Cell and Gene therapy leaders from mid Atlantic forge connections Children's National

Nearly 200 biomedical leaders from Washington, D.C., Maryland, and Virginia gathered at the Children's National Research & Innovation Campus for the 2nd annual Cell & Gene Therapy Symposium. The event showcased groundbreaking developments in rare disease treatments and underscored the importance of regional collaboration.

"By targeting diseases at the cellular level, we are on the cusp of breakthroughs in cell and gene therapy that will transform medicine," said Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research (CCIR) at Children's National Hospital and a host of the symposium. "Progress will accelerate if we build partnerships beyond our own organizations."

The big picture

Scientists and clinicians have worked for more than two decades to develop cell and gene therapies aimed at treating diseases on a cellular level. The past few years have been particularly promising as investment in science has led to advancements. Children's National is at the forefront, as one of the first pediatric hospitals in the world to offer commercial gene therapies for sickle cell disease.

Many more treatments for rare diseases are in development at Children's National and beyond. Leaders at CCIR are actively building collaborations with companies, academic institutions and enterprises across the mid-Atlantic region to accelerate these efforts.

During the symposium, Eugene Hwang, M.D., chief of Oncology at Children's National, addressed the urgent need for more effective and less toxic treatments for pediatric brain tumors. He highlighted the potential of combining immunotherapies with innovations like low-intensity focused ultrasound, which can open the blood-brain barrier temporarily to improve drug delivery to tumors.

"With collaboration between the lab and clinic, alongside industry partners and even between hospitals, we can finally make strides I haven't seen in my entire career," Dr. Hwang said. "It's an incredibly inspiring time for all of us."

Why it matters

Experts from organizations as diverse as MaxCyte, ScaleReady, RoosterBio, PSC Biotech, Qiagen, FujiFilm and the Frederick County Office of Economic Development came together for the daylong conversation.

Michael Friedlander, Ph.D., executive director of the Fralin Biomedical Research Institute at Virginia Tech, emphasized the critical role of regional partnerships in fulfilling the potential of these emerging therapies. He pointed to the collaborative research between Children's National and Virginia Tech on brain tumors, where bioengineers and cancer researchers are working side-by-side to create new treatments.

"We are now able to begin delivering these leading-edge therapies to patients," Dr. Friedlander said. "For example, those who live in rural settings often have much less access to such frontline medical innovations. By collaborating with Children's National and gaining access to urban pediatric populations, as well as patients in our more rural area, we can start to bring these therapies to a much broader audience."

What's next

Patrick Hanley, Ph.D., chief and director of the Cellular Therapy Program at Children's National, observed that other regions in the U.S. are uniting to advance scientific discoveries with the backing of government, academia and industry. He hopes to see similar collaboration across the D.C., Maryland, and Virginia area, known as the DMV. Children's National is leading an initiative called CHARM - the Capital Health and Mid-Atlantic Regenerative Medicine - to bring regional experts together for webinars, networking events and partnership opportunities.

"There's significant interest in cell and gene therapy worldwide," said Dr. Hanley, a symposium host. "I see an even greater interest in creating cell and gene therapy hubs. The time is right for our mid-Atlantic region, and I'm excited to see what unfolds in the next five years."

Public link

Please use the above public link if you want to share this noodl on another website.