Coave wins grant to advance gene therapy CTx-TFEB for ALS
CTx-TFEB is designed to promote autophagy for all types of ALS
Coave Therapeutics has received a grant from the ALS Association to support the development of its gene therapy candidate CTx-TFEB for amyotrophic lateral sclerosis (ALS).
The grant, made through the Lawrence and Isabel Barnett Drug Development Program, is part of the $2.9 million the ALS Association has granted to six organizations committed to advancing novel therapies for ALS.
It will help the company conduct proof-of-concept preclinical studies of CTx-TFEB that may support moving the treatment into ALS clinical trials in the future.
“Our goal is to establish a robust preclinical proof-of-concept, laying a strong foundation for advancing to the clinic in the near future,” Lolita Petit, PhD, Coave’s chief scientific officer, said in a company press release. “Together, we are now on the brink of a breakthrough that promises hope and progress for patients impacted by ALS.”
Importance of moving promising treatments from laboratory to clinical testing
“Getting promising treatments out of the laboratory and into clinical testing as quickly as possible is key to making ALS a livable disease until we can cure it,” said Kuldip Dave, PhD, senior vice president of research at the ALS Association.
The molecular mechanisms that cause ALS aren’t fully known, but virtually all cases are marked by the accumulation of protein clumps. This is in part due to a dysregulation in autophagy — the process by which cells recycle abnormal or defective molecules, which usually happens inside specialized compartments called lysosomes.
It’s anticipated that boosting autophagy could promote the clearance of these toxic protein clumps in ALS nerve cells, thereby slowing or halting neuronal degeneration and disease progression.
The transcription factor TFEB has emerged as a key regulator of the autophagy lysosomal pathway. This protein is found at lower levels in the brains of ALS patients, and activating it is believed to be a promising approach to increase autophagy and remove the harmful protein clumps.
CTx-TFEB is a novel genetic therapy product designed to deliver the TFEB gene specifically to nerve cells in the brain and spinal cord. The gene sequence is transported inside an adeno-associated virus that does not cause disease in humans. The virus was modified with Coave’s proprietary ALIGATER platform to carry certain molecules that make the vector more specific to nerve cells.
“Modulating autophagy using TFEB gene therapy is an exciting approach to promote the clearance of accumulated toxic material in affected neurons, halt motor neuron degeneration, and preserve muscular function in people with ALS,” Petit said.
The therapy is expected to activate autophagy in ALS patients regardless of their genetic subtype, potentially serving as a therapy for all types of ALS.
“We are proud to help drive the crucial transition from preclinical to clinical development for potential new ALS therapies through our Lawrence and Isabel Barnett Drug Development Program,” Dave said.
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