A Renewed Case for Optimism in Cell and Gene Therapy: Progress Outweighs Recent Setbacks

November 25, 2025 — Opinion By Tim Hunt, Gaurav Shah, MD, Amy Atkinson, Jenny Hsieh, Greg Ruf, Nina Wachsman, and Laura Wills, via BioSpace

Despite recent headlines focusing on clinical trial suspensions, safety concerns, and regulatory hurdles, a coalition of stakeholders spanning patient advocacy, clinical foundations, nonprofits, and biopharmaceutical innovation argues that a renewed case of optimism for cell and gene therapy (CGT) is warranted and essential.

The authors—including leaders from the Alliance for Regenerative Medicine and rare disease foundations—believe that underappreciated progress, coupled with key regulatory and market shifts, signals a potential turning point for the sector.

Government and Regulatory Support

The U.S. government is actively working to modernize its approach to CGTs, especially for rare diseases:

• Adaptable Pathways: Health Secretary Robert F. Kennedy Jr. and FDA officials have expressed strong commitment to keeping the U.S. at the center of CGT research. FDA leadership has welcomed the development of patient-centric and adaptable regulatory pathways for rare and incurable conditions.

• New Guidance: The FDA recently released a trio of new draft guidances to assist developers, and the agency is advancing a “plausible mechanism” pathway designed to streamline regulatory review for customized, small-population gene therapies.

• Easing Restrictions: In June, the FDA removed Risk Evaluation and Mitigation Strategies (REMS) and eased monitoring for approved CAR-T therapies for blood cancers, an action expected to expand access beyond the 20% of eligible patients currently receiving treatment.

• Payment Modernization: The Centers for Medicare and Medicaid Services (CMS) announced the Cell and Gene Therapy Access Model, with 33 states and territories participating. This substantial federal initiative features outcomes-based payment agreements for approved sickle cell disease therapies, setting a potential model for other disease areas.

Scientific and Market Inflection Points

Signs of renewed optimism are emerging in clinical outcomes, investor confidence, and the commercial pipeline:

Clinical Breakthroughs

• Personalized Medicine: The case of “Baby KJ,” an infant successfully treated with the first reported use of an individualized CRISPR-based therapy for the lethal metabolic disorder CPS1 deficiency, catalyzed national interest in scaling highly personalized treatments.

• Durable Results: Gene therapy is showing promise in life-threatening rare diseases. For Danon disease, an early-stage study published in the New England Journal of Medicine showed patients who would otherwise face heart transplant or death remained clinically stable after a one-time AAV-based gene therapy.

• Next-Generation Treatments: The approval of the first gene-editing therapy for sickle cell disease has demonstrated that a single treatment can eliminate vaso-occlusive crises for many patients.

Market Confidence

• Stock Rebound: Publicly traded CGT stocks, which significantly trailed the XBI biotechnology index in 2023 and 2024, are now tracking closely with the index, reflecting renewed investor confidence.

• Financial Projections: The CGT market is projected to grow from $8.4 billion in 2024 to $54.4 billion in 2030, representing a compound annual growth rate ($36.5\%$). The number of CGT blockbuster products (>$1 billion annual sales) is expected to reach double digits by 2030.

• Major Investments: Large biopharmaceutical companies, including AbbVie, Bristol Myers Squibb, Eli Lilly, and Pfizer, have collectively spent over $5 billion on acquisitions of small CGT companies in the past two years, signaling strong strategic commitment to the sector

The Path Forward

While acknowledging the inherent risks, the authors maintain that for patients facing life-threatening conditions with limited alternatives, the potential for a one-time, durable therapy often outweighs the risks. The field is continuously learning from setbacks, optimizing AAV vector design, dosage, and tissue targeting, and exploring new delivery platforms like non-viral and re-dosable approaches. The future depends on stakeholders maintaining the foresight to invest, the courage to innovate, and the commitment to ensure access for all who stand to benefit from this new chapter in medicine.