Newsletter | July 2, 2025

07.02.25 -- Hope For People With Spinocerebellar Ataxia

SPONSOR

Explore the evolving role of exosomes and extracellular vesicles (EVs) at the next Cell & Gene Live. Chief Editor Erin Harris moderates a discussion with Capricor’s Linda Marbán and Aegle’s Dr. Gloria Matthews on clinical and commercial advances, including applications in rare and cardiovascular diseases. Gain insights into the future of exosome-based therapies from leaders driving innovation across R&D, manufacturing, and commercialization.

FOCUS ON REGULATORY & COMPLIANCE

Hope For People With Spinocerebellar Ataxia

Spinocerebellar ataxia (SCA) is a rare, progressive, and fatal neurodegenerative disease with no approved treatments, but Biohaven Pharmaceuticals’ new drug troriluzole — designed to modulate glutamate and slow disease progression — has shown promise in clinical trials and is currently under FDA review, offering hope to patients and families facing this devastating disorder.

Cleanroom Conceptual Design Paradigm: Converting Spaces For GMP Operations

Learn essential conceptual design principles for transforming alternative spaces into efficient, regulatory-aligned cleanroom environments for your critical processes.

Shaping Therapeutic Development With Cutting-Edge CRISPR Nucleases

Gain an understanding of how an advanced nuclease portfolio is designed to support CRISPR-based cell and gene therapy development, providing scientists with the best solutions for therapeutic success.

The Critical Role Of Residue Removal In Cleanroom Biosafety Cabinets

Learn about the significance of residue removal, how it is effectively performed in cleanrooms to ensure the compliance of biosafety cabinets, and the impact of proper residue removal.

SPONSOR

Webinar: Improving Quality Control: Tackling Residual DNA Testing in Biotherapy Manufacturing

Explore how to strengthen quality control in biotherapy manufacturing with advanced residual DNA testing in this webinar from Thermo Fisher Scientific. Learn why residual DNA testing is vital for product safety and compliance, uncover common challenges and misconceptions, and discover how molecular-based methods can enhance accuracy, sensitivity, and reliability in your QC workflows. Click here to learn more.

REGULATORY & COMPLIANCE SOLUTIONS

Comprehensive CMC-QC Solutions For Cell Therapy - ACROBiosystems

Mitigate Clinical Risk In Cell Therapy Development With A GMP Compliant Process - MaxCyte, Inc.

SPONSOR

With pressure to both accelerate development and prioritize robustness, CGT companies often have limited time for refining manufacturing, analytics, and logistics. Enter the Cell & Gene Therapy Tech Expo: a dynamic slate of industry-leading suppliers showcasing the latest in solutions across Cell Therapy, Gene Therapy, and GMP/Regulatory Compliance via 15-minute interactive presentations. Register for free and make continuous improvement a reality on your path to scalability, cost reduction and, ultimately, enhanced patient access.

Connect With Cell & Gene: