Abeona is developing CRISPR/Cas9‑based gene therapies, including programs targeting Fanconi anemia, Sanfilippo syndrome, and juvenile Batten disease. Its Fanconi anemia work is preclinical and focuses on delivering a correct copy of defective genes to restore DNA‑repair function. Researchers are engineering a protein–RNA complex using the Cas9 enzyme to recognize faulty DNA sequences and guide corrective genetic material. The company says its Sanfilippo program has involvement from patient advocacy groups. Abeona plans to begin clinical trials this year for at least one program. The company recently raised $8.5M to expand its gene‑therapy pipeline. Abeona Therapeutics is a Cleveland-based biotech developing gene therapy–based treatments for lysosomal storage diseases, with lead programs targeting MPS IIIA and IIIB. The company received Orphan Product Designations for MPS IIIA and IIIB in 2014. Abeona closed a $3.6M funding round to support its programs. The funds are intended to complete preclinical development of therapies for children with Sanfilippo syndrome (mucopolysaccharidosis type III), a rare terminal genetic disorder that typically causes death before the mid-teens. Clinical trials were anticipated to begin in early-to-mid 2015. The company is led by President/CEO Tim Miller, Ph.D.

Total raised

$12M

Funding rounds

2

Latest round

Equity

Latest activity

Jul 2015