Columbia Technology Ventures
Gene Therapy
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This technology is a collection of optimized therapeutic prime-editing strategies targeting four common splicing mutations in the peripherin‐2 gene (PRPH2) that are associated with various retinal degenerations.

This technology is a gene therapy platform to reactivate two transcriptional factors, Islet1 and Lhx3, for increased resilience against ALS degeneration in motor neurons.

This technology is a nucleic acid-based polymer system that can be engineered to target the expression of specific cancer-associated genes for therapeutic benefit.

This technology is a variant, mutant, or modulator of the synaptic chaperon protein Hspa8 which potentiates neurotransmission to suppress spinal muscular atrophy (SMA) phenotypes with further applications in other neurogenerative diseases including Amyotrophic Lateral Sclerosis (ALS) and tau-related dementias.

This technology is a computational algorithm for code prediction and target site matching of RNA-binding pentetricopeptide repeat (PPR) proteins called PPRDecoder.

This technology is a gene therapy which delivers CRB1 isoforms to their cell-specific localizations, for the treatment of retinopathies caused by CRB1 mutations.

This technology describes compositions which enhance the potency and stability of nucleic acid therapeutics for the treatment of diseases or conditions.

This technology is a generally applicable gene therapy approach that restores metabolic dysregulation and treats retinal degenerative diseases regardless of genetic variation.

This technology is a method of reprogramming cellular metabolism to treat neurodegenerative disorders by downregulating catabolism and upregulating anabolism in cells.

This technology utilizes anti-CRISPR proteins for efficient spatial and temporal control of gene editing.

This technology is a gene therapy strategy to treat TK2 deficiency-associated mitochondrial DNA depletion syndromes with an engineered adenovirus-associated viral (AAV) vector to deliver a functioning TK2 gene.