Regeneron (NASDAQ:REGN) Pharmaceuticals, Inc. (NASDAQ:REGN) and Intellia Therapeutics, Inc. (NASDAQ:NASDAQ:NTLA) announced on Tuesday an expansion of their research collaboration to develop additional in vivo CRISPR-based gene editing therapies, focusing specifically on neurological and muscular diseases. This development builds upon the success of the companies' existing collaboration by combining their respective expertise in biology and technology.
The expanded agreement will initially focus on two in vivo non-liver targets. Intellia will lead the design of the editing methodology while Regeneron will spearhead the targeted viral vector delivery approach. Each company will have the opportunity to lead potential development and commercialization of product candidates for one target, with the option for co-development and co-commercialization if not leading development.
Regeneron's proprietary antibody-targeted adeno-associated virus (AAV) vectors and delivery systems will be combined with Intellia's proprietary Nme2 CRISPR/Cas9 (Nme2Cas9) systems adapted for viral vector delivery. This combination aims to precisely modify a target gene.
Aris Baras, M.D., Senior Vice President and Co-Head of Regeneron Genetic Medicines, commented on the expansion, stating that it takes advantage of new technology and innovations to unlock opportunities previously limited by the inability to deliver a genetic payload to cells beyond the liver.
Christos Kyratsous, Ph.D., Senior Vice President, Research, and Co-Head of Regeneron Genetic Medicines, expressed excitement about testing their proprietary AAV approach with Intellia's gene editing systems to potentially create new medicines for serious neurological and muscular diseases.
Intellia President and CEO John Leonard, M.D., also expressed enthusiasm about expanding their successful collaboration with Regeneron to accelerate the development of CRISPR-based therapies for neurological and muscular diseases.
Regeneron, a leading biotechnology company, invents, develops, and commercializes transformative medicines for serious diseases. Intellia Therapeutics is a leading clinical-stage genome editing company developing novel therapeutics leveraging CRISPR-based technologies. Both companies have reiterated their commitment to continue innovating their respective technologies to realize the full potential of gene editing as a new therapeutic modality.
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