Exonics Using CRISPR To Develop One-Time Treatment for Duchenne
Newly formed biotech company hopes to avoid patent melee and use gene editing technology to develop therapy for up to 80% of boys with DMD; trial outcomes to drive terms of future licensing deals.
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CRISPR Therapeutics and Vertex initiated the first human study of CRISPR gene-editing technology with an industry sponsor, following an investigator-sponsored study initiated in China two years ago. These are not the first gene-editing trials, but they're important milestones for the CRISPR field.
Five funds raised more than $1bn to back biotech firms; Arcus leads recent VC rounds with $107m GV-backed Series C; Celgene, Valeant sell notes to retire debt; and Alnylam cashes in on investor goodwill.
National Academies’ report on the fast-advancing genome-editing technologies points out that ways to improve musculature in dystrophy patients will almost certainly raise issues about treatment use in narrow population versus enhancement use in broader population. In most cases, however, specificity of genome-edited products will help limit use of the new therapies to targeted populations.