Gene therapy

The cell and gene therapy company is cutting 47 employees and its entire lupus program to focus resources on two CAR Ts. The move follows a reconfiguration last year to move into immunology.
Despite making an unsolicited bid for gene therapy maker bluebird bio, Ayrmid failed to deliver a binding offer after weeks of due diligence. Bluebird’s board recommended that it go with Carlyle and SK Capital Partner’s original offer to take the company private for $30 million.
Stifel analysts said that Lexeo’s data showing reduced size and thickness of the heart’s left ventricle are “supportive of a drug effect” for the company’s gene therapy in Friedreich’s ataxia cardiomyopathy.
One day after the European Medicines Agency requested that three clinical trials of Elevidys be placed on hold after the death of a U.S. teenager, a data monitoring committee concluded that they should continue unchanged.
Sangamo, which has been having cash problems, will receive $18 million upfront in licensing fees for its AAV capsid that in preclinical studies has shown the ability to cross the blood-brain barrier.
FDA
Already reeling from years of market chaos, the announced departure of CBER chief Peter Marks sent a ripple across biopharma markets.
The layoffs will take place throughout 2025 and will mostly affect Tenaya’s research and manufacturing operations. The company is continuing to test its hypertrophic cardiomyopathy gene therapy.
Ayrmid’s offer is 50% higher than bluebird’s previously announced deal with Carlyle and SK Capital Partners.
Industry updates and investment insights—including investor behavior and fundraising advice—were among the topics experts explored at Charles River Laboratories’ 2025 Cell & Gene Therapy Summit.
Werner held roles at Bristol Myers Squibb, AstraZeneca and Novartis before landing at Alltrna, where she works to develop tRNA-based treatments for a range of diseases.
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