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genengnews.com /2 weeks ago

ASGCT 2026: Beverly Davidson Offers Vehicle and Route for Huntington’s Disease Gene Therapy

Beverly Davidson, PhD, chief scientific strategy officer at Children's Hospital of Philadelphia, shares her research including a novel gene therapy approach for Huntington's diseas...

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bioengineer.org /2 weeks ago

rAAV8 HMR-001 Boosts Liver Gene Therapy in Hemophilia

In a groundbreaking advancement for gene therapy, researchers have unveiled two novel bioengineered AAV8 vectors—HMR-001 and its codon-optimized counterpart HMR-001z—that demonstra...

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hearingreview.com /4 weeks ago

Gene Therapy for Inherited Deafness Shows Lasting Hearing Restoration

A multicenter study found a one-time treatment targeting the OTOF gene restored hearing in most participants, with effects lasting up to 2.5 years.

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genengnews.com /4 weeks ago

Top 10 Best Selling Gene Therapies

As more products reach the market, therapy developers partner with researchers and regulators to deliver N-of-1 treatments to patients. The post Top 10 Best Selling Gene Therapies...

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neurosciencenews.com /4 days ago

Gene Therapy Proven to Shield Brain from TDP-43 Damage

An experimental gene therapy called SynCav1 can protect the central nervous system from the cognitive decline linked to TDP-43-related proteinopathy, a primary driver of FTD, ALS,...

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bioengineer.org /1 month ago

Non-Viral Large DNA Integration in Human T Cells

A groundbreaking advancement in gene editing heralds a new era for immunotherapy, addressing the longstanding challenges of integrating large DNA sequences into primary human T cel...

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genengnews.com /4 weeks ago

Going Non-Viral: Gene Delivery Enters Its Translation Era

Advanced non-viral gene delivery systems are expanding the range of indications and therapy modalities possible for the new generation of genetic medicines. The post Going Non-Vira...

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drugs.com /1 month ago

First Gene Therapy for Genetic Hearing Loss, Otarmeni, Gains FDA Approval

MONDAY, April 27, 2026 — In a historic leap for medical science, the U.S. Food and Drug Administration (FDA) has greenlit the first-ever gene therapy to treat a rare form of inheri...

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nanowerk.com /1 month ago

Building a better delivery system for gene editing machines by re-engineering the cellular factory

A new study improves gene editing delivery by engineering the human cells that produce virus-like particles, boosting the potency of these key therapeutic vehicles.

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genengnews.com /1 month ago

Genetic Medicine Delivery Enhanced by Producer Cell Modifications

A new platform based on genome-wide screening systemically identifies which genes drive or block particle assembly to engineer cells that yield more potent delivery vehicles. The...

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bioengineer.org /1 month ago

Revolutionizing Gene Editing: Enhancing Delivery Systems Through Cellular Factory Re-Engineering

Gene editing represents one of the most transformative advances in modern medicine, offering the potential to correct genetic diseases at their source. However, a major obstacle in...

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fightaging.org /1 month ago

NPPA Gene Therapy to Encourage Greater Regeneration Following Heart Attack

Researchers here describe a novel approach to encourage greater regeneration in heart tissue following the injury and lost function incurred during a heart attack. Their work falls...

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biopharmadive.com /3 weeks ago

With $97M, Latus pursues a different kind of Huntington’s gene therapy

The therapy is part of a plan to develop gene therapies that can be efficiently delivered at lower doses, enabling them to be used against more than just “ultra-rare” conditions.

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fiercebiotech.com /1 month ago

Regeneron ushers in new genetic medicine era with groundbreaking gene therapy approval

Travis Smith was a very serious baby. Born completely unable to hear, his mother, Sierra, struggled to see his personality shine through his mute world.

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genengnews.com /4 weeks ago

Gene Editing at Scale, Clinic Seeks Generalizable Therapies

What’s most exciting about CRISPR is its potential to shift medicine from managing disease to directly correcting its root cause. End-to-end pipelines and regulatory advances aim t...

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futurism.com /1 week ago

Gene Therapy Causes Patient to Grow Alarming Tumor

Tinkering with the genetic code of a living human is risky. The post Gene Therapy Causes Patient to Grow Alarming Tumor appeared first on Futurism.

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arstechnica.com /1 month ago

Clinical trial shows gene editing works for β-Thalassaemia, too

Improved gene editing process reactivates the fetal version of a hemoglobin gene.

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labiotech.eu /1 month ago

The future of cell & gene therapy: Key trends to watch

Cell and gene therapies are moving towards correcting root causes of diseases. Let's take a look at future cell and gene therapy trends. The post The future of cell & gene ther...

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drugs.com /1 month ago

Gene Cure For Inherited Deafness Effective, Long Lasting, Clinical Trial Finds

THURSDAY, April 23, 2026 — A new gene therapy to treat inherited deafness produces a lasting cure, a new international study has found. The treatment, which targets the OTOF gene,...

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bioengineer.org /3 weeks ago

Effective AAV Gene Therapy in Late-Stage Lysosomal Disease

In a groundbreaking advancement in the field of gene therapy, researchers have demonstrated a significant clinical response following systemic administration of adeno-associated vi...

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jdsupra.com /2 weeks ago

Federal Circuit Confirms Patent Eligibility of Gene Therapy Patent in Regenexbio Inc. v. Sarepta Therapeutics

Genetically engineered cultured host cells containing recombinant nucleic acids useful for gene therapy are patent-eligible for being markedly different from anything occurring in...

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genengnews.com /2 weeks ago

Acute Myeloid Leukemia Therapy Improved by CRISPR Stem Cell Transplant

A Phase I/II clinical trial shows that a stem cell transplant that removes CD33 from donor cells using CRISPR can prevent cancer recurrence. The post Acute Myeloid Leukemia Therapy...

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hearingreview.com /1 month ago

FDA Grants Accelerated Approval to Otarmeni Gene Therapy for OTOF-Related Hearing Loss

The adeno-associated virus vector-based gene therapy is indicated for pediatric and adult patients with severe-to-profound hearing loss due to biallelic OTOF gene variants.

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genengnews.com /3 weeks ago

G-Link CAR-T Delivery Platform Showcased at ASGCT

The modular plug-and-play protein adapter developed in collaboration with Menachem Rubenstein, PhD, of the Weizmann Institute allows drug developers to cap and retarget existing le...

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