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Roots Analysis identifies top 10 cell and gene therapy companies

Market research firm Roots Analysis has identified ten leading companies in the cell and gene therapy sector. These companies are developing innovative treatments for rare diseases and genetic conditions.

10 June 2026
Roots Analysis identifies top 10 cell and gene therapy companies

Market research firm Roots Analysis released a list in August 2023 highlighting ten key companies operating within the cell and gene therapy sector. The analysis encompassed nearly 480 entities focused on the development of cell and gene therapies across various stages.

Cell and gene therapies are emerging as promising treatment modalities for conditions previously lacking effective options, including certain cancers and rare genetic disorders. T-cell therapies, such as CAR T-cell therapy, have shown particular efficacy in eradicating tumor cells. As of 2023, over 1,035 clinical studies were underway for these therapeutic candidates.

The field is also addressing over 7,000 documented rare diseases, many stemming from genetic anomalies. Gene therapy strategies are being developed for conditions like sickle cell disease, Parkinson's disease, and spinal muscular atrophy. The drive for curative treatments, as opposed to disease management, fuels the demand for advanced gene manipulation approaches.

Globally, more than 1,000 cell and gene therapy candidates are under investigation, with over 40 therapies already gaining regulatory approval. Notable examples include Gilead Sciences' Tecartus and Bristol Myers Squibb's Breyanzi for cancer, and Sarepta Therapeutics' Elevidys for Duchenne muscular dystrophy. The development and manufacturing of these advanced therapies require substantial investment.

Roots Analysis' research into the T-cell therapy and gene therapy markets provides insights into the current landscape and future opportunities. Companies like BridgeBio and Intellia Therapeutics, featured on the list, have secured significant funding for their research and development efforts.

Original source: rootsanalysis.com