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Glafabra's Japan Strategy: IFFD 2026 and BIO Japan
Glafabra
Six weeks out from two Japan milestones.
October 2: IFFD 2026, the international Fabry symposium at Jikei University in Tokyo — where Glafabra co-founder Dr. Jeff Medin will present Live-cel data to the global Fabry specialist community.
October 7–9: BIO Japan in Yokohama, where the team meets Japan pharma partners directly.
As Director of APAC Strategies, this is the stretch of the calendar I've ...more
Meet Glafabra's Acting COO, Elizabeth Wagner
Meet Elizabeth Wagner, Acting Chief Operating Officer.
Elizabeth brings 18+ years of relationship-building across CROs, labs, consultants, and CDMOs to help startups move their pipelines forward. As former COO of Constant Therapeutics, she led the company to three Phase 2 assets and advanced a peptide oral formulation program through GMP production. Earlier: Corporate Development at Cubist Pharmac...more
Why Is Fabry Disease Hard to Diagnose in Women? — DECODE No. 13
(Answer to the question last time on August 13: Fabry disease can arise from a brand-new, de novo mutation in the GLA gene, with no prior family history at all.)
A standard blood test for Fabry looks for low enzyme activity. In one group of patients, that test can come back looking almost normal — even when they have Fabry disease.
Which group of patients does the standard enzyme test frequently m...more
Why Pre-IND Is the Best Time to Get Involved With Glafabra
Pre-IND is a specific moment, and it doesn't last.
Right now, before Glafabra's IND is filed, is when Japan licensing terms, partnership structures, and — for this crowdfunding round — investment interest can be shaped at the earliest, most favorable stage of the company's life. Once a U.S. cohort is enrolling, the terms of every conversation change.
This isn't urgency for its own sake. It's just ...more
Why Patient Registries Matter for Rare Disease Research
Patient registries don't get much attention, but they're doing quiet, essential work.
Organizations like the Fabry International Network and the National Fabry Disease Foundation maintain the patient communities, education, and advocacy infrastructure that make trials like ours possible to design well — and make sure patients hear about opportunities like this one in the first place.
If you're par...more
One Month After Glafabra's FDA INTERACT Meeting
One month since Glafabra's FDA INTERACT meeting.
That July 16 conversation shapes the IND protocol we're building now: endpoint strategy, patient selection criteria, conditioning approach. None of that gets finalized overnight — it's weeks of translating regulatory feedback into a protocol document.
For those tracking the timeline: IND filing stays targeted for mid-2027. First patient enrolled tar...more
Can Fabry Disease Occur With No Family History? — DECODE No. 12
Answer to the question last time on August 10: a father passes his only X chromosome to every daughter and his Y to every son — so every daughter inherits his Fabry variant, and no son does.)
Most Fabry cases run in families. But every so often, someone is diagnosed with zero family history at all — no parent, no sibling, nothing in the records.
What causes Fabry disease to show up with no family ...more
Why Does Live-cel Use Hematopoietic Stem Cells?
Why build a gene therapy around a blood stem cell instead of a virus alone?
A hematopoietic stem cell (HSC) — the cell that gives rise to your entire blood and immune system — can be corrected once, outside the body, and then put back in. It engrafts permanently, and its corrected descendants keep making the missing enzyme for years.
That's the foundation Live-cel is built on: harvest, correct, re...more
