Treeway is collaborating with the Cedars-Sinai Board of Governors Regenerative Medicine Institute for the development of AAV5-GDNF (TW002) gene therapy for amyotrophic lateral sclerosis (ALS).

Treeway, a biotech company founded by two ALS patients, obtained an exclusive license agreement from uniQure for the use of AAV5-GDNF earlier this year. GDNF has a promising clinical potential in ALS, but optimization of delivery to the site of action is required. AAV5-GDNF is a gene therapy with a novel administration modality for clinical use and is Treeway’s second product in development (TW002).

To advance the development of AAV5-GDNF gene therapy, Treeway will work with a research team led by Clive Svendsen, Ph.D., director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute in Los Angeles. Svendsen is an expert in human neural stem cell biology and has specifically focused on using neural stem cells, modified to release GDNF, to treat ALS.

Treeway and Cedars-Sinai applied for a TREAT ALS Drug Development Contract and received the $500,000 award. The contract, an initiative of The ALS association, was designed to rapidly bring promising potential therapeutics closer to clinical trials. The TREAT ALS Drug Development grant will be used to start a preclinical study in which the safety and efficacy of TW002 in an ALS rat model will be assessed further. The preclinical studies are an important step in the development of TW002 for ALS.