Protocols

CRISPR Therapeutics treats first patient with gene editing tech; Amicus nabs ‘breakthrough’ status for Pompe drug

→ The long awaited first-in-human testing of CRISPR gene editing tech — if you skip past the recent controversial case in China — has arrived. CRISPR Therapeutics is claiming the honor with the dosing of its Vertex-partnered drug, CTX001, in a patient with beta thalassemia. The biotech plans to treat one other patient in the Phase I/II open-label trial before opening enrollment up; it is also planning to treat a sickle cell disease patient with the drug in mid-2019 for a separate trial. Intellia and Editas are fast on its heels.

Amicus Therapeutics $FOLD has picked up a breakthrough therapy designation for its experimental drug for late onset Pompe disease. The BTD for AT-GAA — a program designed to speed a company along an efficient regulatory path — was based on the clinical efficacy results researchers have been seeing in the ongoing ATB200-02 Phase I/II clinical study.

→ As FDA review of Aimmune’s NDA presumably gets back on track, the company is highlighting a slate of new data to bolster the case for its peanut allergy med. Some key findings among the mix pertain to AR101’s ability to tamper patient reaction to accidental exposures and the longterm tolerance toward peanut protein. Researchers document a 70% reduction in allergic reactions due to accidental exposures that require treatment compared to placebo, and a 95% rise in probability of tolerating any dose of peanut protein for those who completed the exit challenge.

Taiho Oncology, the US subsidiary of Japan’s Taiho Pharma, has nabbed a supplemental FDA approval for its colorectal cancer drug. The new approval covers the use of Lonsurf as a third-line treatment for metastatic gastric or gastroesophageal junction adenocarcinoma — in patients previously treated with at least two prior lines (and specific types) of chemotherapy. The drug has previously demonstrated improvements in overall survival compared to placebo, the company said.


With contribution by John Carroll.


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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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