Gene Therapy

Big pharma executives at ARM’s Cell and Gene Meeting on the Mesa shared some of the practical challenges of bringing one-time treatments to market in hemophilia, cancer and beyond.

Trevor Martin talked to In Vivo about the importance of sharing a strategic vision with partners, the company’s superpower, and how a PhD is the best prep for a startup.

With more advanced therapies gaining approval, companies at ARM’s Cell and Gene Meeting on the Mesa considered hurdles to making their products more broadly available and developing them for larger indications.

Purespring has just raised $105m from an investor syndicate who are betting it can develop the first gene therapy for kidney disease and learn from setbacks experienced in the space.

Hemgenix has now secured reimbursement in several European markets, with more talks ongoing. While the path to reimbursement has not always been easy, innovative access deals have helped to smooth the way.

An upfront payment of $110m from BMS has come just in time for Prime Medicine, but its real test will be the first Phase I data, expected early next year.

Long-term follow-up requirements have taken a conservative approach, but could be ripe for re-examination and global harmonization, Kite Pharma executive director says, while former FDA gene/cell therapy office head Wilson Bryan wants the classwide REMS eliminated.

The move is positive for manufacturers and follows the agency’s decision to drop another contentious proposal that would have ‘stacked’ drug discounts when calculating Medicaid best prices.

An FDA question to a patient concerned about the negative impacts of the CAR-T boxed label warning during a recent listening session indicates the agency may be thinking about improving its dissemination of the information. 

Japan plans to offer wider support to foreign firms and ventures with innovative candidates to start clinical trials in the country, as part of key measures from current prime minister Fumio Kishida.