Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

New protein shows potential for treating systolic heart failure

The heart is a muscle like no other, beating 60 to 100 times per minute on average, around the clock. But when it grows weak, it can lead to serious problems: from debilitating shortness of breath and swelling in the legs and feet, to fluid in the lungs and even death.

16 Oct 2024

Adenine base editors show potential to correct X-CGD-causing mutations

A collaborative effort between investigators at the National Institutes of Health's National Institute of Allergy and Infectious Diseases (NIAID) and Massachusetts General Hospital (MGH), a founding member of the Mass General Brigham healthcare system, demonstrates the potential of precise genome editing technologies, called adenine base editors, to correct disease-causing mutations in stem cells from patients with X-linked chronic granulomatous disease (X-CGD), a rare genetic disorder characterized by high susceptibility to infections.

16 Oct 2024

Cellular Origins, Fresenius Kabi sign development agreement for scalable automation of cell and gene therapy manufacturing

Cellular Origins and Fresenius Kabi, an operating company of Fresenius, announce today they have signed a development agreement that leverages each company’s expertise in cell and gene therapies (CGTs).

16 Oct 2024

New gene therapy offers hope in combatting childhood obesity

According to the Centers for Disease Control, nearly 20% of children and teens are considered obese.

14 Oct 2024

MaxCyte appoints Ali Soleymannezhad as Chief Commercial Officer

MaxCyte, Inc., a leading, cell-engineering focused company providing enabling platform technologies to advance the discovery, development and commercialization of next-generation cell-based therapeutics and innovative bioprocessing applications, today announced the appointment of Ali Soleymannezhad as Chief Commercial Officer, effective as of September 11, 2024.

From MaxCyte, Inc. 11 Oct 2024

New magnetogenetics technology holds promise as a powerful tool for studying the brain

A new technology enables the control of specific brain circuits non-invasively with magnetic fields, according to a preclinical study from researchers at Weill Cornell Medicine, The Rockefeller University and the Icahn School of Medicine at Mount Sinai.

10 Oct 2024

Grant supports gene-editing research for rare metabolic diseases

A $14M grant will fund research on gene-editing therapies for rare metabolic diseases at the Perelman School of Medicine at the University of Pennsylvania (Penn) and Children's Hospital of Philadelphia (CHOP).

10 Oct 2024

Novel gene therapy holds potential in treating patients with epilepsy

Researchers from the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine) are working on a therapy that holds potential in treating patients with epilepsy, a neurological disorder defined by recurring seizures due to abnormal brain activity.

9 Oct 2024

Gene therapy offers hope for children with cerebral adrenoleukodystrophy

Cerebral adrenoleukodystrophy (CALD) is a rare progressive, genetic brain disease that primarily presents in young boys, causing loss of neurological function and ultimately leading to early death.

9 Oct 2024

Scientists overcome major challenge in gene therapy and drug delivery

Imagine trying to poke a hole in the yolk of a raw egg without breaking the egg white. It sounds impossible, but researchers at the University of California San Diego have developed a technology that performs a similarly delicate task in living cells. They created an array of nanopillars that can breach the nucleus of a cell-;the compartment that houses our DNA-;without damaging the cell's outer membrane.

3 Oct 2024

Novel lipid nanoparticles enhance tissue-specific drug delivery

Penn Engineers have discovered a novel means of directing lipid nanoparticles (LNPs), the revolutionary molecules that delivered the COVID-19 vaccines, to target specific tissues, presaging a new era in personalized medicine and gene therapy.

1 Oct 2024

Unlocking the secrets of analytical services: An insider interview with colman byrne

In this interview, News-Medical talks to Mr. Byrne about the crucial role of analytical services.

From DSI, a PLG Company 1 Oct 2024

FUJIFILM Irvine Scientific adds key products to its Life Sciences portfolio from FUJIFILM Wako Chemicals U.S.A.

FUJIFILM Irvine Scientific's expansion includes lab automation and chemicals, streamlining access to essential life science products for researchers worldwide.

From FUJIFILM Irvine Scientific 27 Sep 2024

Study reveals life-changing impact of gene therapy for hemophilia B patients

Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the results of an international Phase III clinical trial published today in the New England Journal of Medicine by researchers from the University of Pennsylvania Perelman School of Medicine and a multicenter group of investigators.

25 Sep 2024

Optimizing research with automation: Solutions and best practices

In this interview, NewsMedical speaks with Cerba's Coen Staplers about the benefits of lab automation, including the integration of AI technologies.

From Cerba Research 25 Sep 2024

News-Medical announces partnership with World Orphan Drug Congress

Engage with over 2000 attendees at the World Orphan Drug Congress Europe, focusing on gene therapy and strategies for rare disease treatment advancements.

From World Orphan Drug Congress 23 Sep 2024

Broken String Biosciences appoints Laurence Reid and Brad Crutchfield to its Board of Directors

Broken String Biosciences enhances its leadership team with Laurence Reid and Brad Crutchfield, aiming to revolutionize cell and gene therapy development.

From Broken String Biosciences 23 Sep 2024

MaxCyte signs strategic platform license with Kamau Therapeutics to accelerate the development of cell therapies for genetic diseases

MaxCyte, Inc., a leading, cell-engineering focused company providing enabling platform technologies to advance the discovery, development and commercialization of next-generation cell-based therapeutics and innovative bioprocessing applications, and Kamau Therapeutics, a clinical-stage stem cell therapy gene correction company, today announced they are entering into a strategic platform license (SPL) agreement.

From MaxCyte, Inc. 23 Sep 2024

Asimov launches AAV Edge, a suite of AI models, host cells, and genetic tools for end-to-end gene therapy development

Asimov, the synthetic biology company advancing the design and production of therapeutics, today announced the launch of the AAV Edge System, a comprehensive suite of tools for adeno-associated viral (AAV) gene therapy design and manufacturing.

18 Sep 2024

$1 million grant awarded to explore myelin repair mechanisms in multiple sclerosis

The National Multiple Sclerosis Society (USA) has awarded a grant of 1 million dollars to Dr. Isabel Pérez-Otaño, who leads the Plasticity and Remodeling of Neural Circuits laboratory at the Institute for Neurosciences (IN), a joint center of the Spanish National Research Council (CSIC) and the Miguel Hernández University (UMH) of Elche.

18 Sep 2024

Gene Therapy News and Research

Further Reading

New protein shows potential for treating systolic heart failure

Adenine base editors show potential to correct X-CGD-causing mutations

Cellular Origins, Fresenius Kabi sign development agreement for scalable automation of cell and gene therapy manufacturing

New gene therapy offers hope in combatting childhood obesity

MaxCyte appoints Ali Soleymannezhad as Chief Commercial Officer

New magnetogenetics technology holds promise as a powerful tool for studying the brain

Grant supports gene-editing research for rare metabolic diseases

Novel gene therapy holds potential in treating patients with epilepsy

Gene therapy offers hope for children with cerebral adrenoleukodystrophy

Scientists overcome major challenge in gene therapy and drug delivery

Novel lipid nanoparticles enhance tissue-specific drug delivery

Unlocking the secrets of analytical services: An insider interview with colman byrne

FUJIFILM Irvine Scientific adds key products to its Life Sciences portfolio from FUJIFILM Wako Chemicals U.S.A.

Study reveals life-changing impact of gene therapy for hemophilia B patients

Optimizing research with automation: Solutions and best practices

News-Medical announces partnership with World Orphan Drug Congress

Broken String Biosciences appoints Laurence Reid and Brad Crutchfield to its Board of Directors

MaxCyte signs strategic platform license with Kamau Therapeutics to accelerate the development of cell therapies for genetic diseases

Asimov launches AAV Edge, a suite of AI models, host cells, and genetic tools for end-to-end gene therapy development

$1 million grant awarded to explore myelin repair mechanisms in multiple sclerosis

Decoding Cannabis: Differentiating Hemp and Marijuana

Deciphering the Microbiome with The Microsetta Initiative

Leveraging the power of automation to boost research

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