Gene therapy
As Q1 2025 earnings season continues, tariffs remain top of mind for pharma CEOs and investors. Meanwhile, the American Association for Cancer Research’s annual event kicks off this year’s oncology conference season. Plus, will the FDA become politicized under HHS Secretary RFK Jr.?
The condition, recessive dystrophic epidermolysis bullosa, causes chronic wounds and has an 84% mortality rate by age 40.
As tariffs, HHS workforce cuts and the ouster of CBER Director Peter Marks threaten the “lifeblood” of the cell and gene therapy space, experts express wariness over the unknowns and optimism that Marks’ legacy will carry on.
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.
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.
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