This is a guest post by Sam Savello, the Marketing and Communications Manager at the Penn Center for Innovation, a Technical.ly client and one of the financial supporters that helps ensure our site remains a free news resource.
Precision medicine is getting real results, helping people who’d lost hope regain a path to health. The next step is making it scale. A recent sold-out event hosted by the Penn Center for Innovation offered stakeholders a chance to discuss how to move the field forward.
Held at Wharton’s San Francisco campus in early January, alongside the JP Morgan Healthcare Conference, this edition of the annual Innovation @ Penn program attracted professionals from across the life sciences industry, the investor community and innovation space.
The theme, “Precision Medicine: Translating Discovery into Targeted Therapies,” provided a platform for in-depth discussions about emerging healthcare technologies and their practical applications, with a focus on innovative, personalized approaches to disease prevention and treatment that tailors care based on an individual’s genes, environment and lifestyle.
One key example is the inspiring story of baby KJ Muldoon, who was born with a rare metabolic disease known as severe carbamoyl phosphate synthetase 1 (CPS1) deficiency. In a historical medical breakthrough last spring, 9.5-month-old KJ was successfully treated with a personalized CRISPR gene editing therapy by a team led by Kiran Musunuru, MD, PhD and Rebecca Ahrens-Nicklas, MD, PhD at Penn Medicine and the Children’s Hospital of Philadelphia.
KJ’s story was highlighted during the discussion, which was moderated by Laura Shawver, PhD, most recently CEO and President of Capstan Therapeutics, and it raised a pressing question:
How can one-and-done personalized treatments like KJ’s be scaled to help other children with rare diseases?
Panelist Peter Marks, MD, PhD, the senior vice president of molecule discovery and head of infectious disease at Eli Lilly (and former director of the FDA’s Center for Biologics Evaluation and Research), suggested one approach.
Currently, he explained, FDA requirements often force drug developers to repeat testing and regulatory procedures for each new therapy, which is both costly and time-consuming and can involve repeated animal testing. By standardizing drug development requirements for personalized therapies within qualified laboratories, he said, these drugs could be delivered to multiple patients without duplicating safety studies.
Translating academic breakthroughs to market-ready therapies
One-and-done personalized therapies by definition have a limited market, and investor interest in supporting that kind of research has waned, said panelist Jim Wilson, MD, PhD.
Wilson, an emeritus professor of medicine at Penn who is now President, CEO and founder of GEMMABio, as well as founder and executive chair at Franklin Biolabs, has experience in both academic research and commercialization. Because of the shift in investor interest, he said, he has redirected his focus to platform approaches, keying in on strategies that allow well established gene therapy scaffolds to be quickly modified and used for treating other disease indications. That approach should reduce cost, he said, while increasing the probability of medical and commercial success.
He also highlighted the importance of public-private partnerships in the precision medicine space. When governments act as payers, share development risk and provide R&D investment, therapies can be developed and delivered at more affordable prices. GEMMABio, for example, has a partnership with the Oswaldo Cruz Foundation (Fiocruz), a public health research institution within the Ministry of Health of Brazil. The partnership provides funding for gene therapy research and manufacture for Brazil’s public health system. The ultimate goal is to expand access to rare disease treatments while lowering costs.
Several other Penn spinouts are developing technologies in the precision medicine field.
- SpaxaBio is using AI to build intelligent tissue maps.
- Lucidigene is developing digital health solutions like AI chatbots that guide patients through genetic testing processes.
- Brainstorm Diagnostics is creating a blood biomarker technology for the early diagnosis of traumatic brain injury.
- Trevarx Biomedical is combining molecular imaging and radionuclide therapy to precisely target specific tumor tissue.
And that’s not all: See a list of other Penn startups raising capital here.
Expanding precision medicine across critical care
Precision medicine could eventually find its way into routine diagnostics, said panelist Allison Rae Greenplate, PhD, director of immune health at Penn Medicine.
A blood draw, for example, could potentially tell clinicians about how a patient might respond to a particular therapeutic or vaccine, or which drugs have the best chance of treating their condition. Greenplate predicted a whole wave of innovations that scale clinicians’ ability to read the immune system in cheaper, faster, more accessible ways.
Leveraging precision medicine to benefit patients like this will require not only scientific breakthroughs, the panel underscored, but also new regulatory frameworks, scalable platforms and collaborative and innovative investment models. It won’t be easy, but there are people at Penn dedicated to making it happen, translating personalized discoveries into real-world therapies. If you’d like to learn more, watch a video of the full panel here.
