Abeona Therapeutics

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Founded: 2013

Location: Cleveland, OH

Employee range: 50 - 200

Clinical stage: Clin3 (fast track)

Therapy area: Recessive dystrophic epidermolysis bullosa (ODD)

Drug types: GEN, DRM, PED, RAR, OPH

Lead product: EB-101

Funding: $25M stock Jul 2023; $35M stock Nov 2022; $25M stock Apr 2022

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Business:

Therapies for Sanfilippo (SF) Syndrome Type A and Type B.

Drug notes:

AB0-504 RD/Clin0 Stargardt disease; ABO-503 RD/Clin0 x-linked retinoschisis; ABO-505 RD/Clin0 autosomal dominant optic atrophy; AIM Vectors RD undisclosed

About:

Abeona Therapeutics is developing cell and gene therapies for patients with serious diseases. Delivery of gene therapies to the desired tissue without causing an immune response is important for their success. Abeona is using their next-generation adeno-associated viruses (AAVs) to selectively deliver genetic payloads to the central nervous system, lungs, eyes, muscle, liver and other tissues. Abeona’s AAVs are non-replicating and have shown potential to evade immune responses generated by exposure to naturally-occuring AAV vectors. Abeona is applying their AAV technology for several diseases with therapies already in clinical trials. This includes EB-101, in Phase III trials for patients with Epidermolysis Bullosa and other undisclosed targets in preclinical phases.