CRISPR Therapeutics AG (NASDAQ: CRSP) shares are rising on the financial charts today as the market prepares to open. CRISPR Therapeutics stock has appreciated by 8.78%, reaching $42.35 at last check during the pre-market trading session. The rise in CRSP stock is following a significant development in a clinical trials.
CRISPR Therapeutics (CRSP) today announced the successful conclusion of the advisory meeting held by the U.S. Food and Drug Administration (FDA). The meeting was conducted by FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee. It was convened to deliberate on exagamglogene autotemcel (exa-cel), a treatment designed for individuals aged 12 and above suffering from recurrent vaso-occlusive crises (VOCs) due to sickle cell disease (SCD).
Notably, exa-cel stands as the inaugural therapeutic solution to emerge from the strategic collaboration between CRISPR Therapeutics and Vertex Pharmaceuticals. Panel advisors assessed that CRISPR Therapeutics could undertake an evaluation of potential safety risks associated with their gene therapy for sickle cell disease following regulatory approval.
The partnership between CRISPR Therapeutics and Vertex was initiated in 2015 with a primary focus on leveraging CRISPR/Cas9 technology for the discovery and development of novel treatments targeting the fundamental genetic underpinnings of various human diseases. Exa-cel is the first fruit borne from this collective research endeavor.
Under a revised collaboration agreement, Vertex has assumed leadership in the global phases of development, manufacturing, and commercialization of exa-cel, with program expenses and proceeds being equitably divided worldwide, at a 60/40 ratio with CRISPR Therapeutics. In the event of therapy approval, Vertex has proposed a 15-year monitoring period for patients to scrutinize the therapy’s safety outcomes.
Sickle cell disease, an inherited red blood cell disorder, induces an abnormal sickle shape in the cells due to anomalous hemoglobin levels in the body. Vaso-occlusive crises transpire when these sickled red blood cells obstruct blood flow to the extent that tissues undergo oxygen deprivation, leading to severe pain.
The advisory panel members have underscored the importance of a 15-year follow-up period for the purpose of acquiring real-time data on the therapy’s efficacy, which incorporates the innovative gene-editing CRISPR technology. Analysts have expressed optimism regarding the prospects of this pioneering therapy, which is the first of its kind to undergo FDA review, and they anticipate regulatory approval by December 8.