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How Did Gene Therapy Patients Go Home the Same Day? — DECODE No. 6
Glafabra
(Last time: lentiviral vectors, and why Live-cel can be re-dosed.)
Older cell-based gene therapies required hospital admission and weeks of recovery — a barrier so heavy many patients and doctors said no. In the FACTS trial, four of five patients did this same step and went home the same day.
What gentler approach made same-day discharge possible? Drop your guess in the comments belo...more
Why Can Some Gene Therapies Be Re-Dosed and Others Can't? — DECODE No. 5
(Last time: the biweekly ERT infusion schedule.)
Some gene therapies can only be given once — the immune system slams the door on a second dose. Live-cel can be given again when it's needed. The difference comes down to what carries the gene into the cell.
What kind of delivery system lets Live-cel be re-dosed? Drop your guess in the comments below.
Follow the whole series: ht...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
How Can Fixing a Few Cells Treat the Whole Body? — DECODE No. 3
(Last time: lyso-Gb3, the biomarker that tracks Fabry disease.)
Here's a beautiful shortcut in biology: to treat the whole body, you don't have to fix every cell. Fix some, and they secrete a helper the neighboring cells absorb and put to work — spreading the benefit outward.
There's a two-word name for this generous phenomenon. What is it? Drop your guess in the comments below.
&nbs...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
The top symptoms needing to be addressed for Fabry disease
We asked 51 people living with Fabry disease a single question: if a new treatment could improve just one symptom, which would matter most to your quality of life?
A snap shot of the results are in the figure and they matter to how we think about where a new therapy creates value.
Neuropathic pain of the feet and hands came first at 31%, followed by fatigue at 22% and gastrointestinal symptoms at ...more
Biotech Funding Portal debut at BIO
Biotech Social debuted the BioTech Funding Portal at the BLPN Field of Dreams Summit in San Diego at the BIO2026 meeting. Glafabra Therapeutics is proud to be one of the first companies on the platform.
Starting with a Testing the Waters campaign under Regulation Crowdfunding, retail and accredited investors alike can get an early look at companies they can follow before a formal raise opens.
Soo...more
