Investors Shift Focus from Gene Therapy to Weight-Loss Drugs

Gene therapy, once hailed as a revolutionary cure for rare diseases like sickle cell, is losing favor with investors who are shifting focus to high-reward sectors such as obesity and cancer treatments.

The decline comes as sales for new gene therapies fail to meet expectations.

Several major pharmaceutical companies have scaled back on gene therapy. Pfizer, for instance, recently discontinued its hemophilia gene therapy, priced at $3.5 million per patient.

Bluebird Bio, a once-prominent player in the field valued at nearly $10 billion, was sold to private equity firms for just $30 million.

In 2024, gene therapy and gene-editing companies raised less than $1.4 billion across 39 venture rounds, according to DealForma data for Reuters.

Some companies, however, remain committed to gene therapy research.

Similarly, Vertex Pharmaceuticals remains focused on sickle cell gene therapy, despite modest 2024 sales of just $10 million.

Gene therapy involves inserting modified genes to replace faulty ones or alter protein production.

However, the treatment process is complex, often requiring hospitalization, and insurance coverage remains uncertain.

The U.S. Food and Drug Administration (FDA) supports these breakthrough treatments despite their multimillion-dollar price tags.

“We need a streamlined regulatory process and manufacturing improvements to lower costs and make these treatments more accessible,” he said.

Sarepta Therapeutics recently reported the death of a 16-year-old boy due to acute liver failure months after receiving its gene therapy for muscular dystrophy, highlighting the risks associated with these treatments.

Meanwhile, the weight-loss drug market is booming, with projected annual sales expected to reach $150 billion in the coming years.

Demand for highly effective obesity treatments has soared, attracting $1.75 billion in venture capital in 2024—nearly triple the $630 million raised in 2023.