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.
Invasive fungal infections pose a significant global health challenge due to their severity and the scarcity of effective and safe treatment options. Unlike antibacterial drug development, creating new antifungals is especially challenging because fungal and human cells share a eukaryotic structure, highlighting the need for innovative treatment strategies.
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.
HCW Biologics Inc. has developed second-generation, tissue factor-targeting T-cell engagers (TCEs) to treat solid tumors, particularly pancreatic cancer and glioblastoma, constructed with its novel proprietary TRBC product discovery and development platform technology.
Tidewave Bio has completed an FDA Interact meeting that provided regulatory feedback as the company advances its lead program, TBC-1, toward IND-enabling studies. The company is developing a universal, off-the-shelf immunotherapy platform that is converted into a patient-specific precision medicine in real time using a tumor biopsy.
Lineage Cell Therapeutics Inc. has entered into a research collaboration with William Demant Invest A/S to develop Lineage’s auditory neuronal cell transplant Resonance (ANP-1) for hearing loss.
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