How a Medical First Fixes Genetic Disease in Living Patients
Scientists have achieved a medical first – successfully correcting a disease-causing genetic mutation in living patients. Beam Therapeutics announced early results from its Phase 1/2 trial showed that a single dose of its drug fixed a specific genetic error that causes a hereditary condition affecting both lungs and liver.
This milestone is the first time a technique called base editing has worked to correct a mutation in humans. The treatment acts like a molecular pencil, changing just one letter in the genetic code. Unlike many genetic therapies that add new genes to cells, this approach directly repairs the existing mutation at its source.
Beam tested the therapy in patients with alpha-1 antitrypsin deficiency (AATD), a condition that affects about 100,000 Americans. Currently, no other treatments address the disease’s its genetic cause. Patients eventually need lung transplants, liver transplants or both.
Roche Bets $5 Billion on Obesity Partnership with Zealand
“Best case scenario” — that’s how analysts at Jefferies described Roche’s newly inked deal with Zealand Pharma, signaling that the Swiss pharma giant is making a decisive move to challenge the dominance of Novo Nordisk and Eli Lilly in the multibillion-dollar obesity market.
In a $5.3 billion agreement, Roche has partnered with Denmark’s Zealand Pharma to co-develop and co-commercialize petrelintide, an experimental long-acting amylin analog for weight management. Unlike existing blockbuster GLP-1 receptor agonists like Novo’s Wegovy and Lilly’s Zepbound, amylin-based therapies offer a different mechanism of action — restoring sensitivity to leptin, the satiety hormone, to promote weight loss.
Roche’s entry into the space could shake up an increasingly crowded field. Novo and Lilly are setting record sales, but Roche is betting that alternative weight-loss mechanisms and better tolerability could give petrelintide a competitive edge.
Amylin-based therapies could be positioned as complementary to GLP-1 drugs or even preferred for patients struggling with tolerability issues like nausea and vomiting. Zealand has suggested that petrelintide could achieve GLP-1-like weight loss with fewer side effects. Jefferies projects peak sales of $10 billion globally.
Why FDA is Cracking Down on Dangerous Fat-Dissolving Injections
The FDA is cracking down on unapproved and potentially dangerous injections meant for dissolving fat. The unregulated aesthetic shots, sold under brand names Aqualyx, Lipodissolve, Lipo Lab and Kabelline — are promoted for fat reduction in areas like the chin, stomach, thighs and upper arms. Many contain phosphatidylcholine (PPC) and sodium deoxycholate (DC), ingredients that the agency warns pose serious health risks when used without proper regulatory oversight.
Some people who received these injections have reported permanent scarring, infections, skin deformities, cysts and painful nodules. The FDA issued warning letters to six companies, including Amazon, for selling these injections.
Currently, the only FDA-approved fat-dissolving injectable is Kybella, which is specifically indicated for reducing submental fat under the chin. The agency warns that no injectable fat-reduction product has been approved for use in other areas of the body.
How New Noninvasive Hearing Aid Uses Vibration to Improve Sound
Researchers at Wake Forest University School of Medicine have developed a flexible, Band-Aid-like hearing aid that could provide a less invasive solution for children with hearing loss.
Conductive hearing loss, which blocks sound transmission through the ear, most commonly occurs in childhood due to infections, blockages or structural abnormalities.
Current treatment options often involve invasive surgeries or implantable devices that can be especially challenging for pediatric patients. The new device creates tiny vibrations directly on the skin behind the ear to help sound reach the inner ear.
By using multiple tiny vibration points instead of a single one, the researchers found they could improve sound transmission more effectively. It's similar to how multiple speakers can create a richer sound than a single speaker.
In a study of 10 participants, the device significantly improved sound perception. The researchers tested two different designs of these vibration points, with the stacked design showing particular promise. Participants reported clearer sounds and better ability to detect even soft noises.