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BioWorld - Wednesday, December 10, 2025
Home » Topics » Drugs » Gene therapy

Gene therapy
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mRNA vaccines are composed of messenger RNA encapsulated in lipid nanoparticles
Drug design, drug delivery & technologies

Animosity toward mRNA COVID vaccines puts basic science at risk

Sep. 16, 2025
By Mar de Miguel
No Comments
An ongoing concern for scientists is that there will be across-the-board funding cuts. This is already happening in mRNA research, where reductions affected coronavirus-related projects. During the pandemic, efforts focused on this pathogen, and once the health emergency was over, grants for antivirals were eliminated. However, these drugs could stem future outbreaks. Despite the cuts, recent research continues to demonstrate the potential of mRNA, not only for the development of antivirals, but also for obtaining more effective and longer-lasting vaccines.
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Brain and DNA

Capsida's gene therapy trial paused upon first patient's death

Sep. 11, 2025
By Karen Carey
No Comments
Two months after starting the phase I/II Synrgy trial with its gene therapy, CAP-002, enrolling 12 pediatric patients with rare disease STXBP1 encephalopathy, Capsida Biotherapeutics paused the study following the death of the trial’s first patient.
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DNA and cancer cells
Infection

Gene therapy kills KSHV-infected cells and halts tumor growth

Sep. 10, 2025
No Comments
Researchers from the University of California, Davis have designed a novel gene therapy vector that selectively targets and kills cells infected with Kaposi’s sarcoma-associated herpesvirus (KSHV).
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Neurology/psychiatric

MAPT-directed miRNA broadly reduces tau pathology in Alzheimer’s

Sep. 10, 2025
No Comments
Researchers at Sanofi SA have developed a promising gene therapy approach targeting the microtubule-associated protein tau (MAPT) for the treatment of Alzheimer’s disease.
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Art concept for gene therapy research
Endocrine/metabolic

Remedium and Lilly partner on gene therapies for obesity and diabetes

Sep. 9, 2025
No Comments
Remedium Bio Inc. has entered into a multitarget research and development collaboration with Eli Lilly & Co. to advance gene therapies for type 2 diabetes and obesity using Remedium’s Prometheus dose-adjustable gene therapy platform.
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Neurology/psychiatric

Evaluation of AAV-delivered miRNA therapy for myotonic dystrophy type 1

Sep. 9, 2025
No Comments
Myotonic dystrophy type 1 (DM1) is a rare, progressive genetic disease that causes severe muscle weakness and other debilitating symptoms, such as compromised respiration and cardiac conduction abnormalities. No disease-modifying therapy exists for DM1, so care focuses on managing symptoms like arrhythmia, myotonia, hypertension, cataracts, respiratory issues and sleep disorders.
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Eye with digital overlay
Ocular

Grant to advance Axovia’s AXV-101 for BBS1 sight loss

Sep. 5, 2025
No Comments
Axovia Therapeutics Inc. has been awarded a new $1.0 million grant by the nonprofit organization A Race Against Blindness to support the clinical development of AXV-101, an investigational gene therapy aimed at combating childhood blindness due to retinitis pigmentosa caused by Bardet-Biedl syndrome 1 (BBS1).
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Ear, nose & throat

Gene therapy rescues hearing loss in murine model of CLIC5 deafness

Sep. 4, 2025
No Comments
Adeno-associated virus (AAV)-based gene therapy is considered a promising strategy to treat hearing loss. However, its clinical application is limited by the genetic heterogeneity of hereditary hearing loss, requiring gene-specific analysis and approach optimization for broader treatment applications.
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DNA double helix illustration with section being removed in red
Cardiovascular

Editas Medicine nominates lead in vivo development candidate

Sep. 2, 2025
No Comments
Editas Medicine Inc. has nominated a lead in vivo development candidate, EDIT-401, a potential one-time therapy designed to significantly reduce LDL cholesterol (LDL-C) levels. The in vivo gene editing medicine is designed to treat hyperlipidemia by directly editing the LDLR gene to increase LDLR protein expression and reduce LDL-C levels.
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Rocket’s RP-A501 back on track as FDA lifts hold on Danon trial

Aug. 20, 2025
By Jennifer Boggs
No Comments
With a few tweaks to the protocol, Rocket Pharmaceuticals Inc.’s phase II trial testing RP-A501 in Danon disease is expected to resume following the lifting of the clinical hold, issued by the U.S. FDA in May in response to the death of a patient in the pivotal gene therapy study.
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