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.
What's New in Glafabra's Testing the Waters Campaign
Glafabra
One month into Testing the Waters. Here's what's changing for month two.
More real faces. Starting this Tuesday, we're introducing you to the people building Glafabra — one team member at a time, starting with our CEO.
DECODE keeps going, but simpler: no more separate answer-comment days later. Each new riddle now opens with last time's answer, right in the post.
And more of Glafabra's actual stor...more
How Is Fabry Disease Inherited? — DECODE No. 10
(Answer to the question last time on August 3: Pompe disease and Gaucher disease — the same Live-cel platform, one gene swapped in, reaches both.)
Fabry disease runs in families, but not the way most genetic conditions do. One chromosome carries the whole story — and it changes who's affected depending on whether it's inherited from mom or dad.
What inheritance pattern is this? Drop your guess in ...more
Five Patients, Five Years: The Fabry Gene Therapy Data — DECODE No. 8
(Last time: CD34+ hematopoietic stem cells, the engine of Live-cel.)
Here's the number that anchors everything. In the FACTS trial, this many patients were followed for five years — and every one still had therapeutic enzyme levels at the end, with zero product-attributable serious adverse events.
How many patients? Drop your guess in the comments below.
Follow the whole serie...more
The Treatment Routine Fabry Patients Call 'Two-Week Increments' — DECODE No. 4
(Last time: cross-correction — fix some cells, they help the rest.)
Patients describe it as "living in two-week increments." It isn't a figure of speech — it's a calendar that never ends. Every other week. For life.
What treatment routine are they describing? Drop your guess in the comments below.
Follow the whole series: https://bit.ly/Glafabra-TTW
@Brian Christie @Nev...more
The Blood Marker That Tracks Fabry Disease — DECODE No. 2
(Last time: the missing enzyme in Fabry is alpha-galactosidase A.)
When that enzyme goes missing, one particular molecule builds up in the blood and becomes the fingerprint doctors track to measure the disease. Cut it roughly in half, and you've shown a therapy is working.
What's this telltale biomarker? Drop your guess in the comments below.
Follow the whole series: https://b...more
Which Enzyme Is Missing in Fabry Disease? — DECODE No. 1
I'm a single enzyme. When I go missing, a fatty molecule quietly piles up inside your cells — silently, for years — until it starts to damage kidneys, heart, and nerves.
My name begins with the first letter of the Greek alphabet.
Who am I? Drop your guess in the comments — the answer posts with DECODE No. 2.
Follow the whole series: https://b2f.bz/5k9n3
@Brian Christie @Neva W...more
LSDs + protein deficiency: a $13B Market and Rising
The Fabry Market Is Bigger Than the Headlines, and the Platform Behind It Is Bigger Still
Every few months a new acquisition reminds the market that lysosomal storage disorders are quietly one of the most durable franchises in rare disease. BioMarin's $4.8B acquisition of Amicus in April 2026 was the latest. So it is worth doing the arithmetic out loud, from primary sources, on exactly how large t...more
Glafabra Therapeutics Receives FDA Acceptance for Face-to-Face INTERACT Meeting for GT-GLA-S03, Its Cell-Based Gene Therapy for Fabry Disease
Glafabra granted a face-to-face INTERACT meeting slated to occur July 16, 2026.
We will be discussing with the FDA key questions that will set the development path of our GT-GLA-S03 cell-based gene therapy for Fabry in its journey towards the market.
This milestone achievement was only made possible with the amazing contributions of the internal team and our contractors. Many thanks to Elizabeth W
- 1
