Gene therapy
Analysts did not seem very concerned by the treatment-related serious adverse event, noting that NGN-401 was well-tolerated at a lower dose and showed promising efficacy outcomes.
The investigational therapy, vesleteplirsen, had been positioned as an updated version of Sarepta’s original exon 51-skipping Duchenne muscular dystrophy drug Exondys 51.
BEAM-101 seems to be competitive with approved sickle cell treatments, William Blair analysts said in a note to investors, but a patient death underscores the need for less-toxic preconditioning treatments.
The biotech beat Wall Street’s third-quarter revenue forecast by 6%, driven by increased uptake of its achondroplasia drug Voxzogo. However, William Blair downgraded BioMarin’s shares to market perform due to a “lack of near-term catalysts” and uncertainty around Voxzogo’s potential revenue growth.
With Sarepta’s gene therapy Elevidys now available to a majority of Duchenne muscular dystrophy patients, experts express cautious optimism while emphasizing the need for further data.
With an upfront payment of $50 million from Roche, the partnership will leverage Dyno Therapeutics’ in vivo gene therapy delivery technology, which synthesizes virus capsids with better functionality and manufacturability.
The pediatric patients, with a rare neurodegenerative disease, were treated with bluebird bio’s Skysona to slow the progression of neurologic dysfunction. Six patients developed myelodysplastic syndrome and one patient developed acute myeloid leukemia.
Large pharmaceutical companies were out in force at this week’s 2024 Cell & Gene Meeting on the Mesa, as they look to expand their presence in the industry.
Tim Hunt, CEO of the Alliance for Regenerative Medicine, said Monday at the 2024 Cell & Gene Meeting on the Mesa that investments reached $10.9 billion in the first half of this year—outpacing 2019’s $9.8 billion total—but far below the pandemic peak.
The Financial Times reported Thursday that WuXi AppTec is looking to sell its cell and gene therapy manufacturing unit, with facilities in Philadelphia, while WuXi Biologics wants to offload some of its production sites in Europe.
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