CRISPR-based cell therapies continued to gain steam Sept. 27 with the announcements of a potentially valuable big pharma collaboration and an ambitious global regulatory push.
Vector Biopharma AG has secured a $30 million series A funding commitment from founding investor Versant Ventures to take forward a new gene delivery platform developed by Andreas Plückthun, of the University of Zurich, in Switzerland.
Scientists will investigate whether cutting-edge technology such as base editing could be used to cure inherited heart muscle conditions after an international team co-led by Harvard Medical School won a research challenge. The $36 million Big Beat Challenge, run by the British Heart Foundation, is one of the largest non-commercial awards ever given and will focus on inherited heart muscle diseases known as genetic cardiomyopathies.
Controlling the epigenetics of a patient, figuring out what genes are expressed and understanding their level of expression, is at the center of Epic Bio, a new company founded by Stanley Qi, co-inventor on the CRISPR patent held by the University of California.
Although there has been huge progress in treatment of cystic fibrosis over the last decade, with Vertex Pharmaceuticals Inc. becoming the first to address the underlying cause of the disease with its Kalydeco (ivacaftor), approved in 2012, there are still many patients who aren’t eligible for treatment.
As fellow gene editing firm Crispr Therapeutics AG hosted an innovation day in which it confirmed plans for regulatory filings by year-end for an ex vivo gene editing therapy in sickle cell disease and beta-thalassemia, Precision Biosciences Inc. announced plans to develop an in vivo gene editing approach through a collaboration with Novartis AG that brings Precision an initial $75 million with up to $1.4 billion in potential milestones.
Researchers from the Center for Genome Engineering within South Korea's Institute for Basic Science have developed a new gene-editing platform that could be the final missing piece of the puzzle in gene-editing technology, by making editing of mitochondrial DNA possible.
Versant Ventures is committing $50 million in series A funding to Cimeio Therapeutics Inc., which aims to bring new possibilities in terms of disease targeting and safety to bear on a wide range of cell therapy applications, including hematopoietic stem cell transplantation (HSCT) and adoptive cell transfer.
Spotlight Therapeutics Inc., a company developing cell-targeted in vivo CRISPR gene editing biologics, has raised $36.5 million in series B financing to support advancement of its first-in-class immuno-oncology program and further applications of its in-house technology platform. The financing round was co-led by new investors GordonMD Global Investments and Epiq Capital Group, with participation from Magnetic Ventures, as well as existing investors GV, Emerson Collective and others.
