Cell And Gene Therapy Summit Recap

Scientists, industry, and advocates came together to discuss the future of CGT

The 2026 North America Cell and Gene Therapy Summit took place in Boston on March 26, bringing together investors, scientists, patient advocates and more for a fruitful discussion of the future of cell and gene therapy.

The event started with an interactive welcome speech by Ramin Baghirzade, Executive Director of CDMO for Charles River. He welcomed the attendees and polled them on their goals and visions for the industry.

“We are here to promote the ecosystem and spark new ideas,” he said.

The first speaker was Palani Palaniappan, Chief Technical Operations & Quality Officer for Flagship Pioneering. Palaniappan gave a state of the union update on cell and gene therapy, touching on challenges from safety to regulatory to technology.

One of the themes of the day was the importance of addressing challenges in manufacturing. Cell and gene therapy products are notoriously difficult and expensive to manufacture at scale, which could limit their ability to reach the patients who need them.

Palaniappan also addressed the fragmented regulatory landscape, with countries moving at different speeds and with seemingly different objectives in dealing with this growing but new technology.

“These are interconnected problems, so they require integrated solutions,” he said.

He closed by describing a promising new approval in India, the NexCAR19 cancer treatment, which is showing great promise at a fraction of the cost of other similar treatments.

Palaniappan was followed by Brett Kopelan, Executive Director of the patient advocacy group debra of America. The group aims to improve the lives of patients with the rare disease Epidermolysis Bullosa (EB), which Kopelan’s own daughter suffers from.

EB is an inherited genetic disorder with a wide range of severity that impacts patients’ skin. In severe cases like Kopelan’s daughter, blisters and open wounds form on the skin and even within the body that never heal, leading to risks, such as infection and cancer. The condition also causes severe pain from constant wounds, and some patients must wear clean bandages at all times.

Kopelan emphasized the importance of the patient’s own voice in the drug development process. The patient’s needs and wants must be addressed, as they know firsthand which symptoms cause the most daily distress. He also underscored the importance of advocacy in rare disease development and offered an example from his own experience.

When negotiating with regulators over the endpoints of a drug, regulators thought a candidate that only averaged 50% wound closure was not clinically meaningful. Their criteria was based on the closest type of patients on record, which for EB was burn victims. Kopelan was successfully able to argue, along with his doctors, that when a patient is living with chronic open wounds, any relief is beneficial. But he says the negotiations are ongoing, for at every step both researchers and regulators are dealing with new territory in any rare disease that does not have a known cure.

The last session before lunch was a panel discussion moderated by Charles River’s James Cody, Associate Director Technical Sales and Evaluations. Panelists included Isaac Chan, Co-Founder of InterAct Therapeutics; Shyam Ramachandran, an Executive Director from Sanofi; Jacinthe Gingras, Chief Scientific Officer and Co-Founder at Stealth Biotech; Abraham Scaria, Chief Scientific Officer at Beacon Therapeutics; and John Lee from Mercury Consulting.

Panelists covered a wide range of scientific topics and touched on issues brought up by the previous speakers. Cody asked them about how they might go about selecting a modality for a new candidate; good payload design; how to narrow several candidates to the most promising; and clinical trial design and enrollment.

On the last point, the panelists brought up the challenges of working on rare diseases, when patients are scattered across the globe. For rare disease groups that mainly consist of infants, the inconsistent rules for clinical trial design involving children from different countries can also contribute to the difficulties.

Panelists agreed with Kopelan on the importance of listening to patients and caregivers when creating a drug development plan. They spoke of patients they have met who themselves know there is nothing that can be done for them but still want to help with potential treatments for future patients.

After lunch, the next session featured company case studies, where they shared their work, explained how CRL supports them, and discussed their future direction. 

The final part focused on investors, discussing topics like growth in the Middle East, alternatives such as crowdfunding, and what the next big thing might be. Some responses to the last questions included crowdfunding, quantum computing, and discovering the next big thing.