RARE Daily

Taysha Transfers Rights to Waylaid Gene Therapy Programs

February 16, 2024

Rare Daily Staff

Taysha Gene Therapies, which last year stopped work on serval gene therapy programs to extend its cash runway, said it has transferred the rights to several programs to allow their development to continue.

In November, the company terminated its existing loan and security agreement and entered into a new loan and security agreement that allows it to transfer intellectual property for several of its deprioritized programs to third parties more efficiently. The company’s new loan and security agreement also extended its cash runway into 2026.

Taysha had appeared to solve a translational research program for promising ultra-rare gene therapies developed at universities, often with the support of patient organizations, and was poised to bring them through development. It built a large pipeline by gaining rights to these therapies.

But the difficult financial environment for biotechs in recent years stymied the company’s vision and it was forced to pare back its ambitions, much to the frustration of patient groups that had counted on the company advancing these therapies to patients. As Taysha sought to extend its runway, the fate of many programs appeared destined to sit on shelves.

Taysha said it has initiated transfer of its U.S. Food and Drug Administration application to begin human clinical trials clinical trial materials for TSHA-120, the company’s experimental gene therapy for giant axonal neuropathy (GAN) to a clinical trial collaborator National Institute of Neurological Disorders and Stroke to allow an opportunity for continued clinical evaluation of TSHA-120 in GAN. Taysha also said it entered into discussions with the originating advocacy organization regarding TSHA-120 in an effort to transfer rights back to the advocacy organization to move the program forward.

The company transferred rights to TSHA-101 in GM2 gangliosidosis back to Queen’s University. It transferred TSHA-104 in SURF1-associated Leigh syndrome and TSHA-112 in adult polyglucosan body disease (APBD) back to the originating institutions. And it provided investigational clinical trial material for TSHA-118 in CLN1 to support an individual-patient investigator-initiated investigational new drug request from RUSH University Medical Center for the treatment of a patient with CLN1 disease.

“Creating options for these programs has been a focus since the company completed a management change in December 2022, and the new loan and security agreement afforded the flexibility to implement certain opportunities,” said Sean Nolan, chairman and CEO of Taysha. “As we continue to focus on advancing our lead TSHA-102 program for the treatment of Rett syndrome, we are pleased that we can ensure these programs are provided to the right advocates, clinicians, and scientific experts who can potentially move these programs forward for the benefit of patients.”

Photo: Sean Nolan, chairman and CEO of Taysha

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