CAMBRIDGE, Mass. — Elpida Therapeutics and Catalent Inc. have entered a strategic partnership to support late-stage manufacturing for Elpida’s lead gene therapy program for Spastic Paraplegia Type 50, or SPG50.
Elpida, a nonprofit biotechnology company based in Cambridge, is developing gene therapies for ultra-rare diseases. Catalent, based in Tampa, Fla., provides development and manufacturing services for treatments across a range of therapeutic areas.
Under the agreement, Catalent will support manufacturing of Elpida’s AAV9 gene therapy for SPG50. Catalent also will receive exclusive manufacturing rights to Elpida’s other pipeline adeno-associated virus gene therapy programs.
SPG50 is an ultra-rare neurodegenerative disorder caused by AP4M1 mutations. The disease begins in infancy and, if untreated, can lead to cognitive impairment, epilepsy and progressive paralysis by early adulthood.
“This collaboration with Catalent represents a critical step in ensuring continued patient access to our SPG50 gene therapy program. Without this support, the final patient planned for treatment by the end of this year could have marked the end of this therapy’s availability,” said Terry Pirovolakis, Founder and Chief Executive Officer, Elpida Therapeutics. “Together, we are building a path to reach all patients living with SPG50 and, ultimately, other devastating ultra-rare diseases.”
Catalent said it will use its UpTempo AAV manufacturing platform to support the program. The platform includes the company’s proprietary HEK293 cell line, pre-validated off-the-shelf AAV plasmids and pre-qualified analytical assays.
The companies said the arrangement is intended to accelerate production of research and GMP-grade material needed for SPG50 process validation and to support Elpida’s planned Biologics License Application submission.
“This partnership reflects Catalent’s commitment to applying our broad gene therapy manufacturing expertise and Patient First approach to programs with significant unmet need,” said David McErlane, Biologics Group President for Catalent. “By leveraging our broad expertise in end-to-end AAV capabilities, we look forward to supporting Elpida’s SPG50 program through late-phase manufacturing so it can advance toward regulatory submission.”
Catalent said it has worked with clients on more than 80 gene therapy programs, including four commercial programs, spanning early development, clinical stages and commercialization.
