$ABEO (+0.88%) Abeona Therapeutics is a US biotechnology company specializing in the development of gene therapies for rare genetic diseases. Its flagship product is the gene therapy PZ-Cel for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), an extremely painful and potentially life-threatening skin disease for which there are currently no approved therapies.

Why Abeona has great potential:

• FDA priority review: PZ-Cel is in the final stages of approval by the US Food and Drug Administration (FDA), with an expected decision in the third quarter of 2025. Approval would position Abeona as a first mover in this market and enable a market launch.
• Market potential: RDEB disease affects only a few thousand patients worldwide, which puts Abeona in an exclusive niche with high medical need and little competition. The prices for gene therapies in such areas are often very high, which greatly increases the sales potential.
• Financial basis: At the end of 2024, Abeona had cash and cash equivalents of around USD 98 million, which will secure further clinical development and preparations for market launch. At the same time, although the company is still operating at a loss (approx. USD 63.7 million net in 2024), it is making targeted investments in research and development.
• Share price performance & analyst opinions: The share is currently trading at around USD 5. Analysts see average price targets of just under USD 20, some even up to USD 96 - which corresponds to a possible price increase of up to 1700%. This shows the enormous growth potential if PZ-Cel is successfully approved and marketed.

Risks:

• Approval: The biggest hurdle is the FDA's decision. A rejection or delay could weigh heavily on the share price.
• Market launch: Even after approval, the distribution network, approvals in other countries and cost reimbursements must be clarified - there are also uncertainties here.
• Industry risks: As with all biotech companies, the development of new therapies is associated with high risk, for example due to unexpected side effects or technical problems.

Conclusion:

Abeona Therapeutics offers an exciting opportunity for risk-tolerant investors to get in early on a potential breakout story in the field of gene therapy. If PZ-Cel is successfully brought to market, the stock could become a real multi-bagger. At the same time, the risks and volatility should not be underestimated.