Cell and Gene Therapy

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.

While some of the initial excitement around immunotherapies has waned, companies—particularly smaller biotechs—are developing newer iterations that will take cancer care to the next level.

The biotech beat Wall Street’s third-quarter revenue forecast by 6%, driven by increased uptake of its achondroplasia drug Voxzogo. However, William Blair downgraded BioMarin’s shares to market perform due to a “lack of near-term catalysts” and uncertainty around Voxzogo’s potential revenue growth.

With Sarepta’s gene therapy Elevidys now available to a majority of Duchenne muscular dystrophy patients, experts express cautious optimism while emphasizing the need for further data.

With an upfront payment of $50 million from Roche, the partnership will leverage Dyno Therapeutics’ in vivo gene therapy delivery technology, which synthesizes virus capsids with better functionality and manufacturability.