Abeona Therapeutics

Beacon Therapeutics has exercised its option to license Abeona's patented AAV204 capsid technology for developing gene therapies targeting prevalent and rare retinal diseases that cause blindness.

Bavarian Nordic announced the sale of its Priority Review Voucher for $160 million, awarded following FDA approval of its chikungunya vaccine VIMKUNYA in February 2025.

Stanford Medicine-led phase 3 trial demonstrates gene therapy skin grafts achieved 81% healing in epidermolysis bullosa wounds compared to 16% with standard care after 24 weeks.

The US orphan drug market is projected to surpass $190 billion by 2030, with over 7,300 molecules receiving FDA Orphan Drug Designation to date, of which approximately 17.9% have gained approval.

The FDA has granted approval for Abeona Therapeutics' cell therapy, marking a significant advancement in treatment options for patients with a rare genetic skin condition.

A comprehensive new industry report analyzes over 1,670 clinical-stage partnering agreements in pharma and biotech from 2020-2025, providing unprecedented access to deal structures and financial terms.

Priority Review Vouchers (PRVs) have surged in value to $150-158 million in recent months, up from the typical $100 million, following Congress's failure to reauthorize the rare pediatric disease program.

Oppenheimer launches coverage of Abeona Therapeutics with an Outperform rating and $16 price target, highlighting potential of their lead candidate pz-cel.

The FDA has accepted Abeona Therapeutics' resubmitted Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) to treat recessive dystrophic epidermolysis bullosa (RDEB).

• The FDA is reviewing Dupixent for chronic spontaneous urticaria, with a PDUFA date of April 18, 2025, potentially offering a novel targeted therapy after a decade of limited options. • Abeona Therapeutics' pz-cel for recessive dystrophic epidermolysis bullosa has a resubmitted BLA with a PDUFA date of April 29, 2025, following a Complete Response Letter earlier in 2024. • Arcutis Biotherapeutics' roflumilast foam for scalp and body psoriasis has a PDUFA date of May 22, 2025, offering a steroid-free alternative for hard-to-treat areas.