Novo Nordisk A/S terminated its global licensing deal with Japan’s Heartseed Inc. for its allogeneic induced pluripotent stem cell (iPSC)-derived cardiomyocytes.

Two independent studies applied CRISPR-based genetic editing – one to treat leukemia and the other to target myeloma – to overcome the challenges faced by CAR T cells, such as exhaustion, impaired activation and fratricide, a phenomenon in which they attack each other.

The U.S. FDA released a trio of draft guidances to help sponsors in developing and monitoring cell and gene therapies, as well as other regenerative medicine therapies.

Renal cell carcinoma accounts for approximately 90% of kidney cancers, and current treatments fail to prevent metastasis in up to 40% of patients. Potentially effective is immunotherapy based on CAR T cells that recognize CD70, which is little expressed in normal tissues but is expressed in more than 80% of renal cell carcinomas. However, such CAR T immunotherapy has so far not shown overwhelming success against renal cell carcinoma, and the therapeutic cells must be derived for each patient individually.

The winding regulatory road for the BLA to Capricor Therapeutics Inc.’s cell therapy for Duchenne muscular dystrophy has more clarity. Out of a recent type A meeting between Capricor and the U.S. FDA, prompted by a complete response letter in July regarding lead asset CAP-1002 (deramiocel), the two are in agreement about a path to potential approval.

Amyotrophic lateral sclerosis (ALS), formerly known as Lou Gehrig’s disease, is a progressive and fatal neurodegenerative disorder with no known cure. While three therapies have gained U.S. FDA approvals to date, including Rilutek (riluzole), Radicava/Radicava ORS (edaravone) and tofersen (BIIB-067, the lack of a disease-modifying drug has spurred the continual search for novel therapies.

As Wall Street awaits phase IIb data from Kala Bio Inc. with KPI-012 in persistent corneal epithelial defect, odds are being weighed for that candidate – and others in development – against the lone approved treatment used in a market already worth more than $1 billion.

Mutations in the F9 gene cause factor IX (FIX) deficiency, leading to severe bleeding in people with hemophilia B. Current therapies rely on frequent FIX infusions or newer agents that rebalance coagulation, but both approaches still require weekly to bimonthly dosing and often fail to entirely prevent breakthrough bleeding events, which remain common in patients. Therefore, new therapeutic strategies providing a predictable, durable, active and redosable source of FIX are urgently needed.