Dive Brief:

The fields of synthetic lethality (a combination of DNA defects that work together to destroy a cancer cell) and DNA repair, fueled by gene editing technologies such as CRISPR/Cas9, have only been around a couple of decades but they are creating a solid foothold in basic research and are now moving into active development.

Repare Therapeutics is using its high-throughput, genomic synthetic lethal screening platform, which includes CRISPR/Cas9 genome editing, to find and exploit DNA damage repair (DDR) defects found across virtually all cancers.

"Versants commitment to and confidence in Repares distinct science has enabled the company to build the team, operations and initial programs away from the spotlight,"said Repare CEO Lloyd M. Segal. "With the added leadership of MPM and this syndicate, we are financed to achieve our goal of testing our multiple new, precision oncology therapeutics in a clinical setting."

CRISPR/Cas9 is becoming a busy field, with a number of companies and groups edging towards the clinic. The first in human study of CRISPR/Cas9 was approved by a Federal panel of the National Institutes of Health in mid-2016. The University of Pennsylvania study will target myeloma, melanoma and sarcoma. This was beaten to the clinic by Chinese scientists, who gave a patient with aggressive lung cancer cells edited using CRISPR/Cas9 in October 2016.

Editas Medicine expected to begin clinical trials by the end of 2017 for Leber congenital amaurosis type 10, a rare eye disorder, but these have now been delayed until mid-2018 because of issues with a third-party manufacturer. This is in development in collaboration with Allergan.

CRISPR Therapeutics expects to file a European clinical trial authorization for beta-thalassemia by the end of 2017, using CRISPR techniques to create variants that artificially induce hereditary persistence of fetal hemoglobin (HPFH), an asymptomatic and naturally-occurring condition that has been linked with better outcomes in people with beta-thalassemia and sickle cell disease.

Intellia Therapeutics, CRISPR Therapeutics and Editas Medicine all floated in 2016, though share prices for Intellia and Editas slumped towards the end of the year.

Continued here:

Celgene-backed CRISPR company emerges with $68M Series A - BioPharma Dive

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