Cell and Gene Therapy
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Following strong treatment response data for Adaptimmune’s lete-cel, the biotech is planning to initiate a rolling BLA submission to the FDA, set to start by the end of 2025.
The FDA has followed in the footsteps of its European counterparts and granted accelerated approval to PTC Therapeutics’ gene therapy Kebilidi for AADC deficiency. It is the first approved gene therapy to be delivered directly to the brain.
BioSpace takes a look at the headlines across a heavy earnings week for biotechs, checking in on Allogene, Rapport and more.
Analysts did not seem very concerned by the treatment-related serious adverse event, noting that NGN-401 was well-tolerated at a lower dose and showed promising efficacy outcomes.
The investigational therapy, vesleteplirsen, had been positioned as an updated version of Sarepta’s original exon 51-skipping Duchenne muscular dystrophy drug Exondys 51.
Truist Securities analyst Asthika Goonewardene in an investor note said data for anito-cel—particularly its safety profile—will help differentiate the CAR T therapy from Legend Biotech and J&J’s entrenched Carvykti in relapsed and refractory multiple myeloma.
In our anniversary episode, we discuss a rare earnings miss for Eli Lilly, a pivotal metabolic dysfunction–associated steatohepatitis victory for Novo Nordisk’s Wegovy, growing excitement about CAR Ts for autoimmune disease and the ongoing controversy over HeLa cells.
In a tough fundraising space, cell therapy biotechs pursuing autoimmune indications review staffing to ensure the right expertise is in place to tackle the new disease area.
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.
Multiple players are exploring whether modalities designed to combat B cell malignancies can be repurposed against lupus, myasthenia gravis and other conditions traced to misdirected immune response.