News Related to RNA-based therapies

Novartis finalizes seventh new US facility in $23 billion expansion

May 05, 2026

Novartis finalized a seventh new US facility as part of a $23 billion expansion. The new API site in Morrisville, North Carolina, supports end-to-end US manufacturing across multiple therapy platforms.

FDA Introduces New Approval Pathway for Individualized Rare Disease Therapies

Mar 11, 2026

The FDA has established a "plausible mechanism pathway" to approve personalized genome editing and RNA-based therapies for rare and ultra-rare diseases without requiring large randomized controlled trials, streamlining access to individualized treatments.

FDA Issues Draft Guidance to Accelerate Individualized Therapies for Ultra-Rare Diseases

Feb 24, 2026

The FDA issued draft guidance establishing a plausible mechanism framework for approving individualized therapies targeting ultra-rare diseases when randomized controlled trials are not feasible due to small patient populations.

FDA Proposes Accelerated Approval Pathway for Ultra-Rare Disease Gene Therapies

Feb 24, 2026

The FDA released draft guidance establishing a new approval framework for individualized gene-editing and RNA-based therapies targeting ultra-rare diseases, allowing approvals based on small studies when traditional trials are not feasible.

FDA Proposes Framework to Speed Rare Disease Gene Therapy Approvals

Feb 24, 2026

The FDA proposed a new framework to accelerate approvals of personalized treatments for rare genetic diseases, allowing drugmakers to rely on small studies when traditional trials are not feasible while requiring post-approval evidence collection.