After a two-month 32% swoon in the value of the BioWorld Gene Therapy index, it has managed to recover 6% of that loss since the end of September.
Contributing to the recovery was Menlo Park, Calif.-based Adverum Biotechnologies Inc., whose shares (NASDAQ:ADVM) jumped almost 80% in the period. The increase was catalyzed by the company reporting positive progress of ADVM-022 in the first cohort of patients in the phase I Optic trial. Administered as a single, intravitreal shot, ADVM-022 deploys a proprietary vector capsid (AAV.7m8) carrying an aflibercept (Eylea, Regeneron Pharmaceuticals Inc.) coding sequence under the control of an expression cassette designed by the company.
In its third-quarter financial results, the company’s CEO, Leone Patterson, commented on the results noting that “this is the first time that a single intravitreal injection gene therapy has provided sustained efficacy for these patients, who previously required frequent ocular anti-VEGF injections to maintain their vision.”
Adverum has progressed to dosing patients in a third cohort of the Optic study with ADVM-022 at a dose of 2x1011 vg/eye using prophylactic steroid eye drops instead of prophylactic oral steroids. In the first quarter of next year, enrollment is expected to start in the fourth cohort of patients at a dose of 6x1011 vg/eye.
Positive clinical progress for Pittsburgh-based Krystal Biotech Inc., a gene therapy company developing and commercializing treatments for patients suffering from rare dermatological diseases, saw its share value (NASDAQ:KRYS) vault 47% in the period.
The company reported a final update from its phase I/II trial of bercolagene telserpavec (B-VEC), its lead product candidate to treat dystrophic epidermolysis bullosa, an incurable skin blistering condition caused by a lack of collagen in the skin, showing that B-VEC was well-tolerated, with no related adverse events reported. Nine out of 10 of the wounds closed completely following initial administrations of B-VEC, with the average time to wound closure at 17.4 days.
Going forward, the company reported that, following alignment with the FDA on the commercial process and engineering run, it plans to initiate manufacturing of phase III clinical material and begin the pivotal trial for B-VEC in the first quarter next year.
Positive gene editing
Shares of Zug, Switzerland-based Crispr Therapeutics AG (NASDAQ:CRSP) also spiked on exciting data from two patients, one with beta-thalassemia and the other sickle cell disease, treated with its CRISPR/Cas9 gene-editing therapy, CTX-001, in ongoing phase I/II trials. Two treated patients successfully engrafted and demonstrated an initial safety profile consistent with myeloablative busulfan conditioning and autologous hematopoietic stem cell transplant. CTX-001 is being developed under a co-development and co-commercialization agreement between Crispr Therapeutics and Vertex Pharmaceuticals Inc. established in 2015. The companies equally share all research and development costs and profits worldwide. Crispr’s shares are trading up 45% in the period.