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FDA Accepts Investigational New Drug Application for CRISPR/Cas9-Based Sickle Cell Disease Therapeutic Candidate Developed Under Collaboration with Intellia Therapeutics

Posted on March 31, 2020

Intellia Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted the Investigational New Drug (IND) application submitted by its collaborator, Novartis, for a CRISPR/Cas9-based engineered cell therapy for the treatment of sickle cell disease (SCD).

This Phase 1/2 clinical trial will begin investigating OTQ923 in adult patients with severe complications of SCD. OTQ923 is a SCD treatment based on genome editing of hematopoietic stem cells (HSCs), using CRISPR/Cas9 RNA guides identified through Intellia’s cell therapy research collaboration with Novartis. This therapeutic approach results in highly targeted editing of the HSC’s DNA to induce fetal hemoglobin (HbF) expression. The edited cells are returned to the patient, where the expression of HbF is expected to reduce the deleterious effects of sickle hemoglobin (HbS). Novartis’ IND application triggered a milestone payment to Intellia, and the company is eligible to receive additional downstream success-based milestones and royalties.

“We are pleased to have worked alongside our colleagues at Novartis to achieve this important milestone, which moves this CRISPR/Cas9-based engineered cell therapy into the clinic, with the potential to significantly impact the lives of patients who suffer from sickle cell disease,” said Intellia Chief Operating Officer and Executive Vice President Andrew Schiermeier, Ph.D. “Our research with Novartis over the past five years has laid the groundwork for the development of next-generation CRISPR/Cas9-based cell therapies for patients. Intellia looks forward to Novartis’ efforts to advance other targets that were selected to develop as additional CRISPR/Cas9-based cell therapy products.”

Read the full press release here

Source: Intellia Therapeutics

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