Dive Brief:
- Rare diseases biotech Abeona Therapeutics opened its new commercial GMP manufacturing facility for gene and cell therapies in Cleveland, Ohio on May 31, naming it the Elisa Linton Center for Rare Disease Therapies.
- The 6,000-square-foot facility will manufacture clinical and commercial grade products for Abeona's pipeline products, including EB-101 for recessive dystrophic epidermolysis bullosa and ABO-102 for Sanfilippo syndrome.
- The center will also include an adeno-associated virus vector lab, and aims to meet the chemistry, manufacturing and controls (CMC) needs for commercial development. The next step for Abeona will be the completion of an additional 20,000-square-foot facility to meet future commercial demand.
Dive Insight:
As cell and gene therapies get closer to the market, there is a growing need for production capabilities. While some companies are reaching out to specialist manufacturers, Abeona Therapeutics has decided to keep it firmly in house. Building of the facility began in October 2017.
"Our development of internal manufacturing capabilities bolsters our position for commercial readiness as we continue to execute on our vision to bring these therapies to the patient communities that need them," Abeona CEO Carsten Thiel in a statement.
According to Maury Raycroft, an equity analyst at Jefferies who attended the launch, the new facility "will enable [Abeona] to manage commercial supply for diverse products while keeping margins and timelines in check."
Abeona's EB-101 for recessive dystrophic epidermolysis bullosa is ready to move into Phase 3, and the new facility puts Abeona in a stronger position to meet the commercial demand for the first two years, which Raycroft estimates to be up to a hundred patients per year. The planned initial expansion will provide scale-up space.
ABO-102 is in a Phase 1/2 trial for Sanfilippo syndrome, and the new facility includes bioreactors for baculovirus-based production of ABO-102, allowing for continuous production to meet the commercial demands, according to Raycroft.
Abeona has regenerative medicine advanced therapy designations for EB-101 and ABO-102. This designation, for cell therapies in development for serious or life-threatening diseases, can speed such products to the market.
In 1998, four-year-old Elisa Linton was diagnosed with mucopolysaccharidosis IIIB, a rare genetic disorder also known as Sanfilippo syndrome type B. The disorder damages the central nervous system, leading to speech and behavior problems beginning at around age two, as well as loss of motor and cognitive skills after age 10.
It is fatal, and Linton died at 22. Her parents raised money and awareness about the disorder, creating the Sanfilippo Children's Research Foundation. The new Abeona site is named after their daughter.