Abeona breaks ground on gene therapy manufacturing site
- Cleveland-based biopharma Abeona Therapeutics has broken ground on its new commercial gene therapy manufacturing facility, the Elisa Linton Center for Rare Disease Therapies.
- The manufacturing facility will initially produce ABO-101 and ABO-102, in development for Sanfilippo syndrome type A and B respectively, and EB-101, an autologous cell therapy for the treatment of recessive dystrophic epidermolysis bullosa.
- The 6,000 square foot center, due to be built-out and validated over the next 12 months, will also include an expanded viral vector lab.
As gene and cell therapies become a commercial reality, biotech companies and contract development and manufacturing organizations (CDMOs) are expanding their manufacturing capabilities to keep up with late-stage clinical trials and the marketplace. Examples include CDMO Brammer Bio, which completed a major expansion of its Alachua, Florida facility in September, and AveXis, which has its own cGMP manufacturing facility to produce the clinical and commercial supply of its spinal muscular atrophy (SMA) type 1 gene therapy, AVXS-101.
Abeona Therapeutics is currently working with CDMO Brammer Bio for commercial manufacturing support for clinical development of its treatment for Sanfilippo syndrome type A. This includes commercial AAV process development, scale up and assay validation for clinical programs and internal capabilities, and will support the commercial translation of Abeona's investigational drugs. This new facility has potential to bring full-scale manufacturing back in house.
"The Elisa Linton Center for Rare Disease Therapies… will be a global resource for production of gene therapies with the potential to bring new treatments to rare disease patients around the world," said Timothy J. Miller, president and CEO of Abeona Therapeutics. "It is especially fitting that this center is named for Elisa Linton, who was born with Sanfilippo syndrome, a rare terminal disease."
The facility will focus on production of ABO-101, which has completed a Phase 1/2 trial for Sanfilippo syndrome type A, ABO-102, which is in a Phase 1/2 trial for the type B form of the disease, and the Phase 3-ready recessive dystrophic epidermolysis bullosa gene therapy EB-101.
The supply chain is a vital part of the process too. Companies will also need to ensure that they have the shipping and logistics providers in place, particularly for cell therapies, in order to deliver what are complex and personalized medicines to patients potentially across the other side of the globe.
- Abeona Therapeutics Statement
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