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.

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

Scientists develop selective therapy for tau tangles

Scientists have developed new potential therapies that selectively remove aggregated tau proteins, which are associated with Alzheimer's disease, and improve symptoms of neurodegeneration in mice.

14 Sep 2024

Gene therapy improves vision in patients with Bothnia dystrophy

Bothnia dystrophy is a form of hereditary blindness, prevalent in the region Västerbotten in Sweden. A new study at Karolinska Institutet published in Nature Communications shows that gene therapy can improve vision in patients with the disease.

10 Sep 2024

SP-101 gene therapy shows promise for cystic fibrosis treatment

Scientists from Spirovant Sciences describe SP-101, a novel adeno-associated virus (AAV) gene therapy for the treatment of cystic fibrosis (CF) in the peer-reviewed journal Human Gene Therapy.

9 Sep 2024

Cancer drug offers life-changing results for severe lupus patient

The team at Charité – Universitätsmedizin Berlin is astounded by the huge improvement seen in a female patient with severe systemic lupus erythematosus (SLE) after being treated with the cancer medication teclistamab. Not long ago, the 23-year-old was wheelchair-bound due to the autoimmune disease.

6 Sep 2024

Gene therapy shows dramatic vision improvement for rare inherited condition

The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it.

5 Sep 2024

New consensus on assessing carcinogenic risks of gene therapies

A new article in the peer-reviewed journal Human Gene Therapy describes the consensus reached by invited experts who participated in a meeting to evaluate the potential carcinogenicity of gene therapies.

30 Aug 2024

Cell therapy commercialization: Streamline your path to cell therapy commercialization

Moving your cell therapy clinical trial beyond Phase I is an exciting time, marking a significant milestone. There are many therapeutic developers working to address critical patient needs in oncology, auto-immune, regenerative medicine, and other spaces.

From Charles Rivers Laboratories Cell and Gene Therapy 28 Aug 2024

New benchmark: 1 kg of plasmid DNA manufactured

Kaneka Eurogentec sets new benchmark in plasmid DNA manufacturing with the production of 1 kg of plasmid DNA.

From Kaneka Eurogentec S.A 27 Aug 2024

Improved governance and pre-release safety evaluations needed for biological AI models

Concerns over the biosecurity risks posed by artificial intelligence (AI) models in biology continue to grow.

23 Aug 2024

New $400 million donation reinforces Columbia's leadership in biomedical science

Columbia University announced today a new $400 million gift from Roy and Diana Vagelos, which will secure Columbia's leadership in biomedical science research and education and produce a vast array of compelling opportunities for improving society's health and wellbeing.

22 Aug 2024

mRNA therapy shows promise for maple syrup urine disease

Researchers from the University of Pennsylvania, Perelman School of Medicine, Gene Therapy Program, and Moderna, have shown that repeated administration of lipid nanoparticle-encapsulated mRNA therapy significantly extended survival and reduced serum leucine levels in a mouse model of maple syrup urine disease (MSUD).

22 Aug 2024

New insights into retrotransposon-derived DNA zip codes in myeloma cells

A new editorial was published in Oncoscience (Volume 11) on July 13, 2024, entitled, "Unveiling retrotransposon-derived DNA zip code for myeloma cell internalization."

9 Aug 2024

Machine learning revolutionizes gene delivery

Gene therapy could potentially cure genetic diseases but it remains a challenge to package and deliver new genes to specific cells safely and effectively. Existing methods of engineering one of the most commonly used gene-delivery vehicles, adeno-associated viruses (AAV), are often slow and inefficient.

9 Aug 2024

Houston Methodist pioneers new technique for targeted drug delivery to the brain

Houston Methodist researchers have discovered a more accurate and timely way to deliver life-saving drug therapies to the brain, laying the groundwork for more effective treatment of brain tumors and other neurological diseases.

8 Aug 2024

MIP Discovery rebrands as Tozaro as it completes transition to supporting cell and gene therapy viral vector analytics and purification

Tozaro’s rationally designed smart polymer affinity reagents offer novel analytics and purification solutions to enhance the development and manufacture of viral vectors in cell and gene therapy.

6 Aug 2024

New nanosensing technique enhances quality control for gene therapy vectors

Over the past few decades, there has been remarkable progress in genetic manipulation technologies, bringing us closer to the point where genes can be modified in vivo.

1 Aug 2024

Scientists explore early life factors contributing to SMA

Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, scientists at DZNE and the Dresden University of Technology are now drawing attention to previously unnoticed abnormalities in embryonic development. They base their argument on studies of so-called organoids: Laboratory-grown tissue cultures that can reconstruct disease processes. Their findings are published in the journal Cell Reports Medicine.

27 Jul 2024