- Gene therapy developer Abeona Therapeutics has linked up with CDMO Brammer Bio for commercial manufacturing support as it moves forward with clinical development of its treatment for Sanfilippo syndrome type A.
- Brammer Bio, which has been busy building out its capabilities in gene therapy manufacturing, will assist Abeona with process development for commercial adeno-associated viral (AAV) vectors, as well as with assay validation.
- In addition to the collaboration, Abeona also announced it had appointed a new head for cell-therapy manufacturing and a director of AAV manufacturing.
In May, Abeona reported top-line data from its Phase 1/2 trial of ABO-102, a candidate for the treatment of Sanfilippo syndrome type A (also known as MPS IIIA). Results showed a positive dose response in the central nervous system in two patients.
ABO-102 delivers an AAV viral vector to the body, introducing a corrective copy of the gene responsible for the development of MPS IIIa disease.
The collaboration with Brammer Bio will help prepare for potential commercial translation of ABO-102.
"Our autologous cell and gene therapy programs continue to demonstrate remarkable effects in clinical trials of patients with life-threatening diseases with no treatment options, and we are pleased to collaborate with Brammer Bio for commercial AAV process development, scale up and assay validation for clinical programs and internal capabilities," said Timothy Miller, CEO of Abeona.
Abeona plans to detail more of its manufacturing strategy and plans for ABO-102 at its first "R&D Day," scheduled for October 11.
In July, the biotech secured a green light from the Food and Drug Administration to move ahead with a Phase 3 study of its gene therapy candidate for the treatment of epidermolysis bullosa. No drugs are currently approved for the rare skin condition and a treatment from Amicus Therapeutics Inc recently failed in a late-stage trial, leaving the field just as desperately in need of treatment options as before.