Gene Therapy

Eli Lilly Gene Therapy Restores Hearing Within 30 Days

January 25, 2024 - On January 23, 2024, Eli Lilly and the Children’s Hospital of Philadelphia announced the success of an experimental gene therapy for patients with genetic hearing loss. According to the press releases, Lilly’s early-stage gene therapy helped restore hearing in an 11-year-old with genetic hearing loss at the Children’s Hospital of...

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FDA Expands Approval of CASGEVY for Beta Thalassemia

by Veronica Salib

On January 16, 2024, Vertex Pharmaceuticals and CRISPR Therapeutics secured yet another approval for CASGEVY, the first FDA-approved gene therapy in the United States. Last month, the companies’...

FDA Investigates Safety of CAR T-Cell Therapy

by Veronica Salib

In a safety notice published on November 28, 2022, the United States Food and Drug Administration (FDA) announced an investigation into the safety of chimeric antigen receptor (CAR) T-cell...

Gene Therapy Restores Sight in a Pediatric Patient with DEB

by Veronica Salib

Alfonso Sabater, MD, PhD, played a significant role in restoring the vision of 14-year-old Antonio Vento, a dystrophic epidermolysis bullosa (DEB) patient. Alongside two surgical procedures, a recently...

Designing Next-Generation Cell and Gene Therapies

by Alivia Kaylor

A study published in Stem Cell Reports represents a significant initial stride toward personalized therapies for individuals with incurable neurodegenerative disorders. The novel technique utilizes...

CRISPR Gene Editing and Antiretroviral Therapy Eliminates HIV in Mice

by Veronica Salib

On May 1, 2023, researchers from the Lewis Katz School of Medicine at Temple University and the University of Nebraska Medical Center (UNMC) published a study on a combined therapeutic approach that...

Viatris Closes Acquisitions of Oyster Point Pharma and Famy Life Sciences

by Hayden Schmidt

At the beginning of this month, global healthcare firm Viatris Inc announced the completion of its Oyster Point Pharma and Famy Life Sciences acquisitions. The two companies will be combined, and...

Most People with Spinal Muscular Atrophy Utilize Gene Therapy

by Veronica Salib

The Health Union recently conducted a survey called Spinal Muscular Atrophy (SMA) in America to analyze the lives of people living with SMA in the United States. This rare chronic condition impacts...

Bluebird’s Gene Therapy Costs $3 Million per Treatment

by Veronica Salib

On September 16, 2022, the FDA granted bluebird bio accelerated approval for their gene therapy technology, Skysona. This technology is meant to treat active cerebral adrenoleukodystrophy (CALD) in...

CRISPR Treatment in Clinical Trial for Pediatric Sickle Cell Disease

by Veronica Salib

The European Hematology Association (EHA) recently released results from a clinical trial that implied the potential benefits of using CRISPR treatment to address pediatric sickle cell disease (SCD)....

Study Finds Genetic Links to Common Mental Illnesses

by Hayden Schmidt

An analysis of major psychiatric disorders — including depression, anxiety, and others — published in Nature Genetics found that subsets of mental health disorders can be grouped by their...

Pfizer Opens First US Phase 3 Study Evaluating Gene Therapy

by Samantha McGrail

Pfizer will open the first United States sites in the Phase 3 study evaluating its investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory patients with Duchenne...

Thermo Fisher Launches Next-Generation Sequencing Platform for Labs

by Samantha McGrail

Thermo Fisher Scientific recently launched the CE-IVD marked Ion Torrent Genexus Dx Integrated Sequencer, an automated, next-generation sequencing (NGS) platform that delivers results in one...

Researchers Identify 42 New Genes for Alzheimer’s Disease

by Samantha McGrail

A major international study recently identified 75 genes associated with an increased risk of developing Alzheimer's disease, including 42 new genes not previously discovered.  The study...

Mount Sinai Develops CRISPR Technology to Advance Cancer Treatment

by Samantha McGrail

Mount Sinai scientists recently developed CRISPR technology to link genes to complex tumor characteristics at a scale and resolution not previously possible.  Perturb-map CRISPR technology uses a...

BioMarin Pharmaceutical’s Gene Therapy Helps Hemophilia A Patients

by Samantha McGrail

BioMarin Pharmaceuticals recently announced results from the Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy to treat adults with severe hemophilia...

Thermo Fisher Introduces System to Enable Cell Therapy Development

by Samantha McGrail

Thermo Fisher Scientific recently introduced a new large-volume electroporation system that allows cell therapy developers to quickly move from clinical development to commercial...

Boehringer, Partners Boost Gene Therapy Development For CF

by Samantha McGrail

Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium (GTC), and Oxford Biomedica (OXB) will further accelerate the development of a potential new treatment option for patients with...

Inscripta, Hunterian Boost Access to CRISPR Gene-Editing Tools

by Samantha McGrail

Inscripta recently granted a non-exclusive license to Hunterian Medicine, a gene-editing and gene therapy company, to access one of several CRISPR nucleases in its Madagascar family of nucleases...

Thermo Fisher Launches Medium to Support Cell, Gene Therapy

by Samantha McGrail

Thermo Fisher Scientific recently launched a GMP-manufactured cell culture medium that supports the large-scale growth and culture of functional natural killer (NK) cells for cell and gene...

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