Manufacturing, Gene Therapies

The FDA has approved a label expansion for Casgevy, the first CRISPR-based gene therapy, lowering the eligible age from 12 to 2 years in sickle cell disease — a move that significantly broadens the potential patient population and marks another milestone in the clinical maturation of CRISPR-based genomic medicine.

Cytiva’s Pierre-Alain Ruffieux discusses standardization, regionalization, talent gaps, and regulatory pathways shaping advanced biomanufacturing’s future ahead of BIO 2026.

Charles River Laboratories’ Dr. Alan K. Smith explains how early commercial planning, modular manufacturing, and AI-driven automation may accelerate scalable manufacturing for cell and gene therapies.

AI and PAT have been shown to reduce manufacturing variability in advanced therapies, which leads to improved process control and accelerated patient access to lifesaving treatments.

Automated density gradient ultracentrifugation boosts the efficiency of AAV purification.

Astellas taps Dyno’s AI-designed AAV capsid to boost skeletal muscle gene delivery, aiming for lower doses and safer next-gen therapies.

The approval introduces a one-time gene therapy for LAD-I that restores immune function and addresses the underlying cause of a life-threatening pediatric disease.

Key 2025 FDA draft and final guidances emphasize the modernization of biotech regulations, acceleration of rare-disease therapies, and streamlining of biosimilar pathways.

Autolomous and Trenchant’s partnership, begun early in 2025, addresses unmet CGT manufacturing needs, including slashing costs and cutting timelines.

Charles River has launched a second cohort to speed CGT innovation with technical guidance and scalable manufacturing support.

How is the biopharma industry solving manufacturing and scale-up bottlenecks for cell and gene therapies? Read on to find out.

The partnership is aiming to embed digital identification into labware, solving chain-of-identity challenges for individualized cell and gene therapies.

As cell and gene therapy is poised to play a major role in the future of medicine, it’s important to know the basics.

In a session at the Cell and Gene Meeting on the Mesa, Prime Medicine CEO Allan Reine discussed how prime editing offers versatile, safe gene correction, but that delivery to target cells remains a major hurdle.

A panel at the Cell and Gene Meeting on the Mesa discussed how advanced therapy production demands modular platforms, automation, and data governance to drastically improve patient access and affordability.

The new protein-based HDR enhancer aims to improve CRISPR precision for advancing cell and gene therapy development workflows.

This collaboration between Andelyn Biosciences and Tern Therapeutics moves the gene therapy TTX-381 closer to approval for treating vision loss linked to CLN2 Batten disease.

Third-party ICMC certification verifies multi-modality manufacturing readiness, meeting US and EU standards for advanced genetic therapies.

The Hopewell, N.J., site adds scalable, end-to-end viral vector production with integrated quality systems to speed clinical and commercial gene therapy programs.

The newly launched facility is located in The Woodlands, Texas, and will produce plasmid DNA as well as strengthen biopharma supply chains.

The company’s gene therapy, AAVB-039, for the treatment of Stargardt disease progresses to a Phase I/II clinical trial.

The consortium will focus on the delivery of a fully automated robotics cell and gene therapy manufacturing platform.

The industry is diversifying pipelines from traditional small-molecule drugs to embrace complex and exciting new modalities.

Novel nucleases with diverse properties represent promising solutions, emphasizing their potential to overcome current limitations in genome editing and therapeutic delivery.

Global scale-up requires developing replicable processes that work the same no matter where they are performed. This can be accomplished with smart factories that utilize fully automated manufacturing platforms.