Giant axonal neuropathy (GAN) is a rare, autosomal recessive neurodegenerative disorder, characterized by a spectrum of symptoms and a challenging prognosis. Now, a study published recently in the New ...
(RTTNews) - Clinical-stage gene therapy company Taysha Gene Therapies, Inc. (TSHA), Tuesday announced that the company will discontinue the development of its TSHA-120 program in evaluation for the ...
Type B end-of-Phase 2 meeting with U.S. Food and Drug Administration (FDA) provided additional clarity for TSHA-120 for the treatment of giant axonal neuropathy (GAN) ultra-rare disease program -FDA ...
It is the first time a gene therapy has been administered directly into the spinal fluid, allowing it to target the motor and sensory neuron The researchers found that with increasing dose levels, the ...
Gene therapy delivered for the first time via lumbar puncture showed promise of benefit in slowing progression of giant axonal neuropathy (GAN) in a phase I clinical trial. The slope of change in ...
An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy (GAN), was well tolerated and showed signs of therapeutic benefit ...
Taysha Gene Therapies halted the development of one of its gene therapies after discussions with the Food and Drug Administration, the Dallas-based biotechnology company announced Tuesday. Another ...
Researchers from the UCLA Health Jonsson Comprehensive Cancer Center show for the first time that a gene usually linked to giant axonal neuropathy, a rare and severe neurological condition, also plays ...
Following Type C meeting feedback from the U.S. FDA, Taysha is discontinuing development of TSHA-120 in GAN due to challenges with study design feasibility for potentialBiologics License Application ...