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.
Beone Medicines Ltd. (formerly Beigene Ltd.) is selling its worldwide royalty rights on Imdelltra (tarlatamab) sales, excluding China, to Royalty Pharma for up to $950 million.
Despite the success of traditional viral-based CAR T-cell therapies against several blood malignancies, their efficacy remains limited against solid tumors. Non-viral engineering of CAR T cells using electroporation or lipid nanoparticle delivery of CAR-encoding mRNA achieves high but transient CAR expression, highlighting the limitations of current preclinical models for evaluating mRNA-based CAR T cells.
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