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Cambria Receives $5 Million Grants for Neurodegenerative Disease Drug Discovery and Preclinical Development Program

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Cambria Biosciences has announced that it has been awarded five grants from multiple organizations totaling $5.0 million.

The grant funding will support the company’s drug discovery programs for neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease), muscular dystrophy and Parkinson’s disease.

“We are very grateful to receive these awards from leading organizations involved in the search for new treatments for serious neurological disorders,” said Donald Kirsch, PhD, Senior Vice President for Drug Discovery at Cambria Biosciences.

“Because of the rigorous peer-review process employed by these organizations, we are also very pleased with the scientific recognition and support these awards bring to Cambria’s approach of developing new disease models to identify novel small molecules that suppress disease pathology in innovative ways,” Kirsch added.

Cambria has received three grants to support the company’s ALS research and development programs, led by Dr. Kirsch:

• The National Institute of Neurological Disorders and Stroke (NINDS) granted Cambria a Phase 1 SBIR award to identify small molecule compounds that protect against cellular damage caused by the mutant SOD1 protein.

• The ALS Association (ALSA) awarded Cambria a multi-year, multi-million dollar grant as part of a milestone-driven program that will include lead optimization, pharmacology, and preclinical transition to an Investigational New Drug Application for novel compounds that block protein aggregation and resulting cellular damage.

• The ALS Therapy Alliance will fund a project to test orally available small molecule compounds discovered by Cambria researchers for efficacy in preclinical animal models of ALS.

Cambria’s ALS research program will be conducted in collaboration with Dr. Richard Morimoto and Dr. Richard Silverman at Northwestern University, Dr. Robert Ferrante at the Boston University Medical Center and Bedford VA Hospital, and other investigators.

ALS and muscular dystrophy are representative of a broad group of neuromuscular diseases, many of which have a defined underlying genetic basis that may offer clues for pharmacological treatments that may improve neuromuscular function.

The Jain Foundation has awarded Cambria a grant to establish a drug discovery program for type 2B limb girdle muscular dystrophy (LGMD2B). Dr. Mohan Viswanathan, a senior scientist at Cambria, will establish and implement a whole-organism screen to discover small molecule compounds that may correct the cellular dysfunction caused by mutations in the dysferlin gene that are responsible for human LGMD2B and Miyoshi Myopathy.

“Neurodegenerative diseases are among the greatest unmet needs in medicine,” said Leo Liu, MD, President and CEO of Cambria Biosciences. “We are therefore delighted to be part of a new paradigm of cooperation between biotechnology companies and nonprofit organizations to attack these debilitating and life-threatening disorders.”