Cell and Gene Therapy

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.

District Judge Jesse Furman ruled that the plaintiff, UMB Bank, does not have standing to bring the case against Bristol Myers Squibb because it is not a properly appointed trustee for shareholders’ contingent value rights.

In an effort to expand its cash runway beyond 12 months, Prime Medicine has signed a deal with Bristol Myers Squibb worth a potential $3.5 billion, while also streamlining its pipeline to trim costs.

Pfizer’s sudden market withdrawal of sickle cell therapy Oxbryta, which some analysts predicted would reach $750 million in sales by the end of the decade, has left patients and healthcare providers with few options, while investors question the pharma giant’s dealmaking prowess.