CRISPR Therapeutics AG CRSP and its partner Vertex Pharmaceuticals VRTX announced new data from a phase I/II study evaluating their CRISPR/Cas9 gene-editing therapy candidate, CTX001, on 22 patients with transfusion-dependent beta thalassemia (“TDT”), as well as those with sickle cell disease (“SCD”), with follow-up of at least three months.
The gene-editing therapy candidate demonstrated a consistent and sustained response to treatment in the given patient population.
Notably, a single dose of CTX001 is being evaluated in the phase I/II CLIMB THAL-111 study for treating TDT, and in the phase I/II CLIMB SCD-121 study for severe SCD. New data from the studies showed that treatment with CTX001 resulted in all 15 patients with TDT remaining transfusion independent at the last follow-up. Meanwhile, all seven patients with SCD were free of vaso-occlusive crises through last follow-up.
Importantly, five patients with TDT and two with SCD now have follow-ups of greater than one year, indicating a stable and durable response to treatment with CTX001. All patients with TDT and SCD experienced rapid and sustained increase in their total hemoglobin and fetal hemoglobin levels through last follow-up.
The new data was presented at the annual meeting of the European Hematology Association.
Shares of CRISPR Therapeutics were up 2.7% following the announcement of the news. However, the stock has plunged 17% so far this year against the industry’s increase of 1.1%.
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We note that CRISPR Therapeutics and Vertex had entered into a strategic research collaboration in 2015 to co-develop and co-commercialize CTX001.
Meanwhile, in April 2021, Vertex increased its investment in the existing collaboration agreement with CRISPR Therapeutics for the manufacture and commercialization of CTX001. Under the amended deal, Vertex will lead the global development and future commercialization of CTX001, which it believes has significant commercial opportunity.
Enrollment and dosing are currently ongoing for both SCD and TDT studies on CTX001.
Notably, CTX001 has been granted Regenerative Medicine Advanced Therapy, Fast Track, and Orphan Drug and Rare Pediatric Disease designations by the FDA for both TDT and SCD. The European Commission has granted Orphan Drug designation as well as Priority Medicines (PRIME) designation to the gene therapy candidate for both indications.
Please note that, other than CRISPR Therapeutics, companies such as Editas Medicine EDIT, Intellia Therapeutics NTLA and Beam Therapeutics, among others, are also using the CRISPR/Cas9 gene-editing technology to develop their respective candidates for addressing various ailments.
CRISPR Therapeutics currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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