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Find Clinical Drug Pipeline Developments & Deals by Arbor Biotechnologies, Inc
4DMT and Arbor will co-develop and co-commercialize up to six AAV-delivered CRISPR/Cas-based therapeutic candidates for central nervous system (CNS) diseases, including amyotrophic lateral sclerosis (ALS).
Under the terms of the agreement, Vertex Pharmaceuticals incorporated will receive rights to Arbor’s novel precision editing technology for up to three diseases.
As part of the agreement, the companies will combine the optimized delivery of Acuitas' highly validated LNP technology with Arbor’s differentiated, proprietary CRISPR gene editing technology designed for use in vivo in patients with rare liver diseases.
The collaboration leverages Arbor’s proprietary CRISPR gene-editing technology and TCR 2’s’ TRuC platform, which has demonstrated clinical activity in multiple treatment-refractory mesothelin-expressing solid tumor indications with its lead autologous program Gavo-cel.
Arbor plans to use the proceeds to advance its lead programs in liver and CNS disease into the clinic and progress a pipeline of precision editing therapeutics, while continuing to invest in its novel discovery engine to develop the next generation of gene editing technology.
Under this new partnership, Vertex will receive rights to use Arbor’s technology to research and develop ex vivo engineered cell therapies towards Vertex’s goal of generating fully differentiated, insulin-producing hypoimmune islet cells for the treatment of type 1 diabetes.
Lead Product(s):
Ex-vivo Engineered Cell Therapies
The agreement aims to utilize Arbor’s gene editing technologies to engineer hematopoietic stem cells, towards the goal of developing therapies for the treatment of blood cancers, such as acute myeloid leukemia.
Lead Product(s):
Engineered hematopoietic stem cell