Chronic Indications, Non-Viral Vectors, & Next-Gen CRISPR, Oh My!
By Anna Rose Welch, Editorial & Community Director, Advancing RNA
We’re all in our bubble day-in and day-out. So, to help you keep tabs of all the other dreamers and hard workers out there like you, here’s a list of five companies to watch as they strive to shake up the cell and gene therapy space.
Endsulin: As you may have guessed from the name, this new biotech hopes to revolutionize the treatment of Type 1 diabetes. Early animal data for their AAV8 gene therapy look positive so far. Future goals include testing in large animals (naturally diabetic dogs), with a potential entrance into the clinic in 18 months.
SalioGen raised funding earlier this spring to start preclinical development of a non-viral gene therapy for familial hypercholesterolemia and inherited macular degeneration. Instead of jumping into the vector development arena, however, SalioGen will be exploring the use of the enzyme saliogase to deliver the gene of interest.
Kano Therapeutics was the winner of the 2021 Sloan Healthcare Innovations Prize for its single-stranded DNA (ssDNA) technology. ssDNA could lead to more efficient, targeted delivery of DNA and eliminate unwanted side-effects, as well as open up currently untreatable indications. The Kano team, with its goals to help streamline the manufacturing and assembly of C&G therapies using ssDNA tech, aims to become “the Ikea of cell and gene therapy.”
Century Therapeutics is working with iPSCs as opposed to healthy donor cells to create allogenic CAR-T and CAR-NK treatments. Their lead-candidate — a CD19 CAR-T treatment — is targeting the clinic in 2022; in the meantime, they’ve devised a process comprising three genetic edits to help their future treatments escape the immune system’s censure. In turn, this could promote re-dosing opportunities and improved durability.
Caribou Biosciences is also on the quest for off-the-shelf allogenic cell therapies, with its CD19-targeting lead candidate for non-Hodgkins Lymphoma currently in Phase 1 clinical trials. The company, which is the brainchild of CRISPR founder Jennifer Doudna, is pioneering a “next-gen CRISPR editing program”— a hybrid DNA/RNA editing system called chRDNA. This platform offers a more targeted gene editing approach compared to the current generation of CRISPR.