Cell and Gene Therapy

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.

While ex vivo genome editing results in highly effective cell therapies, it can lead to off-target effects. Caribou Biosciences has come up with a novel approach for potentially more precise gene editing compared to all-RNA guides.

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.

Stephen Majors from the Alliance for Regenerative Medicine, which hosts the conference, spoke with BioSpace about what the more than 2,000 attendees can expect to learn next week in Phoenix about the pressing issues confronting the industry.

To say that 2seventy bio’s short two years of existence have been dramatic is an understatement. CEO Chip Baird told BioSpace transparency and a committed staff have kept the biotech going through thick and thin.