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Why Pre-IND Is the Best Time to Get Involved With Glafabra
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
Meet Glafabra's CEO & Co-Founder, Chris Hopkins
Meet the person leading this: Chris Hopkins, CEO & Co-Founder.
Chris is a geneticist, biochemist, and entrepreneur who's spent his career at the intersection of science and company-building. He guided the acquisition of Knudra Transgenics by InVivo Biosystems, raised over $5M in venture capital and $7M in non-dilutive SBIR funding, licensed CRISPR technology in 2014 — shortly after it was inve...more
Why Do All Daughters of an Affected Father Inherit Fabry Disease? — DECODE No. 11
(Answer to the question last time on August 6: Fabry disease is X-linked — the GLA gene sits on the X chromosome, so the pattern plays out differently for men and women.)
Here's the quirk that surprises people. A father with Fabry disease passes the condition to every single one of his daughters — but to none of his sons.
Why does it break down exactly that way? Drop your guess in the comments bel...more
One Month Into Testing the Waters: What We've Learned
A month ago, this was a handful of posts and a hope that people would care about a disease most of them had never heard of. Since then: real questions on this portal, real conversations, and people who registered interest before we'd even finished explaining why.
That's not nothing. Rare disease work runs on people willing to pay attention before there's a finished product to point to.
Thank you f...more
