST-920), is continuing to show promising efficacy and safety in the phase I/II study, Staar, but further ahead, the company needs to either attract a partner or secure financing to move to a registrational trial.
4DMT Molecular Therapeutics Inc. is looking ahead to phase III as positive data continue to roll out for gene therapy candidate 4D-150 in wet age-related macular degeneration (AMD), with the results from the phase II portion of the phase I/II Prism trial showing the single-shot treatment significantly reduced the need for chronic anti-VEGF injections. Presented over the weekend at the Angiogenesis, Exudation, and Degeneration 2024 Conference, results from the 24-week dose-expansion cohort, which comprised wet AMD patients with severe disease and high treatment burdens, showed patients receiving the high dose (3E10 vg/eye) had a 90% reduction in annualized anti-VEGF injection rates.
Sarepta Therapeutics Inc.’s next-generation peptide-conjugated PMO therapy, SRP-5051 (vesleteplirsen), looks set to stake its claim in the Duchenne muscular dystrophy (DMD) space, as phase II data unveiled dystrophin expression that outperforms first-generation exon-skipping drug Exondys 51 (eteplirsen). The question is what that space might look like in the wake of a U.S. FDA decision whether to expand labeling and convert to full approval Sarepta’s DMD gene therapy, Elevidys (delandistrogene moxeparvovec).
The hearing has returned for the first person who has received gene therapy for treating genetic hearing loss in the U.S. Initial results from Akouos Inc.’s phase I/II study showed that within 30 days of receiving AK-OTOF-101, pharmacologic hearing was restored to an 11-year-old who had profound hearing loss from birth.
As gene therapies gain unprecedented traction into 2024, preclinical-stage South Korean biotech Genecraft Inc. said it raised ₩10 billion (US$7.48 million) in a series A financing to further R&D for its therapy against lung cancer.
As gene therapies gain unprecedented traction into 2024, preclinical-stage South Korean biotech Genecraft Inc. said it raised ₩10 billion (US$7.48 million) in a series A financing to further R&D for its therapy against lung cancer.
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