At the 3rd Annual Cell and Gene Therapy Symposium, leaders from research, biotech, and advocacy came together at the Children’s National Research & Innovation Campus to accelerate progress for children with rare and life-threatening diseases.

The 3rd Annual Cell and Gene Therapy Symposium gathered leaders in research, biotech, advocacy and policy at the Children’s National Research and Innovation Campus, reinforcing the Mid Atlantic as a rapidly rising hub for curing rare and life-threatening diseases. This event focused not only on scientific discovery, but also on how this region is transforming ideas into real impact for children and families.

Patrick Hanley, PhD, Chief and Director of the Cellular Therapy Program at Children’s National Hospital, opened the program by acknowledging both the excitement and responsibility that come with treating children who urgently need new options.

The first keynote from Sadik Kassim, PhD, Chief Technology Officer at Danaher Corporation, responsible for the development and implementation of research and development strategies for genomic medicines, highlighted how quickly the world of cell and gene therapy is evolving and how essential it is that progress reaches patients in time.

Regional innovation at scale

Early presentations featured experts from institutions including the University of Virginia, Children’s National Hospital, Johns Hopkins Medicine and area startups. These talks demonstrated real momentum across regenerative medicine, pediatric immunotherapy and translational biotechnology, showing how research teams here focus relentlessly on advancing from concept to clinic. The message was simple. Innovation here does not stop at publication. It continues until those in need feel the impact.

Speakers including Camille Campbell, PharmD, RPh of Bella Peek LLC, Sneha Purvey, MD of VCU Health, and Brett Kopelan, MA of the Debra Foundation described the challenges families face in accessing advanced therapies. They focused on how outcomes for rare disease communities led to multiple drug approvals from the Food and Drug Administration (FDA) and stressed that equity must evolve alongside innovation. A breakthrough does not matter if a child cannot receive it.

The power of partnership

After lunch, moderator Mike Friedlander, PhD, Executive Director of the Fralin Biomedical Research Institute at Virginia Tech, led a panel featuring Catherine Bollard, MBChB, MD of Children’s National Hospital, Erica Cischke, MPH of the Alliance for Regenerative Medicine, Erin Kimbrel, PhD of Astellas Pharma, and Mark Stewart, PhD of Friends of Cancer Research. Their discussion underscored the essential roles of advocacy, regulation and industry alignment in shaping the future of care for children and adults.

A vision for a smarter biotech future

The innovation keynote from Murat Kalayoglu, MD, PhD, Managing Partner of SOAR Bio and co-founder of local biotech company Cartesian Therapeutics which has a CAR T product in a phase 3 trial for an autoimmune disease, offered a direct challenge to the field. He presented a vision where biotechnology companies are built more efficiently and with stronger operational roots to accelerate translation and sustainability. He noted that the Mid Atlantic region is well positioned to lead this approach because of its workforce, institutional partnerships, and growing investment footprint.

Science changing what is possible

The afternoon sessions explored advances that could fundamentally change the treatment landscape for children with complex and rare diseases by using precise treatment modalities.

• Fred Wu, MD, PhD, Assistant Professor at the Fralin Biomedical Research Institute and Radiation Oncologist at Inova, presented focused ultrasound technology designed to temporarily open the blood brain barrier and enable therapeutics to reach tumors including diffuse midline glioma.
• Kajal Chaudhry, PhD, of Children’s National Hospital, shared progress in multimodal T cell therapies that combine multiple immune mechanisms to overcome tumor defenses and strengthen responses in high-risk pediatric cancers. This work is a part of a large Cancer Grand Challenges award.
• Sarah Nasr, PhD, from the Fischell Department of Bioengineering at the University of Maryland, discussed lung inspired RNA delivery strategies that could expand treatment potential for pulmonary disease.
• Zachary Zamore, MD Candidate at Johns Hopkins School of Medicine, presented targeted gene therapy for nerve disorders to reduce chronic pain and spasticity without invasive neurosurgery.
• Chelsea Stamm, PhD, of the Bacteriophage Medical Research Center at Catholic University of America, won the first-place prize for best presentation by an early stage investigator and shared new bacteriophage vector platforms capable of delivering large genetic cargo into stem cells.
• Abby Lee, PhD Candidate at the George Washington University Cancer Center, showed research improving immune responses against ovarian cancer through novel T cell expansion strategies.
• Emily Powsner, PhD Candidate in Biotherapeutic Development and Delivery at the University of Maryland, demonstrated scalable manufacturing of extracellular vesicles designed to support healing in chronic wounds where current options fall short.

Across each talk, the focus stayed consistent. These therapies are being advanced not for the future, but for children now.

Momentum moving forward

The event closed with remarks from Dr. Hanley, followed by a reception where new partnerships formed and plans for the next studies and trials began taking shape in real time. The optimism was grounded in data. The urgency was grounded in the needs of families.

This region is proving that when researchers, clinicians and advocates align their priorities, the pace of progress can accelerate. The Mid Atlantic is not simply part of the conversation. It is leading the way. Children’s National Hospital and its partners across Virginia, Maryland, Delaware and Washington, DC, are demonstrating how to build systems where breakthroughs move faster, reach farther and change more lives.