Beam Therapeutics
NEWS
Vertex unveiled long-term durability data for Casgevy, while Beam presented Phase I/II findings for its investigational base editor BEAM-101, building up to a BLA by late 2026.
BEAM-101 seems to be competitive with approved sickle cell treatments, William Blair analysts said in a note to investors, but a patient death underscores the need for less-toxic preconditioning treatments.
Vertex may have pivoted away from the space, but candidates in development by Arrowhead/Takeda, Wave, Korro and others could address the damage underlying alpha-1 antitrypsin deficiency and make today’s treatments a thing of the past.
Biotech Beam Therapeutics has dosed the first patient in a Phase I/II study of its base-edited CAR-T therapy in relapsed or refractory T-cell acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma.
Gene editing technologies are advancing rapidly in the clinic, with the potential first approval later this year, but challenges remain.
Orbital will use Series A funds to advance RNA-based vaccines, immunomodulators and protein replacement therapies.
Currently, there are no gene editing–based treatments on the market, but the technology continues its march toward potential FDA approval, with several products in mid- and late-stage trials.
The FDA has lifted its clinical hold on Beam Therapeutics’ BEAM-201 and cleared the way for an Investigational New Drug Application, the company announced Friday.
Beam Therapeutics has enrolled the first patient in a Phase I/II trial studying a gene therapy leveraging base editing in adults with severe sickle cell disease.
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