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Thirunavukkarasu Angappan: Gene Therapy’s Slow Road to Routine Hemophilia Care

Thirunavukkarasu Angappan/LinkedIn

Aug 3, 2026, 11:49

Thirunavukkarasu Angappan: Gene Therapy’s Slow Road to Routine Hemophilia Care

Thirunavukkarasu Angappan, Vice President of Operations and Manufacturing at PopVax, shared a post on LinkedIn:

“Gene Therapy Was Supposed to End Factor Concentrate Demand.

It hasn’t.

Three years ago, gene therapy was positioned to make routine factor concentrate infusions obsolete for hemophilia.

That hasn’t happened, and the reasons matter for anyone in plasma or recombinant protein manufacturing.

FDA approved Hemgenix for hemophilia B in 2022, Roctavian for hemophilia A in 2023, and Beqvez for hemophilia B in 2024 — one-time infusions priced between 2.9 million dollars and 3.5 million dollars, positioned to replace years of factor replacement therapy.

Uptake has been low enough that Pfizer terminated its hemophilia A gene therapy partnership with Sangamo in December 2024, then discontinued commercialisation of Beqvez itself in February 2025.

Part of the story is biology.

Both approved therapies deliver their gene into liver hepatocytes — the right cell type for Factor IX, but Factor VIII is normally produced by a different liver cell entirely.

Roctavian’s efficacy in hemophilia A has been comparatively modest, while emicizumab (Hemlibra), a simpler subcutaneous antibody therapy, already gets many patients to workable factor levels without an infusion regimen at all.

The rest is adoption economics: patients are reluctant to trade a therapy they understand and manage well for a one-time treatment with uncertain long-term durability and extraordinarily complex reimbursement.

For plasma and recombinant manufacturers, it’s a useful reminder that ‘one-time cure’ narratives move slower through clinical adoption than through press releases.

Factor concentrate demand — plasma-derived and recombinant — remains the backbone of hemophilia care for now, and capacity planning built around gene therapy displacing it on a five-year horizon looks premature.

For those tracking cell and gene therapy portfolios — is hemophilia a case where the biology was harder than the platform, or an adoption and pricing problem that better data eventually solves?”

Thirunavukkarasu Angappan: Gene Therapy's Slow Road to Routine Hemophilia Care