At the recent Evolution of the Cell and Gene Therapy (CGT) Sector Workshop by the Alliance for Regenerative Medicine (ARM), speakers from across industry, academia, and advocacy explored what needs to happen next for cell and gene therapies to reach full maturity. The consensus was clear that innovation in CGT is progressing faster than the systems that support it. The next challenge is to make these therapies scalable, reliable, and accessible. In this blog, we cover six key themes that emerged from the discussions.
On the latest episode of The Genetics Podcast, we spoke with Ananth Sridhar, Chief Operating Officer for cardiorenal programs at BridgeBio, and Sun-Gou Ji, the company’s Vice President of Computational Genomics. The conversation explored how BridgeBio’s distinctive hub-and-spoke model enables sustainable rare disease drug development, how genetics drives their discovery work, and why the right economic model is essential for tackling small patient populations.
AI is becoming a core component of drug development and clinical research. Models are improving and regulatory frameworks are evolving, but progress still depends on the quality and structure of the underlying data. To move from experimental AI to operational AI, organizations need datasets that are traceable, standardized, and auditable from source to model output.
In drug discovery, data is the key to success. Although more and more large-scale data is being generated every year, much of it is inaccessible or fragmented. Genomic sequences sit in one silo, proteomic measurements in another, imaging data in a third, and clinical information across even more systems. The result is vast potential locked behind barriers due to inconsistent formats and disconnected workflows.
In the last episode of The Genetics Podcast, Patrick spoke with Justin Porcano, co-founder and executive director of Save Sight Now, a nonprofit accelerating therapies for Usher syndrome type 1B (USH1B). Justin and his wife, Rosalyn, launched Save Sight Now after their daughter, Leah, was diagnosed in 2018. The rare genetic condition causes profound deafness at birth, vestibular dysfunction, and progressive vision loss.
Gene editing has progressed significantly over the past decade, evolving from an experimental technology that was associated with safety and efficacy limitations into a legitimate therapeutic avenue that has been tested across diseases and patient populations. Yet, while technical progress has been remarkable, real-world impact remains uneven. Investment, accessibility, and disease diversity continue to lag behind the field’s scientific potential.
In a special live episode of The Genetics Podcast, recorded at the American Society of Human Genetics (ASHG) meeting, host Patrick Short was joined by two of the field’s leading voices: Dr. Heidi Rehm, Chief Genomics Officer at Massachusetts General Hospital and Co-Director of the Medical and Population Genetics Program at the Broad Institute, and Dr. Slavé Petrovski, Vice President of the Center for Genomics Research at AstraZeneca.
On this episode of The Genetics Podcast, Patrick spoke with Chris Hopkins, CEO of Glafabra Therapeutics, a company developing a one-time autologous cell therapy for Fabry disease. The therapy is designed to provide durable enzyme replacement without the limitations of current treatments and may serve as a platform for other lysosomal storage disorders.
At this year’s European Society of Gene & Cell Therapy (ESGCT) meeting, one theme resonated across sessions: adeno-associated viruses (AAVs) are entering a new era of innovation. The field is moving beyond solving manufacturing bottlenecks to designing viral vectors that are more efficient, safer, and ultimately cheaper to deliver at scale.
Participation in clinical trials can be burdensome and disruptive. In addition to practical inconveniences and discomfort, participants may have to take time off work, arrange for childcare, and travel long distances to sites. Nevertheless, patient populations can be highly engaged and motivated to participate in clinical research and impact the drug development process.