Fabrice Cabaud Abeona Therapeutics Inc. ( NASDAQ: ABEO ) is gearing up for a Biologics License Application [BLA] resubmission in the 2nd half of 2024. This would be regarding a resubmission of the BLA filing of Pz-cel for the treatment of patients with recessive dystrophic epidermolysis bullosa [RDEB].
There is a good chance that the next filing should have a better shot at FDA approval being achieved. The reason I believe this to be the case is because the only issue noted in the Complete Response Letter [CRL] was that the Chemistry, Manufacturing and Control [CMC] portion of the application was not sufficient. With no issues noted about safety/efficacy of Pz-cel, plus no mention of needing another study, there is a very good chance that it will receive U.
S. marketing approval of this therapy this next time around. Besides this, there was a collaboration agreement established with Beacon Therapeutics to obtain non-exclusive rights to the AAV204 Capsid platform that Abeona has.
The company can use this capsid for up to 5 gene therapy ophthalmic targets and then up to 4 additional gene targets. This deal doesn't have any money yet in place, but if a non-exclusive agreement option is taken, then it is possible to receive some cash based on this agreement. What puts Abeona in a good position already is that it has already licensed out other gene therapies in its pipeline.
For instance, it licensed its MPSIIIA gene therapy program to Ultragenyx Pharmaceutical ( RARE ) and then th.