Abeona Therapeutics: Advancing Gene Therapy for Rare and Life-Threatening Diseases

Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company based in Cleveland, Ohio, is dedicated to developing innovative gene and cell therapies targeting rare and life-threatening genetic disorders. With a clear focus on underserved medical conditions, the company is advancing several promising treatment candidates designed to address the underlying causes of devastating diseases.

One of Abeona’s lead programs is EB-101, a gene-corrected skin graft therapy developed for patients suffering from recessive dystrophic epidermolysis bullosa (RDEB). This condition is a rare and severe genetic skin disorder caused by mutations in the COL7A1 gene, leading to extremely fragile skin that blisters and tears with minimal friction. EB-101 uses gene-modified autologous skin cells to restore the function of the missing protein, type VII collagen, which is crucial for anchoring the layers of the skin together. The therapy has shown encouraging clinical results, offering hope for long-term relief from the painful symptoms associated with RDEB.

Another major focus for Abeona is its adeno-associated virus (AAV) based gene therapy platform, which includes two key candidates: ABO-102 and ABO-101. These experimental treatments are being developed for different subtypes of Sanfilippo syndrome, a group of inherited lysosomal storage disorders that progressively damage the brain and central nervous system in children.

ABO-102 targets Sanfilippo syndrome type A (MPS IIIA) and delivers a functioning version of the SGSH gene, aiming to restore the production of the enzyme sulfamidase. Without this enzyme, harmful substances build up in the body’s cells, leading to severe neurological decline. Preclinical and early clinical trials have shown that ABO-102 can cross the blood-brain barrier, enabling therapeutic levels of the enzyme to reach the central nervous system and potentially slow or halt disease progression.

ABO-101, on the other hand, is designed to treat Sanfilippo syndrome type B (MPS IIIB) by introducing the NAGLU gene. This therapy seeks to compensate for the genetic defect responsible for the absence of the enzyme alpha-N-acetylglucosaminidase. Like its counterpart, ABO-101 also uses AAV vectors to transport the corrective gene into patients’ cells, where it can begin producing the enzyme necessary to break down complex sugars that would otherwise accumulate to toxic levels.

Founded in 1974, Abeona has gradually evolved into a company at the forefront of rare disease research. With its strategic focus on gene therapy and regenerative medicine, the company has made significant strides in bringing cutting-edge treatments closer to patients who currently have limited or no therapeutic options.

As Abeona continues its clinical trials and regulatory engagements, the biotech community is watching closely. The company’s advancements not only represent potential breakthroughs for those suffering from rare genetic diseases but also contribute to the broader momentum building around gene therapy as a transformative approach to medicine.