CRISPR Therapies, Base Editing and Approvals — 2026-10-30

Top developments

FDA Issues Draft Guidance for Individualized Genome Editing

On October 28, 2026, the FDA announced the availability of a draft guidance document titled "Individualized Human Genome Editing Products: Considerations for BLA Submission and Postapproval Management." This regulatory framework provides recommendations for how to submit Biologics License Applications (BLA) for personalized gene-editing therapies, a critical step for the emerging field of one-patient medicine. The guidance addresses postapproval management, aiming to standardize the pathway for customized treatments that have previously operated under case-by-case authorizations.

Prime Medicine Completes First In Vivo Prime Editing

Prime Medicine reported a significant technical breakthrough: the first direct editing of DNA within the human body using prime editing technology. The procedure was performed on a patient with Wilson disease, a condition caused by ATP7B gene mutations. This marks a departure from ex vivo methods, where cells are edited outside the body and re-infused, demonstrating the feasibility of delivering precise prime editors in vivo. Initial trial results for this Phase 1/2 study (PM577a) are expected to be published in 2027.

Prime Editing and CRISPR Market Forecast Hits $13.33 Billion

A new market analysis released this week forecasts that the global prime editing and CRISPR market will grow from $6.25 billion to $13.33 billion by 2035. The report highlights that clinical programs are expanding the market's potential, with success hinging on delivery manufacturing and safety evidence. As platforms move toward commercial use, the ability to reach non-hepatic tissues and manage off-target risks remains the primary determinant of which therapies will advance.

Chinese Team Publishes First In Vivo Gene Editing Data for DMD

Researchers in China published the first clinical data on in vivo gene editing for Duchenne muscular dystrophy (DMD) in the journal Cell. The study targets the DMD gene to restore the open reading frame, addressing a severe X-linked neuromuscular disease affecting approximately 1 in 3,500 to 5,000 live male births. This development underscores the rapid advancement of gene-editing therapies in China, particularly for rare neuromuscular conditions.

Local view

Chinese media outlets, including The Paper and Sohu, have extensively covered the domestic breakthrough in DMD treatment and the global milestone of Prime Medicine’s in vivo editing. The Paper highlighted the significance of the Cell publication, noting the potential for in vivo editing to transform the treatment landscape for DMD patients in China. Meanwhile, Sohu detailed Prime Medicine’s approach to Wilson disease, emphasizing the complexity of using prime editing to rewrite DNA directly in the body compared to simpler base editing methods.

Context & numbers

The regulatory and economic landscape for gene therapies continues to evolve. The FDA's new draft guidance for individualized products arrives as the field moves beyond the first wave of approved therapies like Casgevy ($2.2 million list price) and Lyfgenia ($3.1 million list price). While specific payer deals for these new in vivo therapies are not yet disclosed, the CMS Cell and Gene Therapy Access Model continues to be the primary mechanism for managing the high costs of these treatments. For 2026 Marketplace plans, out-of-pocket caps remain at $10,600 for individuals and $21,200 for families, though manufacturer copay programs may reduce this burden.

On the radar

• Lilly's VERVE-102 Phase 2 Start: Eli Lilly plans to initiate Phase 2 trials for its in vivo base editing therapy VERVE-102 by the end of 2026. The FDA has granted Fast Track designation for this PCSK9-targeting therapy.
• FDA Decision on Hereditary Angioedema: The FDA is expected to issue a decision early next year regarding an in vivo CRISPR-Cas9 therapy for hereditary angioedema, which would be the first approved in vivo CRISPR therapy if successful.
• CRISPR Therapeutics Lipid Data: Investors and clinicians are awaiting further durability data for CRISPR Therapeutics' lipid-lowering gene editing therapy, following mixed results presented at the European Society of Cardiology meeting in August 2026.