Manufacturing deal prepares Rett gene therapy for potential U.S. launch
Taysha expands Catalent partnership as pivotal testing of TSHA-102 continues
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Taysha Gene Therapies is preparing for the potential commercial launch of TSHA-102, its experimental gene therapy for Rett syndrome now in pivotal clinical testing.
To support the potential launch, Taysha recently announced a commercial supply agreement with Catalent, a pharmaceutical manufacturing company. Under the agreement, Catalent will be the primary commercial manufacturer of TSHA-102 if the therapy receives approval from the U.S. Food and Drug Administration (FDA).
“As we continue to advance TSHA-102, establishing long-term commercial manufacturing capacity is a critical component of our launch readiness strategy,” Sean P. Nolan, chairman and CEO of Taysha, said in a company press release. “Catalent’s deep gene therapy expertise and proven commercial manufacturing capabilities make them an ideal partner as we expand our partnership to further strengthen our commercial infrastructure with a reliable, scalable supply framework.”
Catalent expands role in TSHA-102 manufacturing
Catalent has supported the development of TSHA-102 since 2020. The company has worked on more than 90 gene therapy programs, including multiple commercially available products. Under the new agreement, Catalent will manufacture and supply commercial TSHA-102 at its FDA-licensed gene therapy facility in Harmans, Maryland.
“At Catalent, our priority is to support innovators who are redefining what’s possible for patients with rare diseases,” said David McErlane, biologics group president for Catalent. “Our partnership with Taysha brings together deep [adeno-associated virus] expertise and a shared commitment to advancing a potential therapy for Rett syndrome—a condition with significant unmet need. We are proud to help enable the development and future supply of therapies that have the potential to make a meaningful difference for patients and their families.”
Rett syndrome is a genetic disorder chiefly caused by mutations in the MECP2 gene. TSHA-102, which is administered by injection into the spinal canal, is designed to deliver a functional version of MECP2 to nerve cells, thereby addressing the genetic root cause of Rett syndrome. Early clinical testing suggested that TSHA-102 treatment was associated with improvements in communication abilities, social skills, and motor function among women and adolescent girls with Rett syndrome.
Taysha is now running the pivotal Phase 3 portion of a Phase 1/2/3 clinical trial called REVEAL (NCT05606614) to evaluate the therapy’s efficacy and safety. As of July, the trial was fully enrolled, with 17 girls and young women, ages 6 to 21, who have a confirmed genetic diagnosis of Rett syndrome. All participants receive TSHA-102, and the trial’s main goal is to determine how many participants gain or regain at least one of 28 developmental milestones that people with Rett syndrome typically do not achieve without treatment.
If the results are positive, Taysha hopes to use data from REVEAL to support a biologics license application (BLA) — a formal request asking the FDA for permission to market the therapy. Taysha and Catalent are already preparing to meet manufacturing requirements associated with the BLA process.
“With BLA-enabling Process Performance Qualification activities underway, we believe we have established the manufacturing foundation necessary to support the strong demand we expect following the potential launch and commercialization of TSHA-102,” Nolan said.
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