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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
Beyond Activation: Why Building Brown Fat May Succeed Where Waking It Up Failed
Beyond Activation: Why Building Brown Fat May Succeed Where Waking It Up Failed
In the late 1980s and throughout the 1990s, several large pharmaceutical companies tried to develop obesity drugs that would acutely activate thermogenesis - the body’s process of dissipating energy as heat. The idea was to increase resting metabolic rate and turn stored fat into heat by stimulating the activity ...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
Fabry 101: What is Fabry and what causes it?
By Chris Hopkins, CEO of Glafabra Therapeutics. The author is an employee of Glafabra Therapeutics, which is developing an investigational cell-based therapy for Fabry disease.
Fabry disease is caused by loss of a single lysosomal enzyme, alpha-galactosidase A (α-Gal A), the result of an inherited pathogenic variant in the GLA gene. The absence of this enzyme leads the body to having no way to rem...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
