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Taysha’s gene therapies obtained an exclusive option from the University of Texas Southwestern (UTSW) to license the worldwide rights to a clinical stage gene therapy program for CLN7 disease, a form of Batten disease in late childhood.

A first generation CLN7 therapy is currently being evaluated in a phase 1 clinical trial (NCT04737460) managed by the university, which continues to enroll patients. The first safety and efficacy data are expected by the end of the year.

“The CLN7 program is a strategic addition to our gene therapy pipeline,” said RA Session II, President, Founder and CEO of Taysha, in a statement. Press release. “The first generation construct is currently in a Phase 1 proof of concept clinical trial with two patients treated to date, and we look forward to the availability of preliminary data by the end of the year. . “

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Taysha is also expected to collaborate with UTSW to continue to develop a next-generation version of the therapy. Further advancements in the program are expected to improve potency, safety, and manufacturing ability over first-generation therapy – originally developed by Steven Gray, PhD, at UTSW Medical Center and Taysha’s chief scientific advisor – which was funded by CLN7 patient advocacy groups Miracle de Mila Foundation and Latte hope.

Completion of the next-generation therapy is expected by the end of the year, with commercial grade material expected in 2022, in time to begin a study to evaluate the advanced version.

“With human proof-of-concept clinical data to benchmark, we plan to advance a next-generation construct in a pivotal trial scheduled for 2022, which is expected to improve potency, safety, packaging efficiency and manufacturability. compared to the first generation construction. “said Session II.

CLN7 disease is a progressive neurodegenerative disease caused by mutations in the MFSD8 gene that alters lysosomes, the compartment in cells that store and break down cellular waste.

MFSD8 Mutations lead to the buildup of wastes inside cells, especially nerve cells, leading to seizures, speech disturbances, vision loss, and motor and mental regression, with onset around age from 2 to 5 years. CLN7 therapy contains a functional copy of MFSD8 gene, delivered to cells by a harmless adeno-associated virus.

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natural history study

The phase 1 program is based on preclinical data in mice lacking MFSD8. The intrathecal (into the spinal canal) administration of the first generation treatment at the highest dose in younger CLN7 mice resulted in MFSD8 gene activity in all tissues examined.

Six to nine months after the injection, the mice were found to have almost normalized their impaired behavioral performance in open field and rotaroding, more than doubled their median life expectancy and maintained healthy body weight. The therapy has been found to be safe and well tolerated.

“Encouraging preclinical data generated in relevant rodent models suggest that the first generation construct has the potential to reduce overall disease pathology, preserve motor function, and ultimately prolong survival,” said declared Session II.

The open-label Phase 1 trial is currently underway at Children’s Hospital in Dallas, Texas and will evaluate first-generation therapy in up to four patients, ages 1 to 18. The primary objective of the study is safety and tolerability, and secondary efficacy results include assessments of motor function, psychological and quality of life as well as an overall clinical impression measuring the overall severity of the condition. disease.

So far, one patient has received a low dose (5 × 1014 vector genomes or vg) and another received the highest dose (1.2 × 1015 vg). UTSW is still seeking patients for the highest dose.

“We are also pleased to announce our grant to Batten Hope, the leading nonprofit patient advocacy organization with CLN7 disease, to support patient awareness, disease education and newborn screening initiatives. “said Session II. “We believe that a gene therapy approach has the potential to address a significant unmet need in approximately 4,000 patients worldwide.”

Gina Hann, Founder, President and Treasurer of Batten Hope, added, “Our mission is to support families with children with end-stage and rapidly progressive neurodegenerative diseases like CLN7. We are honored to receive Taysha’s support to raise awareness, increase newborn screening, and help patients access potentially life-changing treatments that offer hope and therapeutic advancements for conditions with significant unmet needs.

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