7 Most Promising Gene Editing Stocks According to Analysts

2. BioMarin Pharmaceutical Inc. (NASDAQ:BMRN)

Price Target Upside: 47.51%

BioMarin Pharmaceuticals Inc. (NASDAQ:BMRN) stands third among the most promising stocks.

TheFly reported on December 19 that BMRN announced it will acquire Amicus Therapeutics in an all‑cash transaction valued at approximately $4.8 billion, paying $14.50 per share. The deal strengthens BMRN’s position in rare metabolic diseases by adding Amicus’s two marketed therapies: Galafold for Fabry disease and the Pombiliti + Opfolda regimen for Pompe disease, which generated about $599 million in combined revenues over the past four quarters.

The corporation expects the transaction to close in Q2 2026, be accretive to non‑GAAP earnings per share within 12 months, and substantially accretive beginning in 2027. It also gains U.S. rights to DMX‑200, a Phase 3 candidate for focal segmental glomerulosclerosis.

Analysts largely praised the strategic fit. Leerink Partners, Oppenheimer, and Needham highlighted the deal’s synergy with BioMarin Pharmaceuticals Inc. (NASDAQ:BMRN)’s global infrastructure and enzyme therapy focus, and its potential to boost sales and long‑term growth. Consensus remains positive (“Moderate Buy”) with an average price target near ~$88, implying significant upside, though earlier ratings had included Holds or downgrades based on competitive pressures and pricing concerns prior to the deal.

BioMarin Pharmaceuticals Inc. (NASDAQ:BMRN) is a genetics‑centric biotech leader that broadly aims to turn genetic discoveries into therapies. Its primary engagement with gene‑based technologies focuses on gene therapy, especially viral vector‑mediated delivery of functional genes for rare genetic diseases.