Bio Pharma Dive

JULY 21, 2021

The approval of Skysona for a genetic brain disease is a milestone for one of gene therapy's pioneering companies, but isn't expected to turn Bluebird's financial fortunes around.

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Bio Pharma Dive

APRIL 1, 2022

Taysha Gene Therapies is narrowing its research efforts and cutting around 35% of its workforce, the latest in a series of layoffs announced by makers of genetic medicines.

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Pharmaceutical Technology

OCTOBER 19, 2022

Eli Lilly and Company has signed a definitive agreement for the acquisition of all outstanding shares of precision genetic medicine firm Akouos for a total deal value of up to nearly $610m or up to $15.50 Akouos focuses on developing adeno-associated viral gene therapies to treat inner ear conditions, including sensorineural hearing loss.

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Pharmaceutical Technology

DECEMBER 23, 2022

Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new genetic medicines. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The companies entered the initial agreement in September last year. By Cytiva Thematic.

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Scienmag

JUNE 19, 2025

In a groundbreaking study that reshapes our understanding of the genetic determinants of serum lipid levels, a consortium of international researchers has unveiled novel genetic loci influenced by the complex interplay between genetic variants and psychosocial factors.

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Genetics Genomics Genome Gene 59

Scienmag

JUNE 17, 2025

The landscape of gene therapy is undergoing a revolutionary transformation fueled by the convergence of synthetic biology, artificial intelligence, and biomedical engineering. Unlike traditional gene therapies that often rely on static expression systems, these circuits enable real-time, fine-tuned control of gene dosage.

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Genetics Gene Therapy Gene Expression Gene 59

Scienmag

JUNE 16, 2025

This novel condition, termed DIAL syndrome, has been identified by an international consortium of cancer genetics experts spearheaded by researchers at the University of Birmingham, with funding support from Cancer Research UK. Detailed cellular analyses unveiled the fundamental biological role of the DIAPH1 protein in DNA repair.

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Pharmaceutical Technology

OCTOBER 25, 2022

Ast ellas Pharma has announced plans to make a strategic investment to back the development of Taysha Gene Therapies’ adeno-associated virus (AAV) development programmes for Rett syndrome and giant axonal neuropathy (GAN). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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AuroBlog - Aurous Healthcare Clinical Trials blog

JUNE 25, 2024

A woman in Colombia with rare genetic resistance to Alzheimer’s disease may have more company than scientists thought.

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Pharmaceutical Technology

JUNE 12, 2025

June 12, 2025 Share Copy Link Share on X Share on Linkedin Share on Facebook Stargardt disease is a genetic retinal condition, resulting in progressive loss of vision and blindness. SpliceBio has closed a Series B financing round, raising $135m for the clinical development of SB-007, its gene therapy candidate for Stargardt disease.

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Bio Pharma Dive

APRIL 5, 2023

The deal hands Ginkgo technology for discovering and engineering capsids — the outer shell that protects the helpful genetic material in gene therapies.

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Gene Therapy Gene Engineer Genetics 259

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 9, 2024

The convergence of gene therapies and clinical research is pushing the boundaries of what’s possible in ophthalmic care, offering hope for more effective treatments and potential cures for a range of vision-threatening conditions.

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Bio Pharma Dive

FEBRUARY 23, 2023

Graphite Bio and Sangamo are stopping work on their respective sickle cell gene therapies, while Intellia revealed partner Novartis ended development of its genetic treatment for the blood disease.

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Gene Therapy Gene Development Genetics 261

Bio Pharma Dive

FEBRUARY 25, 2021

Sean Nolan and Joe Nolan, former leaders at the Zolgensma developer, are behind Jaguar Gene Therapy, which plans to research treatments for a rare metabolic disease as well as genetically linked autism and diabetes.

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Gene Therapy Gene Genetics Development 256

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 22, 2024

(Richard Jones/Science Photo Library/Getty Images) Scientists have discovered hundreds of genes that could potentially promote cancer, new research shows. Cancer is typically triggered by some kind of change to our genetic coding that interferes with a cell’s ability to manage its growth.

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AuroBlog - Aurous Healthcare Clinical Trials blog

DECEMBER 21, 2022

Scientists have found an extremely subtle twist in the genetics of aging cells, one that seems to make them increasingly less functional as time goes on.

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XTalks

NOVEMBER 14, 2024

PTC Therapeutics has gained US Food and Drug Administration (FDA) approval for its new gene therapy, Kebilidi (eladocagene exuparvovec), for treating aromatic L-amino acid decarboxylase (AADC) deficiency. The FDA requires a confirmatory study to validate these findings, which is ongoing.

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Bio Pharma Dive

JULY 28, 2023

The British pharma is deepening its investment in genetic medicine through a deal for a group of Pfizer’s gene therapy delivery tools, while the New York giant cuts back.

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Bio Pharma Dive

DECEMBER 15, 2020

The acquisition marks a significant bet by Lilly on gene therapy's potential to address tough-to-treat neurological disorders like Parkinson's disease.

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Bio Pharma Dive

APRIL 6, 2022

Facing mounting competition to its top-selling drug Eylea, Regeneron is teaming up with ViGeneron to develop a genetic medicine for an inherited retinal disease.

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Bio Pharma Dive

APRIL 25, 2024

“With each passing year, we're more committed to becoming a serious player in the genetic medicine space,” a Regeneron executive said.

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Gene Editing Gene Genetics Medicine 307

Bio Pharma Dive

JUNE 15, 2023

Lilly will pay the genetic medicine specialist $60 million upfront to work together on a treatment targeting a risk factor known as Lp(a).

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Gene Editing Gene Genetics Medicine 322

Pharmaceutical Technology

MAY 4, 2023

Forge Biologics and global life sciences company Labcorp have announced a strategic adeno-associated virus (AAV) gene therapy development and manufacturing collaboration. This will improve accessibility to services for AAV-mediated gene therapy programmes. Topic sponsors are not involved in the creation of editorial content.

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AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 17, 2024

We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics. In […]

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Pharmaceutical Technology

JULY 7, 2022

Genetic mutations, both germline and acquired, are behind a large proportion of the most debilitating and sometimes life-threatening human diseases. Recent years have seen a steadily growing number of approvals for cell and gene therapies, which has spurred on the community to continue innovating in this space.

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Bio Pharma Dive

MAY 27, 2021

Safety monitors cleared the company to test a higher dose after reviewing data from 10 patients, providing some hopeful news after recent setbacks for experimental drugs targeting the genetic disease.

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Bio Pharma Dive

DECEMBER 8, 2023

million, while a competing genetic medicine also cleared Friday is priced at $3.1 Casgevy, the first CRISPR therapy approved by the FDA, will cost $2.2

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Pharmaceutical Technology

OCTOBER 23, 2024

Ocuphire Pharma has announced the acquisition of Opus Genetics to develop gene therapies for inherited retinal diseases (IRDs).

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Medical Xpress

JUNE 6, 2025

Using a tool called Automated Meiotic Mapping (AMM) that was developed at UT Southwestern Medical Center, a team of researchers has identified a gene that appears to be key to regulating food intake.

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Pharmaceutical Technology

MAY 24, 2023

Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Bio Pharma Dive

NOVEMBER 20, 2020

Through a deal with Precision Biosciences, the pharma aims to develop therapies for genetic disorders, including Duchenne muscular dystrophy.

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Scienmag

JUNE 17, 2025

Unlocking these enzymes would enable transferring their genetic blueprints into fast-growing industrial microorganisms, like yeast, converting them into microbial factories that produce Taxol on scales far surpassing what nature offers. This comprehensive gene set promises to transform industrial drug manufacture.

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Bio Pharma Dive

MAY 8, 2024

The results add to an early but growing body of evidence suggesting gene therapy may improve outcomes in young kids with a genetic form of deafness.

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Scienmag

JUNE 9, 2025

A recently published study has uncovered a significant shortfall in the genetic testing of patients diagnosed with endometrial cancer—commonly referred to as womb cancer—across the UK and Ireland. Affecting approximately one in every 300 individuals, it remains underdiagnosed with a staggering 95% of carriers unaware of their genetic status.

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Pharmaceutical Technology

MAY 26, 2023

Krystal Biotech has received approval from the US Food and Drug Administration for topical gene therapy VYJUVEK to treat dystrophic epidermolysis bullosa (DEB) in adults and in children aged six months and above. VYJUVEK is designed to address the underlying genetic cause of the disease. It prevents epidermis adhesion to the dermis.

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Bio Pharma Dive

JANUARY 24, 2024

An 11-year-old boy born deaf can hear after receiving Lilly's genetic medicine. Others, including biotechs in China and Regeneron in the U.S., are taking aim at the same drug target.

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Bio Pharma Dive

SEPTEMBER 12, 2024

Spun out of research at Nationwide Children’s Hospital, Vironexis claims it will be the first company to start a study testing a cancer drug delivered via the adeno-associated viruses commonly used in genetic medicines.

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