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LATEST NEWS
Orforglipron Weight-Loss Pill Trial Results: Eli Lilly Reports 12.4% Body Weight Reduction, Shares Fall 15%
Eli Lilly’s once-daily oral weight-loss pill, orforglipron, helped patients lose 12.4% of their body weight in a Phase 3 trial, but fell short of rival Wegovy’s results. The announcement sent Lilly shares down 15%, the company’s steepest drop in 25 years, despite meeting all primary and secondary endpoints and showing improvements in heart health markers.
Aug 11


Eli Lilly Acquires Verve Therapeutics for $1.3 Billion to Advance Gene Editing for Heart Disease
Eli Lilly has announced its $1.3 billion acquisition of Verve Therapeutics, a leading biotech company specializing in gene editing for cardiovascular diseases. This strategic move aims to accelerate the development of one-time gene therapies using base editing technology, targeting high-risk heart disease patients. The deal marks a significant step forward in Eli Lilly’s expansion into precision genetic medicine.
Jul 20


Capricor's Shares Drop After FDA Rejects Deramiocel Cell Therapy for Heart Disease in DMD
Capricor Therapeutics experienced a major setback after the FDA rejected its cell therapy, Deramiocel, for treating heart disease in Duchenne muscular dystrophy (DMD) patients. The decision led to a 30% drop in Capricor’s stock and highlighted the need for more efficacy data. Deramiocel, which uses cardiac-derived stem cells, aims to regenerate heart tissue and slow cardiomyopathy progression in DMD. Capricor plans to resubmit its application with additional clinical data lat
Jul 16


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Eli Lilly Acquires Verve Therapeutics for $1.3 Billion to Advance Gene Editing for Heart Disease
Eli Lilly has announced its $1.3 billion acquisition of Verve Therapeutics, a leading biotech company specializing in gene editing for cardiovascular diseases. This strategic move aims to accelerate the development of one-time gene therapies using base editing technology, targeting high-risk heart disease patients. The deal marks a significant step forward in Eli Lilly’s expansion into precision genetic medicine.
Jul 20


Base Editor Gene Therapy for CPS1 Deficiency Treats Baby’s Rare Disorder
In a historic first, doctors used base editor gene therapy to treat a baby with CPS1 deficiency, a rare genetic liver disorder. The personalized treatment—delivered using lipid nanoparticles—led to improved ammonia levels and reduced medication needs. Unlike traditional CRISPR, base editing made precise DNA changes without cutting, offering a safer, more targeted option. This breakthrough marks a major step forward in treating rare diseases with gene-editing technology.
Jul 1


Light-Induced Gene Therapy Using Nanoparticles Targets and Destroys Cancer Cells' Mitochondria
A groundbreaking new approach in cancer treatment has emerged, utilizing light to disable the mitochondria, the energy-producing...
Feb 8


Gene Therapy Restores Enzyme Levels to Treat Hypophosphatasia - A More Permanent Treatment
Hypophosphatasia (HPP), or "soft bone disease" is a rare genetic disorder that impacts bone and tooth development, leading to weak bones...
Feb 4


Gene and Cell Therapy Clinical Trials to Notice in 2025
Several clinical trials in 2025 are pushing the boundaries of gene and cell therapy, aiming to address a range of complex diseases. Below...
Jan 31


Novartis Announces Positive Results for Zolgensma Trial in Older Children with Spinal Muscular Atrophy
Novartis's gene therapy, Zolgensma, is already approved in the U.S. for children under the age of two with spinal muscular atrophy (SMA),...
Jan 4


Five-Year Follow-Up Shows Sustained Visual Improvements in LHON Patients Treated with AAV Gene Therapy
A recent international study has shown that lenadogene nolparvovec, a gene therapy based on adeno-associated virus (AAV), can provide...
Dec 27, 2024


CRISPR Gene-Editing Therapies Make Strides, but Cost and Accessibility Remain Key Challenges
As CRISPR-based therapies edge closer to becoming mainstream, groundbreaking treatments for blood disorders and cancer are making their...
Dec 19, 2024


Sickle Cell Disease Gene Therapy is Now COVERED under Medicare and Medicaid
Sickle cell disease (SCD), which affects over 100,000 people in the U.S., is about to see a major shift in treatment accessibility. With...
Dec 5, 2024


StitchR: New Technology for Treating Genetic Diseases with Large Genes
Gene therapy has shown promise in treating many genetic diseases, but some conditions, like muscular dystrophy, involve very large genes...
Nov 18, 2024


AI Creates Precise DNA “Switches” for Gene Therapy - A Step Closer to Targeted Treatments
Imagine controlling genes with the precision of a light switch— controlling specific parts of the body as you will. That’s what the...
Oct 23, 2024


6-Year Update on Gene Therapy for CALD: Positive Results with Continued Caution
A recent 6-year checkup on boys treated with gene therapy for cerebral adrenoleukodystrophy (CALD) reveals lasting benefits for most...
Oct 19, 2024


Positive Phase 2 Results for Gene Therapy in Parkinson’s Disease
In exciting news for Parkinson’s disease research, MeiraGTx has announced positive results from a Phase 2 clinical bridging study of...
Oct 17, 2024


Magnets: The New Way to Control the Brain?
Imagine switching brain circuits on and off using only magnetic fields. Researchers have just developed a cutting-edge gene therapy that...
Oct 14, 2024


New Gene-Editing Technique: A Better Alternative to CRISPR for Fixing Cystic Fibrosis Mutation
A team from the Broad Institute of MIT and Harvard, along with Harvard University and the Howard Hughes Medical Institute, has made...
Sep 10, 2024


Gene Therapy: A New Era for Diabetes Treatment
The fight against diabetes is being revolutionized with an innovative approach from Genprex, with its gene therapy candidate, GPX-002....
Sep 3, 2024


Afami-cel: First T-Cell Therapy Approved for Solid Tumors
On August 2nd, 2024, the FDA granted accelerated approval for afami-cel -- the first gene-engineered T-cell Therapy for advanced...
Aug 27, 2024


New Gene Therapy Breakthrough Paves Way for Better Treatment of Brain Disorders
In a project funded by the National Institute of Mental Health (NIMH) and the National Institute of Neurological Disorders and Stroke...
Aug 19, 2024


FDA Expands Approval of ELEVIDYS: A Breakthrough Gene Therapy for Duchenne Muscular Dystrophy
On June 20th, 2024, the FDA expanded its approval of ELEVIDYS , an innovative AAV-based gene therapy developed by Sarepta Therapeutics ,...
Jun 19, 2024


FDA Approves BEQVEZ: A Breakthrough One-Time Gene Therapy for Adults with Hemophilia B
Gene therapy is transforming the landscape of treatment options, and the recent FDA approval of BEQVEZ marks a significant milestone for...
May 1, 2024
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