RARECast

Tessera Therapeutics is seeking to move beyond gene editing and gene therapy to what it calls “gene writing.” The company said its technology can be used to change base pairs, make small insertions or deletions, and integrate entire genes into the genome. We spoke to Geoffrey von Maltzahn, co-founder and CEO of Tessera, about the company’s gene writing technology, how it works, and the potential for it to accelerate the pace of genetic medicine.

Leslie Gordon, co-founder and medical director of the Progeria Research Foundation, discusses the role her organization has played in creating an understanding of progeria, identifying and advancing a therapy for the condition, and how a windfall from the sale of a priority review voucher from the approval of the first treatment for the condition will help advance future research.

Eric Dube, CEO of Travere Therapeutics, discusses the company’s new identity, its experimental therapy sparsentan, and the rare kidney diseases it is being developed to treat.

Jude Samulski, a gene therapy innovator and chairman and chief scientific officer of the nonprofit Columbus Children’s Foundation, discusses how the foundation operates, the range of capabilities it is able to bring together to accelerate the development of gene therapies for children with ultra-rare disorders, and the potential to scale what it does to reach more patients in need of treatments.

Daniel de Boer, CEO of ProQR Therapeutics, discusses the evolution of his company, its pipeline of antisense oligonucleotide therapies, and the advantage of this approach in treating rare eye diseases.

P.J. Brooks, program director in the Office of Rare Disease Research at the National Center for Advancing Translational Sciences, discusses the PaVe-GT program, the potential for developing a toolkit of plug-and-play vectors, and how this can alter the cost of developing gene therapies for ultra-rare and individual patients.

Janet Lambert, CEO of the Alliance for Regenerative Medicine, discusses the state of the gene therapy industry, the challenges developers face in advancing therapies to the market, and emerging pricing approaches to make them accessible to the patients who need them.

Kim Nye, founder and president of the TESS Research Foundation, discusses her own journey to becoming a rare disease advocate, how a gene therapy fit into her organization’s research agenda, and the considerations she is weighing before dosing her children with an experimental gene therapy.

In this first of our four-part series on gene therapies, genetic counselor Stephanie Gandomi, discusses the role genetic counselors play in working with rare disease patients, and considerations for patients who may want to pursue a gene therapy through a clinical trial.

Nell Meosky Luo, founder and CEO of Folia Health, discusses spoke to Luo about how patients are using the platform, its potential to gather real-world data, and how the data it gathers could be used to advance the understanding of rare diseases.