Cell and Gene Therapy

The all-cash buyout, which gives AbbVie access to Capstan Therapeutics’ in vivo edited CAR T therapy for B cell–mediated autoimmune diseases, adds to a growing sense of momentum in M&A, according to BMO Capital Markets.

Changing how biopharmas package their products, how regulators review new drugs and how mutated genes are fixed could make ultrarare disease treatments possible.

Jefferies analysts called the proxy filing, which is a standard disclosure after a merger agreement, “much more intriguing than normal” given the regulatory turmoil it revealed.

The FDA is assessing the need for “further regulatory action” on Sarepta’s Duchenne muscular dystrophy gene therapy in the aftermath of two patient deaths, though the regulator has not yet specified what action this could be.

Robert F. Kennedy Jr. testified in front of largely combative congresspeople on vaccine policy, his MAHA report and more; the mass leadership exodus at the FDA continues as CDER and CBER shed key staff; Kennedy’s revamped CDC vaccine advisors convene for their first meeting; Novo and Lilly present new data at the American Diabetes Association’s annual meeting; and BioSpace recaps BIO2025.

Isaralgagene civaparvovec is a “potential best-in-class gene therapy for Fabry disease,” according to analysts at H.C. Wainwright. Sangamo plans to use pivotal Phase I/II data to build an accelerated approval case for the asset.

While BMO Capital Markets said that zimislecel is “highly encouraging” for type 1 diabetes, questions regarding its target population and Vertex’s execution hang over the cell therapy’s commercial potential.

The well-respected director of the FDA’s cell and gene therapy office was seen as a stabilizing and trustworthy voice inside the quickly reshaping FDA, especially since the late-March exit of CBER Director Peter Marks.

Another patient has died from acute liver failure after receiving Sarepta’s gene therapy for DMD ; After a quiet start to the year, M&A is back with one deal for a gene editing biotech reinvigorating that sector; and RFK Jr. installs a suite of new vaccine board members who share his skeptical views on vaccines.

The pending deal was rumored overnight after a report from the Financial Times, spurring analysts to speculate that if true, the entire gene editing space would see a boost at the markets.