Sirion and Denali Collaborating to Develop Gene Therapies for Neurodegenerative Diseases

José Lopes, PhD avatar

by José Lopes, PhD |

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Veterans with Parkinson's

Sirion Biotech and Denali Therapeutics have agreed to partner on the development of gene therapies for Parkinson’s disease and other neurodegenerative disorders.

According to the license and collaboration agreement, the two companies will pursue the development of harmless, next-generation adeno-associated viral vectors (AAV) able to cross the blood-brain barrier — a semipermeable membrane that protects the brain against the external environment.

Besides Parkinson’s, these potential gene therapies will also target Alzheimer’s, amyotrophic lateral sclerosis, and other diseases of the central nervous system — brain and spinal cord.

“This ground-breaking collaboration will help Denali Therapeutics to increase the availability of protein therapeutics in the brain, and to quickly enter clinical trials with efficient, safe and scalable therapeutic candidates,” Christian Thirion, PhD, Sirion’s founder and CEO, said in a press release.

According to Sabine Ott, PhD, Sirion’s vice president of business development and licensing, the company believes that through its partnership with Denali, AAV-based gene therapies can reach the market “in the fastest possible way, providing novel treatment options to many millions of patients.”

Specifically, the aim is to create new and modified AAV capsids (the protein shell of a virus) that are safe and have greater specificity and high efficiency to deliver therapeutic levels of medicines to the brain. AAV vectors are regarded as the most promising gene delivery system for therapies.

Sirion’s collaboration with Dirk Grimm, PhD, a professor of viral vector technologies at Heidelberg University Hospital in Germany, will play a key part in the research.

“By contributing and harnessing our unique and proprietary expertise in the engineering and high-throughput in vivo screening of AAV capsid libraries, we will significantly accelerate this joint endeavour and increase our chances to realize its pivotal aims,” Grimm said.

The main terms of the agreement between Germany-based Sirion and San Francisco-based Denali include development expenses, milestone payments to Sirion, and royalties from future products or therapies.

According to Alexander Schuth, Denali’s chief operating officer, the company is “excited to partner with Sirion” and combine its “expertise around the blood-brain barrier and neurodegenerative diseases with Sirion’s leading expertise on viral vectors for gene therapy to enable new treatments for diseases of the brain,” further saying that the partnership will add a new therapeutic modality to the company’s portfolio and is complementary to other programs from Denali.

In October 2018, Denali and Centogene announced a collaboration to identify and recruit Parkinson’s patients carrying mutations in the LRRK2 gene for future clinical trials. Mutations in LRRK2 are a well-known genetic cause of Parkinson’s. These patients will participate in trials of Denali’s LRRK2 inhibitor therapy program, which includes DNL201 and DNL151.