Skip to main content

Expensive gene therapy receives its first patient in a commercial treatment

CRISPR
A patient with an extremely rare immune disease has been treated with commercial gene therapy for the first time, GlaxoSmithKline, the company behind the therapy, told MIT Technology Review on Tuesday. The treatment come almost a year after the therapy was approved for sale in Europe.

Known as Strimvelis, the therapy treats a rare inherited immune deficiency by fixing a problem within the patient’s DNA.

Gene therapy has been used extensively in clinical trials but has had a slow start commercially. This is only the second commercial use of gene therapy, the first of which was with a drug called Glybera in 2015.

Gene therapies are relatively complex compared to standard pills, often requiring invasive procedures. The conditions they treat are also exceptionally rare — each year only about fifteen children in Europe and twelve in the United States are affected by ADA-SCID, the disease Strimvelis treats. Due to their complexities, the drugs are also some of the most expensive in the world. Strimvelis is priced at $648,000. Glybera was listed at $1 million. Uniqure, the company behind Glybera, announced last month that it would remove its drug from the market due to lack of demand.

The project lead for Strimvelis, Jonathan Appleby, told Technology Review that the year-long delay between European approval and treatment was due to cross-border reimbursement for the therapy, which is only available in Milan, Italy.

“It’s definitely a bad sign for patients,” said Casey Quinn, a health economist with a focus in European drug pricing. “It remains to be seen whether this represents some kind of watershed, or it will take just as long to go from one [patient] to two?”

Strimvelis treatment is complex, necessitating a “specialized environment,” according to Lucia Monaco, chief scientific officer at Fondazione Telethon, the institution that initially developed the therapy. During the procedure, doctors remove cells from a patient’s bone marrow. The cells are modified externally before being replaced through an infusion into the patient’s veins.

Though commercial use has been slow for gene therapies, many patients continue to undergo treatment through clinical trials. Spark Therapeutics, a United States-based biotech company, is scheduled to request approval for commercial sale of a gene therapy that treats a condition that causes blindness later this year.

Editors' Recommendations

Sleep Number’s New 360 Smart Bed monitors and improves sleep health as you age
The Sleep Number New 360 Smart Bed set up in a bedroom

Today at CES 2022, Sleep Number, a leader in sleep health and research, has unveiled the latest entry in its smart bed lineup, the new 360 Smart Bed. With the assistance of A.I. and machine learning, this new smart bed has unique features that will help it monitor and improve your sleep health as you age.

Sleep is a significant part of our lives and vital to our general health. Knowing that, Sleep Number created it smart bed years ago to help track sleeping patterns. This new iteration of the smart bed has even more features to track sleep patterns, as well as predict issues and react when they arise.

Read more
French startup Circular unveils promising Oura fitness-tracking competitor
Movano Ring. Credits: Movano official.

Several smartwear products took the center stage at CES 2022. Apart from the heavy hitters such as Garmin and Skagen, a Movano ring was also unveiled, with a design that specifically has female users in mind. Finally, a French company called Circular also unveiled its first product, simply called the Circular Ring. The Circular Ring has changeable outer shells for user customization. The ring is extremely lightweight at only 4 grams. It is also water-resistant up to 5 meters. It also offers up to 22 days of built-in data storage without a Bluetooth connection. The biocompatible resin-based product will be available in different sizes.
The circular ring could give stiff competition to the Movano ring in the coming years. Movano official.
Tracking and features
A 14-day calibration period begins as soon as the user wears the ring for the first time. During this period, the ring establishes the user's baseline health parameters, which helps it to give them personalized and actionable recommendations to make healthy changes in their lives. Circular tracks multiple health metrics, including temperature, heart rate variability, energy levels, and VO2 Max. Circular has a dual PPG sensor in its ring combined with the Circular app that allows it to read heart signals and blood oxygen. It detects if the heart rate shows any signs of cardiac arrhythmia, and if the heart rhythm is steady. The data tracked is purported to be clinically accurate and can be shared with a professional for further advice. Wearing it both during the day and the night can help Circular to correlate the data using machine learning.Circular also seems to be extremely efficient while tracking sleep. It can tell a user how much time they spend in each sleep stage -- light sleep, deep sleep, REM sleep, and when they are lying awake. More importantly, it also calculates how efficiently a user has slept. The app shares data on a number of important criteria to examine sleep quality, including time taken to fall asleep, sleep debt, real sleep time, disturbances while sleeping, etc. It also provides silent vibrations to users in their light sleep stage if they wish to be awakened, without disturbing their surroundings.
Price and availability 
A single charge of the ring gives it four days of battery life. A single charge from 0% to 100% can be completed in one hour. Pre-orders, which will begin soon, will run until February 27, 202. The device will have a cost of 259 euros ($293) during that time. Starting February 28, the price will jump to 289 euros ($327). Shipping and other details are expected to be announced soon by the company.

Read more
How taking your blood pressure is about to be as easy as taking your heart rate
Valencell Blood Pressure monitoring.

“We think blood pressure monitoring will change more in the next five years than it has in the last one hundred years,” Ryan Kraudel, Vice President of Marketing at biometric sensor maker Valencell, said as we talked about the company’s innovation in blood pressure monitoring over Zoom for CES 2022.

Such a bold statement could often be glossed over as hyperbole, but the thing is, after hearing what Valencell is working on, his prediction doesn’t sound farfetched at all. Its cuff-less, calibration-free blood pressure monitor could make taking your blood pressure as simple as measuring your heart rate is with a smartwatch today.
What is Valencell working on?
When you want to measure blood pressure, you use either one of those familiar cuffs or a slightly higher-tech cuff-less product to do so. Both are clearly medical devices, though, they are rarely particularly portable and certainly not friendly to use in that they’re noisy and somewhat intrusive. The cuff-less versions are better, but still regularly need to be paired with a cuffed version to make sure they remain accurate. Valencell’s innovative new technology is very different.

Read more