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
How does a human-cell discovery platform find brown-fat-building compounds?
Finding the Right Lock and Key
To make a new medicine, we first need a drug target. Think of the target as a specific lock on or in a cell, and the drug as the key. When the key fits the lock, it triggers a specific reaction.
For Energesis, the goal was to find a lock that triggers human adult stem cells to turn into active brown fat cells.
Using Biologically Meaningful Cells
Cells grown in a lab ...more
Muscle Matters: The Lean-Mass Problem with Appetite-Based Weight Loss
GLP-1 receptor agonists have revolutionized obesity treatment, making significant weight loss pharmacologically achievable for millions of patients. However, these medications come with notable trade-offs. From a clinical perspective, one of the most concerning is the simultaneous loss of muscle mass. Studies show that when patients lose weight on GLP-1 medications, more than 30% of the lost ...more
Understanding Brown Fat: A New Path for Treating Obesity and Metabolic Health
Understanding Brown Fat: A New Path for Treating Obesity and Metabolic Health
Brown adipose tissue (BAT), commonly called brown fat, is a specialized type of body fat containing cells called brown, beige, or brite adipocytes. Unlike standard white fat - which primarily stores excess calories - brown fat cells are thermogenic, meaning they burn energy and dissipate it as heat. Brown fat is dyn...more
One Platform, Three Rare Diseases — DECODE No. 9
(Last time: five patients, five years, durable enzyme — the FACTS proof.)
Here's the twist we've been building toward. The approach behind Live-cel for Fabry isn't limited to one disease. Swap the corrected gene, keep everything else, and the same platform takes aim at two more inherited enzyme disorders — one that weakens muscle, one that affects the spleen, liver, and bone.
Can you...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 One Cell Behind Every Live-cel Treatment — DECODE No. 7
(Last time: gentle outpatient conditioning with melphalan.)
Every Live-cel treatment starts with a rare cell harvested from the patient — one that can rebuild the entire blood system and carry a corrected gene to the body for years. Scientists find it by a marker on its surface: a protein tag written as "CD" followed by a number.
Which cell — and which number? Drop your guess in the ...more
