Content reflects the views of the posting party and is provided for informational purposes only. It does not constitute a solicitation, recommendation, endorsement, or investment advice by BioTech Funding Portal. All content is subject to the portal’s Community Content Guidelines.
Where Would Live-cel Fit Among Today's Fabry Treatments?
Glafabra
Enzyme replacement therapy is the standard of care: an IV infusion about every two weeks, for life. Roughly 40% of ERT patients develop anti-drug antibodies over time, which can reduce its effectiveness, and ERT does not fully reach every tissue, notably the heart and nervous system. Oral chaperone therapy (migalastat) works only for the roughly 35-50% of patients with an amenable GLA variant. AAV...more
Meet Dr. Ronan Foley, MD: Co-Founder and Transplant Specialist
Dr. Ronan Foley, MD, is a Co-Founder of Glafabra and a transplant specialist.
With co-founder Dr. Jeffrey Medin, he co-led the Canadian FACTS trial (NCT02800070), which followed five Fabry patients for five years, including the non-myeloablative, melphalan-based conditioning that allowed 4 of 5 patients to go home the same day.
For diligence purposes: the clinician who led the pilot study's transp...more
What Are the Known Risks of Lentiviral Stem Cell Gene Therapy?
The main recognized risks of lentiviral HSC gene therapy are (1) insertional risk: the vector integrates into the genome, and in rare cases in the wider field, integration has been linked to abnormal cell growth; (2) conditioning risk: the chemotherapy used to make room for corrected cells carries its own side effects; and (3) procedure risk from stem cell collection and re-infusion. In FACTS, ins...more
Is There a Cure for Fabry Disease?
here is currently no approved cure for Fabry disease. Approved treatments, enzyme replacement therapy (an IV infusion about every two weeks) and, for patients with an amenable GLA variant, an oral chaperone (migalastat), manage the disease. Several gene therapy approaches aiming for durable enzyme production are in clinical development.
Interpretation: Live-cel (GT-GLA-S03) is one of those approac...more
Why Promising Medical Breakthroughs Don't Always Reach Patients
From laboratory discoveries to clinical trials, the path to developing new treatments is filled with scientific and technical challenges. Understanding those challenges helps explain why even the most promising research doesn't always lead to a new medicine.
It seems like every week we hear about a new scientific breakthrough that could completely change how we treat a devastating disease. For pat...more
Why Did FDA Recommend at Least Two Years of eGFR Follow-Up?
eGFR (estimated glomerular filtration rate) measures kidney function. Kidney involvement (Fabry nephropathy) is one of the main ways the disease progresses. In its INTERACT feedback, FDA recommended at least two years of eGFR follow-up for GT-GLA-S03.
Interpretation: eGFR changes slowly, so a short study can't tell a meaningful trend from normal variation. Two years is the minimum window to see wh...more
Why Is Plasma Lyso-Gb3 the Primary Readout, and What Can't It Tell You?
Lyso-Gb3 is the circulating derivative of Gb3, the lipid that builds up when alpha-Gal A is deficient, and the main blood biomarker used to track Fabry disease activity. In FDA's written INTERACT feedback, plasma lyso-Gb3 was recommended as the primary pharmacodynamic readout for GT-GLA-S03. In the FACTS trial, plasma lyso-Gb3 fell 48% vs. no-ERT baseline (p<0.0001) at 5-year follow-up, with 4 ...more
What We Heard at BIO Japan
Evidence: From October 7 to 9, Glafabra took part in BIO Japan in Yokohama, one of Asia's largest biotech partnering events. The team was there for two kinds of conversations: manufacturing and analytical development partners with experience in autologous lentiviral stem-cell therapy, and Japan-based pharma and academic groups working in lysosomal storage disorders. Both follow from FDA's INTERACT...more
