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Sarepta's Duchenne Muscular Dystrophy (DMD) gene therapy has been granted the much wider label. This news comes just about a week after Pfizer, which was fast on Sarepta’s heels with their own gene therapy for DMD, suffered a key Phase III trial failure.

Precision Oncology Today

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Into the Dark Unknown

The dark genome—it sounds like a work of science fiction. For a dozen or so biotech companies, investigating this mysterious genetic “dark matter” is at least as exciting as anything dreamt up by a movie studio. Once dismissed as “junk DNA,” the dark genome is increasingly looking like an untouched resource that can provide answers to fundamental processes in life.
Weight loss pills and unhealthy dieting with medication concept

Precision Medicine for Obesity: Targeting a Multifactorial Condition

A report published in 2023, estimates that over 40% of adults in the U.S. have obesity, defined as a body mass index (BMI) of...
Astronaut in the desert looking at the moon

New Frontiers in CDx Development

Where is the field of CDx development headed? Today, machine learning and artificial intelligence (AI) are beginning to make an impact on pharma-diagnostic co-development.

Trending on Inside Precision Medicine

Human pancreas, gall bladder organ model, 3d rendering.

Fractyl Health’s GLP-1 Gene Therapy Outperforms Semaglutide in Mice

Upon finishing his cardiology fellowship, Harith Rajagopalan, MD, PhD, founded Fractyl Health to develop transformative therapies that can prevent and reverse obesity and type 2 diabetes rather than manage them medically. Rajagopalan said that the two products being produced at Fractyl stay true to that vision--what's going wrong in the body and how to fix it in a way that can have long-lasting benefits for people.
Man with Duchenne muscular dystrophy sitting in a motorized wheelchair using power controller with degenerated hands

Sarepta Wins Key Label Expansion for DMD Gene Therapy, in Wake of Pfizer Failure 

Sarepta's Duchenne Muscular Dystrophy (DMD) gene therapy has been granted the much wider label. This news comes just about a week after Pfizer, which was fast on Sarepta’s heels with their own gene therapy for DMD, suffered a key Phase III trial failure.
Parkinson's disease. 3D illustration showing neurons containing Lewy bodies small red spheres which are deposits of proteins (alpha-synuclein) accumulated in the brain cells.

Vaxxinity’s Immunotherapy Meets Phase I Goals in Parkinson’s Patients

Currently, there are no approved therapies that can change the course of Parkinson's disease. Researchers are developing immunotherapies, such as monoclonal antibodies and vaccinations, for clinical use to target α-synuclein and improve Parkinson's symptoms. In phase I clinical trials, monoclonal antibodies that target α-synuclein have shown promise but have failed in phase II trials. Vaxxinity has developed a vaccine-based immunotherapy for Parkinson's disease.

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