Startups, BioPharma

Abeona Therapeutics using CRISPR to treat Fanconi anemia, raises $8.5M

New York biotech Abeona Therapeutics is taking a CRISPR/Cas9 approach to treating a rare anemia - and just raised $8.5 million in equity to bring it a little closer to clinic.

New York startup Abeona Therapeutics just raised $8.5 million to expand its gene therapy pipeline for rare disease like Sanfilippo syndrome, juvenile Batten disease and Fanconi anemia.

Notably, the company’s using CRISPR/Cas9-based techniques to edit genes in treating Fanconi anemia. The rare blood disorder stems from a genetic defect in a cluster of proteins that help repair DNA. Because this mechanism is fault, however, patients with this form of anemia tend to develop leukemia at a young age.

Abeona Therapeutics is using CRISPR gene editing techniques here by delivering a normal copy of what’s otherwise a defective gene to a patient. The work is still preclinical study, but the idea is that inserting the correct gene will help reverse the anemia.

Right now, the only quasi-effective treatment for Fanconi anemia is bone marrow transplant. Abeona researchers are developing a protein-RNA complex made up on the Cas9 enzyme to recognize the faulty DNA sequence, then guide further genetic molecules to fill in the gaps.

The biotech is also developing gene therapy for Sanfilippo syndrome, a metabolic disorder, with involvement from patient advocacy groups. It plans to begin clinical trials this year.

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