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Glafabra Therapeutics Completes FDA INTERACT Meeting for Its Live-cel Fabry Disease Gene Therapy, Clarifying Its Path to an IND
Glafabra
FDA’s written feedback generally noted that the nonclinical package supports a proposed exploratory clinical study, recommended prioritizing plasma lyso-Gb3 as the pharmacodynamic readout, agreed that assessing eGFR is reasonable while recommending at least two years of follow-up, said a within-subject exploratory design is reasonable, and advised that manufacturing knowledge may be leveraged acro...more
How Outpatient Conditioning Enabled Same-Day Discharge in the FACTS Trial
Same-day discharge isn't a footnote. For a patient and their family, it's the difference between one afternoon and a hospital stay.
Four of five FACTS trial patients received their conditioning regimen and went home the same day — because it used a gentler, non-myeloablative approach instead of bone-marrow-transplant-intensity chemotherapy. Glafabra built Live-cel around that outpatient approach f...more
What Conditions Is Fabry Disease Often Mistaken For? — DECODE No. 14
(Answer to the question last time on August 17: women — X-inactivation can leave female patients with near-normal enzyme levels on a standard blood test, so genetic testing of the GLA gene is the reliable way to diagnose them.)
Fabry's symptoms touch so many organ systems that it regularly gets mistaken for other conditions entirely — sometimes for years.
Name one condition Fabry disease is common...more
Glafabra's Japan Strategy: IFFD 2026 and BIO Japan
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
