Abeona Therapeutics Raises $8.5 Million in Venture Capital

Abeona Therapeutics has raised $8.5 million to expand its gene therapy pipeline. In the works are therapeutics for rare diseases like Sanfilippo syndrome, juvenile Batten disease and Fanconi anemia.
According to an article on Med City News:

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.

Click here to read the full article on Med City News.