miRagen Therapeutics Inc. is developing microRNA-based therapeutics for treating cardiovascular and muscle disease. The company is farthest along with researching potential treatments for heart disease. It has advanced several candidates including the miR-15/195 family to improve cardiac remodeling that occurs in heart muscle following a heart attack, miR-208 and miR-499 to address heart failure, and miR-29 to treat cardiac fibrosis.
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CEO Paul Stoffels said gaining US clearance for an IND for its novel CAR-T product was demanding, but now opens up a pathway towards a pivotal study starting in 2025.
A final rejection of Leqembi could also spell the same fate for Lilly’s rival drug but public outcry and demand for Alzheimer’s therapies might force the regulator’s hand
The bispecific is the first in its class to record a Phase III survival win in small-cell lung cancer, although antibody-drug conjugates and other rivals could challenge in the coming years.
Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.
The Oxford group has linked up with the NHS Cancer Vaccine Launch Pad.
