SIRION Biotech, Denali Therapeutics to Develop New AAV Vectors to Help Treat ALS

SIRION Biotech and Denali Therapeutics have signed a new licensing agreement to develop a new generation of adeno-associated virus (AAV) vectors, harmless viruses used as vehicles to deliver gene therapies, to allow therapies to reach into the central nervous system (CNS) of people  with amyotrophic lateral sclerosis (ALS).

The AAV vectors may also be used to carry therapies for other neurodegenerative diseases, including Parkinson’s and Alzheimer’s disease.

“This groundbreaking 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, chief technology officer of SIRION, said in a press release.

Sabine Ott, SIRION’s vice president of business development and licensing, said: “We believe that through our partnership with Denali, CNS-directed AAV-based gene therapies can reach the market in the fastest possible way, providing novel treatment options to many millions of patients worldwide suffering from devastating neurodegenerative diseases such as Parkinson’s, Alzheimer’s, and ALS.”

The adeno-associated virus (AAV) is a common, naturally occurring and non-infectious virus used as an effective gene therapy delivery vehicle to take therapies into the brain. For that, AAV needs to pass the blood-brain barrier, a key natural protection against harmful invaders entering the brain.

Advances in AAV vector design proved AAV enabled widespread gene delivery into the CNS (the brain and spinal cord), making the technology particularly useful for ALS and other neurological diseases.

Denali Therapeutics and SIRION Biotech, together with Dirk Grimm, PhD, an expert in AAV biology, will combine their expertise to develop the next generation of AAV vectors.

They will work to develop new and optimized virus with more efficient capsids — the outer shell of the gene therapy vector, which allows it to be delivered safely and with high specificity and efficiency into the brain. This will ensure that the gene therapy-carrying virus delivers the therapy in the CNS at therapeutic levels to effectively treat patients.

“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 endeavor and increase our chances to realize its pivotal aims,” said Grimm, a professor at University Hospital Heidelberg, Germany.

“We are excited to partner with SIRION Biotech and combine our 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. This partnership will add a new therapeutic modality to our portfolio and is complementary to our existing biotherapeutic and small molecule programs,” said Alexander Schuth, COO of Denali Therapeutics.