Cell therapy weekly: Intellia and Regeneron’s research collaboration expands to neurological and muscular diseases
This week: The US FDA granted Priority Review for an investigational gene therapy to address severe Leukocyte Adhesion Deficiency-I, Regeneron Pharmaceuticals (NY, USA) and Intellia Therapeutics (MA, USA) announced an expanded research collaboration to develop in vivo CRISPR-based gene therapies for neurological and muscular diseases and RoslinCT (Edinburgh, UK) appointed Dean Morris as Chief Operating Officer at its Boston facility.
The news highlights:
- Gene therapy for rare immune disorder receives Priority Review
- Intellia and Regeneron’s research collaboration expands to neurological and muscular diseases
- Dean Morris appointed as Chief Operations Officer at RoslinCT’s Boston facility
Gene therapy for rare immune disorder receives Priority Review
The US FDA has accepted the Biologics License Application and granted Priority Review for Rocket Pharmaceuticals’ (NJ, USA) investigational gene therapy, RP-L201 (marnetegragene autotemcel). The lentiviral vector-based therapy is designed to address severe Leukocyte Adhesion Deficiency-I, a rare immune disorder caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18.
“Today’s acceptance of the [Biologics License Application] by the FDA marks a significant milestone for Rocket towards our goal of delivering a one-time gene therapy to patients facing the devastating effects of severe [Leukocyte Adhesion Deficiency-I]. For these patients, survival beyond childhood is uncommon. Bone marrow transplant is currently the only treatment option, has substantial morbidity and mortality and may not be available in time for these children,” said Kinnari Patel, President and Chief Operating Officer of Rocket Pharmaceuticals. “We are incredibly grateful to the patients, caregivers and researchers who have shared this journey with us and look forward to continuing our close collaboration with the FDA during the review period as we work to bring RP-L201 to patients as quickly as possible.”
Intellia and Regeneron’s research collaboration expands to neurological and muscular diseases
Regeneron Pharmaceuticals and Intellia Therapeutics have announced an expanded research collaboration to develop in vivo CRISPR-based gene therapies focusing on neurological and muscular diseases. The existing collaboration sought to develop gene therapy products to treat hemophilia through CRISPR/Cas9-mediated transgene insertion in the liver.
The expansion of this partnership will utilize Regeneron’s proprietary antibody-targeted adeno-associated virus (AAV) vectors and Intellia’s Nme2 CRISPR/Cas9 systems, adapted for precise gene modification via viral vectors. Initially, they will research two in vivo non-liver targets, with Intellia overseeing the editing methodology design and Regeneron leading the development of targeted viral vector delivery approaches.
Christos Kyratsous, Senior Vice President of Research and Co-Head of Regeneron Genetic Medicines stated: “Regeneron has invented and preclinically validated a proprietary antibody-directed AAV approach that builds on our decades of experience in antibodies and newly developed AAV capsid engineering technologies to deliver innovative payloads across many targeted tissue types and disease settings. We’re excited to put this approach to the test in combination with Intellia’s industry-leading gene editing systems, in hopes of generating important new medicines for people with serious neurological and muscular diseases.”
Dean Morris appointed as Chief Operations Officer at RoslinCT’s Boston facility
RoslinCT, a cell and gene therapy contract development and manufacturing organization (CDMO), announced the appointment of Dean Morris as the Chief Operating Officer at their Boston facility. Morris previously held the position of Vice President of Operations and Site Head at ReciBioPharm (Stockholm, Sweden), where he oversaw biopharmaceutical ATMP development and manufacturing, including oncolytic viruses, viral vaccines, mRNA and gene therapy, and live biotherapeutic products.
“We are excited to welcome Dean to the role of Chief Operations Officer at RoslinCT,” said Patrick Lucy, President and CEO at RoslinCT Boston. “His extensive experience and successful track record in the biopharmaceutical industry make him the ideal leader to drive our operations and continue our commitment to delivering high-quality advanced therapies to patients in need.”
