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Two Months, One Platform, Three Diseases: Glafabra's Progress So Far
Glafabra
Two months. One platform. Three diseases. Building in public the whole way.
Fabry, Pompe, and Gaucher disease — three diseases the platform is designed to address by correcting one cell type and swapping in the disease-specific gene. An FDA meeting behind us, an IND protocol taking shape, University of Utah Health planned as the clinical site, and a Japan strategy running in parallel from day one.
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Two Months Into Testing the Waters: What's Ahead for Glafabra
Two months of Testing the Waters, almost to the day.
Since June 22: 18 questions answered through DECODE, four members of the Glafabra team introduced in person on LinkedIn, and a real-time record of an FDA meeting, a protocol taking shape, and a Japan strategy moving in parallel with the U.S. one.
What's ahead: more team introductions, continued DECODE questions, and whatever comes out of IFFD an...more
How Common Is Fabry Disease? — DECODE No. 18
(Answer to the question last time on August 31: hands and feet — acroparesthesia, caused by small-fiber nerve involvement, often starting in childhood.)
Fabry disease is rare, but estimates for exactly how rare span a wide range — and most researchers think the real number is higher than any of them.
Roughly how many people are estimated to be born with Fabry disease? Drop your guess in the commen...more
How Glafabra's Platform IP Covers Fabry, Pompe, and Gaucher Disease
One platform, three diseases, and a body of intellectual property built to protect all three at once.
Glafabra's patent position covers the Live-cel approach across its lysosomal storage disorder programs — Fabry (GT-GLA-S03), Pompe (GT-GAA-S04), and Gaucher (GT-GBA1-S05) — because the underlying platform (correct the HSC, let cross-correction do the rest) is the same regardless of which gene gets...more
Meet Glafabra's Director of Medical Affairs, Dr. Kevin Marhenke
Meet Dr. Kevin Marhenke, PharmD, Director of Medical Affairs.
Kevin brings more than four decades in pharmaceutical and biotech leadership — hospital pharmacy, medical affairs, external engagement, and strategy. He held senior roles at Immunex and Amgen, leading market introduction for Enbrel, Prolia, EVENITY, and biosimilars, and later served as Amgen's Director of Global External Engagement acro...more
Where Does Early Fabry Disease Pain Usually Occur? — DECODE No. 17
(Answer to the question last time on August 27: roughly 40 percent of ERT patients develop anti-drug antibodies over time, which can reduce how well the infused enzyme works.)
It's often the very first symptom Fabry patients notice, frequently in childhood — a burning, tingling pain in a specific part of the body.
Where in the body does this early, telltale Fabry pain usually show up? Drop your gu...more
Why Rare Diseases Like Fabry Stay Underfunded by Big Pharma
A pharma company will almost always choose a blockbuster over a small patient population. That's not cynicism — it's how the economics of a multibillion-dollar portfolio actually work.
It's also exactly why rare diseases like Fabry, Pompe, and Gaucher stay underserved relative to their severity, and why community funding models — ours included — exist at all. A therapy addressing a few thousand pe...more
Why Fabry Disease Requires a Multidisciplinary Care Team
Fabry disease touches the kidneys, heart, nerves, skin, and GI tract — so no single specialist manages it alone.
Patients typically see a rotating team: a metabolic or genetics specialist, a nephrologist, a cardiologist, a neurologist, sometimes a pain specialist. Coordinating all of that is its own quiet burden on patients and families.
Glafabra's own team mirrors that same instinct toward breadt...more
