Cell and Gene Therapy
THE LATEST
2026 is shaping up to be a pivotal year for rare disease drugmakers, with key approvals, filings and readouts lined up for this year.
AstraZeneca is relying on several upcoming products to help hit its target of $80 billion in revenue by 2030, including drugs for hypertension, breast cancer and generalized myasthenia gravis, all of which are currently under FDA review.
Jefferies analysts forecast a $1 billion market opportunity for each of Sarepta’s siRNA programs for facioscapulohumeral muscular dystrophy and myotonic dystrophy type 1.
FDA Commissioner Marty Makary called these changes “common-sense reforms” that could expedite the development of cell and gene therapies.
Aurora joins the clutch of companies linked to Nobel Prize winner and CRISPR trailblazer Jennifer Doudna.
The prevalence of serious inflammatory safety issues such as cytokine release syndrome and immune effector cell–associated neurotoxicity syndrome limits the reach of these transformative cancer therapies.
Rampart Bioscience was working on a platform to deliver gene therapies without the need for viral vectors.
Next-generation automation is closing the gap between curative science and real-world demand, enabling faster development, global consistency and broader patient access to CAR T therapies.
Industry leaders are focused on the resilience of key starting material supply and the knock-on effects of automation in the new year.
With a pair of Phase III trial flops, Ultragenyx will explore cost reductions as analysts turn attention to an upcoming Angelman syndrome readout.