Gene Therapy CMC & Analytics

Improving Quality, Control, and CMC of Genetic Medicines and in vivo CAR Ts

8/11/2026 - August 12, 2026 ALL TIMES EDT

Cambridge Healthtech Instituteâ€™s 11th Annual Gene Therapy CMC and Analytics conference explores the key CMC and control strategies supporting viral and non-viral gene therapy development. Key sessions include latest CMC and analytical strategies for late-stage assets, evolving regulatory expectations, quality, emerging technologies, and the (re)emergence of N-of-1 manufacturing. Additional themes include potency and comparability, starting-material qualification, impurities, NGS and AI-enabled analytics, late-stage characterization, empty/full capsid quantification, particle analysis, engineered capsid and vector challenges, formulation and stability, degradation pathways, fillâ€“finish, and emerging CMC requirements for in vivo CAR T.

Preliminary Agenda

Session Block

CMC FOR IN VIVO CAR T

KEYNOTE PRESENTATION:
CMC for in vivo CAR T Manufacturing: Opportunities, Challenges, and the Road Ahead

Photo of Nripen Singh, PhD, Executive Director and Site Head, Process Development, TRD CGT, Novartis , Executive Director and Site Head , Process Development, TRD CGT , Novartis — Nripen Singh, PhD, Executive Director and Site Head, Process Development, TRD CGT, Novartis , Executive Director and Site Head , Process Development, TRD CGT , Novartis

In vivo CAR T manufacturing represents a potential paradigm shift by eliminating patient-specific ex vivo manufacturing and enabling scalable, off-the-shelf therapies. The platform offers a promising alternative approach, but its success will be largely determined by Chemistry, Manufacturing, and Control (CMC) readiness. This talk evaluates CMC for in vivo CAR T production through a CMC lens, focusing on consistency, scalability, formulation stability, analytical control, and comparability.

CMC and Analytical Challenges in vivo CAR T

Photo of James Richardson, PhD, Senior Director, Analytical Development, Kite Pharma , Senior Director , Analytical Development , Kite Pharma — James Richardson, PhD, Senior Director, Analytical Development, Kite Pharma , Senior Director , Analytical Development , Kite Pharma

Special Consideration for in vivo CAR-T Products

Photo of Mo Heidaran, PhD, Chief Regulatory Scientist, Cellx Inc. , Chief Regulatory Scientist , Cellx Inc — Mo Heidaran, PhD, Chief Regulatory Scientist, Cellx Inc. , Chief Regulatory Scientist , Cellx Inc

USP Standards for Analytical Testing of Lentiviral Vectors

Photo of Anthony Blaszczyk, PhD, Senior Scientist, Global Biologics, US Pharmacopeia , Senior Scientist , Biologics , USP — Anthony Blaszczyk, PhD, Senior Scientist, Global Biologics, US Pharmacopeia , Senior Scientist , Biologics , USP

CMC, CONTROL, AND ANALYTICAL STRATEGIES

CMC and Analytical Strategyâ€”Case Study from Alexion

Photo of Lauren M. Drouin, PhD, Director, Analytical Development, Genomic Medicine, Alexion, AstraZeneca Rare Disease , Director , Analytical Dev , Alexion, AstraZeneca Rare Disease — Lauren M. Drouin, PhD, Director, Analytical Development, Genomic Medicine, Alexion, AstraZeneca Rare Disease , Director , Analytical Dev , Alexion, AstraZeneca Rare Disease

Accelerated CMC Development for AAV Product

Photo of Santoshkumar L. Khatwani, PhD, Director, Analytical Development, Sangamo Therapeutics , Senior Director , Analytical Dev & CMC , Sangamo Therapeutics — Santoshkumar L. Khatwani, PhD, Director, Analytical Development, Sangamo Therapeutics , Senior Director , Analytical Dev & CMC , Sangamo Therapeutics

Potency Assay Developmentâ€”A Case Study from Sensorium

Photo of Christine Le Bec, PhD, Head, CMC Gene Therapy, Sensorion , Head , CMC Gene Therapy , Sensorion Pharmaceuticals — Christine Le Bec, PhD, Head, CMC Gene Therapy, Sensorion , Head , CMC Gene Therapy , Sensorion Pharmaceuticals

The Hidden Link between CMC Decisions and Patient Access in Gene Therapy

Photo of Scott A. Jeffers, PhD, CTO, Gensight Biologics , CTO , Gensight Biologics — Scott A. Jeffers, PhD, CTO, Gensight Biologics , CTO , Gensight Biologics

Scaling Personalized CRISPR Therapy: Regulatory, Manufacturing and Platform Strategies

Photo of Kok-Seong Lim, PhD, Independent Consultant, Member of the USP Biologicsâ€”Cell and Gene Therapy Expert Committee , Sr Dir Analytical Sciences & Quality Control , Analytical Sciences & Quality Control , Independent Consultant — Kok-Seong Lim, PhD, Independent Consultant, Member of the USP Biologicsâ€”Cell and Gene Therapy Expert Committee , Sr Dir Analytical Sciences & Quality Control , Analytical Sciences & Quality Control , Independent Consultant

In 2025, Baby KJ became the world's first patient to receive personalized CRISPR therapy in just six months. The critical next step is translating this breakthrough into scalable, cost-effective standard care for thousands with rare genetic diseases. This presentation explores the strategic and technical foundations for building scalable gene editing platforms, examines regulatory innovations that enable rapid deployment, and discusses how industry is reshaping the economics of personalized CRISPR therapy.