The quest to get the first drug approved to treat fragile X is heating up with three companies – Tetra Discovery Partners Inc., Zynerba Pharmaceuticals Inc. and Ovid Therapeutics Inc. – starting clinical trials this month.
Approved in late 2016, Spinraza (nusinersen) remains the only drug cleared to treat spinal muscular atrophy (SMA), but multiple companies are looking to grab a share of the blockbuster market established by Cambridge, Mass.-based Biogen Inc. and its development partner, Ionis Pharmaceuticals Inc., of Carlsbad, Calif.
Backed with an upsized $85 million series C financing, Sutrovax Inc. is looking to supplant New York-based Pfizer Inc.'s Prevnar 13 (pneumococcal polysaccharide conjugate vaccine [13-valent, adsorbed]) as the go-to vaccine protecting against Streptococcus pneumonia infection in the $6 billion annual market.
At the World Federation of Hemophilia (WFH) 2018 World Congress, the old and new guard of companies developing hemophilia treatments presented their plans to change the market.
For new drug classes, there’s often the easiest way and the best way. The former often wins in the short term, but the best way will end up being superior – as long as technology can overcome the obstacles.
SAN DIEGO – Feng Zhang, along with Emmanuelle Charpentier and Jennifer Doudna, were awarded the Tang Prize in 2016 for their work in the discovery and development of CRISPR/Cas9, but Zhang used most of his time giving the Tang Prize Award Lecture at the Experimental Biology 2018 conference to highlight the diversity of the CRISPR/Cas family and how his lab has exploited the gene editing system.
CHICAGO – About two years after the formation of the $471 million UBS Oncology Impact Fund (OIF), which is managed by MPM Capital, the fund is living up to its namesake, making an impact by doling out $1.25 million to a new AACR-MPM Transformative Cancer Research Grants Program and another $1.25 million to the UBS Optimus Foundation.
