CRISPR-Cas9 gene editing as a treatment for Duchenne muscular dystrophy. One preclinical program, at the University of Texas Southwestern Medical Center, has moved to Boston startup Exonics Therapeutics with $5 million in seed funding from the nonprofit group CureDuchenne.
—Bill & Melinda Gates Foundation-backed Affinivax, which Xconomy profiled in 2014, got $10 million to develop a vaccine for pneumococcal disease.
—Princeton, NJ-based Advaxis (NASDAQ: [[ticker:ADXS]] signed a research partnership with Sellas Life Sciences Group that will combine their respective technologies to develop a more precise cancer immunotherapy treatment.
UNDER REVIEW
—Shares of Sarepta Therapeutics (NASDAQ: [[ticker:SRPT]]) slid 15 percent after the company projected about $80 million in 2017 sales of its Duchenne muscular dystrophy drug eteplirsen (Exondys 51), falling short of analyst estimates. The drug has faced pushback from insurers since its September approval.
—The FDA has begun a review of the blood cancer medicine enasedenib from Agios Pharmaceuticals (NASDAQ: [[ticker:AGIO]]) and will make a decision by Aug. 30.
—After shoring up manufacturing problems, Aerie Pharmaceuticals (NASDAQ: [[ticker:AERI]]) resubmitted its approval application for glaucoma drug netarsudil (Rhopressa).
—La Jolla Pharmaceticals (NASDAQ: [[ticker:LJPC]]) plans to file for FDA approval for its low blood pressure drug LJPC-501 later this year. The San Diego biotech’s synthetic hormone showed promising results in a Phase 3 clinical trial.
—Cempra (NASDAQ: [[ticker:CEMP]]) of Chapel Hill, NC, laid off 67 percent of its workforce. The firm was stung by trial failures of its solithromycin antibiotic in separate trials for pneumonia and gonorrhea. It did, however, recently report positive Phase 3 results from fusidic acid (Taksta) in treating aggressive skin infections.
—Shares of PTC Therapeutics (NASDAQ: [ticker:PTCT]]) fell about 20 percent after its drug ataluren (Translarna) failed its third trial, this time a Phase 3 study in cystic fibrosis. Ataluren is conditionally approved in Europe for Duchenne muscular dystrophy.
Map image by Dave Winer via a Creative Commons 2.0 license.
Ben Fidler and Frank Vinluan contributed to this report
