Newsletter | September 19, 2025

09.19.25 -- Gene Therapy For Inherited Retinal Diseases

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Advancing the Future of Cell & Gene Therapy

This 11-12 November, Cell 2025 unites 1,000+ experts across the CGT value chain to solve key challenges, from scalable bioprocessing and regulatory hurdles to accelerating development. Gain insights across cell culture, therapy development, and CGT manufacturing, and learn from world-renowned leaders Bobby Gaspar (Orchard Therapeutics), Christof Von Kalle, (Research Clinic Luxemburg), and Dame Molly Stevens (University of Oxford). Register Now

FOCUS ON SUPPLY CHAIN

Assessing Residuals In Processing And Fill/Finish For iPSCs

In this segment of Cell & Gene Live, our expert panelists, Pratik Jaluria, Ph.D., SVP Technical Development Strategy at BlueRock Therapeutics, Bruno Marques, Ph.D. VP, Process and Product Development at Century Therapeutics, and Kate Rochlin, Ph.D., COO at IN8bio, explain why detecting and quantifying trace levels of cryoprotectants and excipients in cell therapy products involves a risk-based approach, starting with theoretical worst-case calculations based on formulation concentrations and patient exposure limits.

Small Volume ATMP Cryo-Freezing Strategies

Discover best practices for cryopreserving small-volume ATMPs, focusing on sterility, viability, and efficiency with advanced containers and packaging.

SUPPLY CHAIN SOLUTIONS

Flexible Freeze Containers - W.L. Gore & Associates

FOCUS ON CLINICAL TRIALS

Gene Therapy For Inherited Retinal Diseases With Opus Genetics' Dr. George Magrath

In this episode of Cell & Gene: The Podcast, George Magrath, CEO of Opus Genetics, shares with Host, Erin Harris, how the company is advancing gene therapies for inherited retinal diseases (IRDs), with a focus on LCA5, a rare and severe form of childhood blindness.

Innovations In The Therapeutic Paradigm Of Cell Therapy

What is the curative potential of protein design? Marc Lajoie, CEO at Outpace Bio, discusses how de novo proteins can be used to reprogram immune cells. 

Mobilizing Community Hospitals To Improve Patient Access To CGTs

The patient, product, and value chains for cell and gene therapies are far different than traditional healthcare delivery and requires new and specialized sets of capabilities at hospitals.

Overview Of Idiopathic Pulmonary Fibrosis: Global Clinical Trials Landscape

Explore the developments in Idiopathic Pulmonary Fibrosis treatment, focusing on innovative drugs, ongoing clinical trials, and their potential impact on patient outcomes.

The Resurgence Of Antibody-Drug Conjugates

While scientific and operational hurdles remain, ongoing innovation and improvements in clinical trial design are rapidly expanding the therapeutic reach of ADCs.

Crafting Scalable Solutions For Maximum Reach And Success

Is your supply chain ready for a blockbuster cell and gene therapy launch? Industry leaders discuss selecting partners, managing risk, and scaling operations for maximum reach and success.

From Missing To Meaningful: Solving EHR Data Gaps

When data is missing or delayed, it can slow down trial progress. Improving EHR data quality strengthens clinical research and ensures faster access to life-changing therapies for patients in need.

Rapid Results In A High-Stakes Dry Eye Disease Trial

This time-sensitive Phase 3 trial in dry eye disease required precision project management leveraging strong site relationships, ophthalmology-specific expertise, and data-driven site selection.

CLINICAL TRIALS SOLUTIONS

An Easy-to-Use Analytics Platform For Vendor Selection And Management - ClinAI

Central IRB Services Combining A Service-First Philosophy And Expertise - BRANY

An End-To-End Clinical Data Science Solution - Revvity Signals Software, Inc.

Life-Changing Therapies Without A Lifetime Of Development - Precision for Medicine

EVENTS

Advancing the Future of Cell & Gene Therapy

This 11-12 November, Cell 2025 unites 1,000+ leading experts across the CGT value chain to solve key challenges, from scalable bioprocessing and regulatory hurdles to accelerating development timelines.

 

Gain practical strategies across cell culture, therapy development, and CGT manufacturing, and hear from world-renowned leaders Bobby Gaspar (CEO, Orchard Therapeutics), Christof Von Kalle, (Director & BIH Chair, Research Clinic Luxemburg), and Dame Molly Stevens (University of Oxford). Register Now

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