12 Best Young Stocks with Huge Upside Potential

3. Lexeo Therapeutics Inc. (NASDAQ:LXEO)

Number of Hedge Fund Holders: 15

Average Upside Potential as of October 10: 142.72%

Lexeo Therapeutics Inc. (NASDAQ:LXEO) is one of the best young stocks with huge upside potential. On October 7, Lexeo Therapeutics announced progress regarding its gene therapy candidate, LX2006, for Friedreich ataxia/FA cardiomyopathy. The company shared positive updates from discussions with the US FDA on an Accelerated Approval pathway, along with encouraging new interim clinical data from ongoing Phase I/II studies.

Lexeo’s leadership believes this combination of regulatory support and compelling data could significantly accelerate the overall timeline for a Biologics License Application/BLA submission. The FDA indicated that it is open to an accelerated approval BLA submission that includes pooling clinical data from the ongoing Phase I/II trials with new data generated in the planned pivotal study.

To enable this, Lexeo will need to submit enhanced manufacturing comparability data and meet an additional nonclinical requirement before starting the pivotal trial. This is because the company intends to utilize its optimized, high-yield Sf9-baculovirus manufacturing platform for future commercial supply, a departure from the adherent HEK293 process employed for the Phase I/II supply. The FDA previously agreed to evaluate the co-primary endpoint of left ventricular mass index at a time point earlier than 12 months. Lexeo believes this regulatory flexibility will enable a smaller and shorter pivotal study, with plans to initiate the trial as soon as possible in H1 2026.

Lexeo Therapeutics Inc. (NASDAQ:LXEO) is a clinical-stage genetic medicine company that focuses on hereditary and acquired diseases with high unmet need in the US.