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4D Molecular Therapeutics, Arbor Biotechnologies Partner on Nerve Disorder Gene Therapies

NEW YORK – 4D Molecular Therapeutics and Arbor Biotechnologies announced on Wednesday a strategic partnership to develop and commercialize gene editing therapies for rare and common central nervous system (CNS) diseases with high unmet medical needs.

Under the partnership, the financial details of which were not disclosed, the partners will collaborate on up to six adeno-associated virus (AAV)-delivered CRISPR-Cas-based therapeutic candidates, evenly sharing all costs and profits based on mutually agreed plans.

Cambridge, Massachusetts-based Arbor will lead research, development, and if approved, commercialization efforts on the first candidate therapy, which aims to address a molecular target implicated in amyotrophic lateral sclerosis (ALS).

ALS, also called Lou Gehrig's disease, is a progressive neurodegenerative disease in which the motor neurons atrophy and die, causing patients to lose their abilities to speak, move, eat, and eventually, breathe. Despite multiple approved treatments available for ALS and its symptoms, the disorder currently lacks a cure.

Emeryville, California-based 4DMT will then take lead on the second candidate therapy, the target and associated disease of which will be disclosed at a later date.

Arbor seeks to develop genomic editors for a wide range of genetic diseases via its proprietary artificial intelligence and machine learning-driven discovery engine, although it focuses primarily on liver and CNS-related conditions. The company also recently inked an agreement with Ginkgo Bioworks for the use of that company's technology to further identify and optimize genetic editors.

4DMT has applied its Therapeutic Vector Evolution platform to designing targeted AAV vectors used to help treat ophthalmologic, pulmonologic, and cardiologic disorders including wet age-related macular degeneration and cystic fibrosis. The partnership will also leverage the company's AAV product design and engineering, manufacturing, clinical, and regulatory development expertise.

"CNS disorders include some of the most devastating diseases, many of which have a genetic origin," Arbor CEO Devyn Smith said in a statement. "The technology to effectively edit the underlying genetic mutations within these diseases did not exist until an AAV-compatible genomic editing technology was developed. Utilizing our expansive toolbox of AAV-compatible genomic editors, we aim to advance the development of potential lifesaving therapies for CNS diseases."

"The recent landmark [US Food and Drug Administration] approval of the world's first CRISPR-based treatment is a revolutionary event for genetic medicines and the biotech industry," said David Kirn, 4DMT's cofounder and CEO. "Gene editing technology continues to rapidly advance, and we believe the safe and efficient delivery of these potentially transformative therapies can address CNS diseases of high unmet need."