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Gene Therapy

The Practice-Changing Drugs That Will Graduate In 2025

The Practice-Changing Drugs That Will Graduate In 2025

A wealth of new therapies are set to successfully graduate from the pipeline in 2025. Within this cohort are a mixture of therapeutic areas, drug classes, first-time approvals, label expansions, and treatments that will meaningfully change how diseases are treated.

Big Pharma Faces A Big Reality Check In Cell And Gene Therapy

Big Pharma Faces A Big Reality Check In Cell And 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.

From PhD To Unicorn: Mammoth CEO On Pharma Deals and Squishy Biology

From PhD To Unicorn: Mammoth CEO On Pharma Deals and Squishy Biology

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.

Manufacturers Eye Challenges Of Broadening Cell And Gene Therapy Access

Manufacturers Eye Challenges Of Broadening Cell And Gene Therapy Access

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.

Still Looking For A Gene Therapy Hit, Syncona Backs Purespring In IgA Nephropathy Market

Still Looking For A Gene Therapy Hit, Syncona Backs Purespring In IgA Nephropathy Market

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.

Gene Therapy Hemgenix Gains Ground In Europe With Spanish Reimbursement Deal

Gene Therapy Hemgenix Gains Ground In Europe With Spanish Reimbursement Deal

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.

BMS Inserts Cash Into Prime Medicine’s Gene-Editing Ambitions

BMS Inserts Cash Into Prime Medicine’s Gene-Editing Ambitions

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.

CAR-T Therapies: Stakeholders Urge Rethinking Of Postmarketing Requirements And REMS

CAR-T Therapies: Stakeholders Urge Rethinking Of Postmarketing Requirements And REMS

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.

CMS Withdraws Medicaid Cell, Gene Therapy Price ‘Verification’ Survey Plan

CMS Withdraws Medicaid Cell, Gene Therapy Price ‘Verification’ Survey Plan

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.

US FDA Acknowledges Patient Frustration With CAR-T Boxed Warning

US FDA Acknowledges Patient Frustration With CAR-T Boxed Warning

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.