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.

First cell-based gene therapy approved by the FDA for treating multiple myeloma

The U.S. Food and Drug Administration approved Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adult patients with multiple myeloma who have not responded to, or whose disease has returned after, at least four prior lines (different types) of therapy.

29 Mar 2021

Mogrify wins Hewitsons Award for Innovation in Business and Price Bailey Award for Business of the Year at CambridgeshireLive Business Excellence Awards 2020

Mogrify has been awarded the Hewitsons Award for Innovation in Business and Price Bailey Award for Business of the Year.

From Mogrify 26 Mar 2021

Novel gene therapy reprograms glioma cells into functional neurons

Glioma is a fatal neurological disorder that has limited interventional treatment, despite extensive research over the past several decades.

24 Mar 2021

First model of early human embryo generated from skin cells

In a discovery that will revolutionise research into the causes of early miscarriage, infertility and the study of early human development - an international team of scientists led by Monash University in Melbourne, Australia has generated a model of a human embryo from skin cells.

22 Mar 2021

Alliance for Cancer Gene Therapy provides new opportunity to develop cancer cell and gene therapies

Alliance for Cancer Gene Therapy wants to change the C-word from Cancer to Cure -- building on the life changing success of CAR-T therapies for blood cancers, the organization is now focused on the urgent need to translate this success into therapies to fight more types of cancer, such as solid tumor cancers.

19 Mar 2021

Novel cancer-fighting gene therapy enters phase I clinical trial

Researchers from the MDC and Charité have developed a new gene therapy based around a special T-cell receptor that helps the immune system effectively recognize and fight cancer cells.

18 Mar 2021

Beneficial effects of gene therapy can be seen in SCID-X1 patients decades after treatment

An international collaboration between Great Ormond Street Hospital, the UCL GOS Institute for Child Health and Harvard Medical School has shown that the beneficial effects of gene therapy can be seen decades after the transplanted blood stem cells has been cleared by the body.

17 Mar 2021

UVA researcher developing innovative gene therapy to help children with Rett syndrome

A University of Virginia School of Medicine scientist is developing an innovative gene therapy she hopes will slow disease progression and improve movement, coordination and communication in children with Rett syndrome.

17 Mar 2021

New approach could treat infertility caused by abnormal GnRH secretion

Reports suggest that at least 25% of ovarian disorders are due to dysfunction of the brain mechanism in the hypothalamus that controls the gonadotropin-releasing hormone (GnRH), which is a molecule that governs the reproductive function in men and women by regulating the release of a range of reproductive hormones into the body, particularly gonadotropins, from the pituitary gland, which is located underneath the brain.

12 Mar 2021

Phase 2 clinical trial is now open at Columbia to test gene therapy for dry macular degeneration

A phase 2 clinical trial is underway at Columbia University and NewYork-Presbyterian to test an investigational gene therapy for dry age-related macular degeneration, a progressive, chronic eye disease with no cure and a leading cause of blindness in the United States.

9 Mar 2021

New academic spin-out based in Finland introduces a nasal spray vaccine against COVID

Rokote Laboratories Finland Ltd., a newly-founded academic spin-out based in Finland is working to develop and introduce to the markets a nasal spray vaccine against COVID.

8 Mar 2021

Discovery offers a precise route to treat inflammation associated with neuropathic pain

One of the hallmarks of chronic pain is inflammation, and scientists at the UNC School of Medicine have discovered that anti-inflammatory cells called MRC1+ macrophages are dysfunctional in an animal model of neuropathic pain.

5 Mar 2021

PICI awards $3 million to support early career cancer researchers

From improving immunotherapy response in childhood cancer patients, to better understanding how cancer's spread to the liver affects the body's immune system, to helping T cells stay energized to fight cancer, The Parker Institute for Cancer Immunotherapy (PICI)'s 2021 class of early career researchers award recipients are pursuing ideas that have the potential to change the face of oncology.

3 Mar 2021

ACGT awards $500,000 to advance immunotherapy approach for treating solid tumors

A scientific team led by Brian Brown, PhD, Professor of Genetics and Genomic Sciences and Associate Director of the Precision Immunology Institute at the Icahn School of Medicine at Mount Sinai (New York, New York), has been awarded $500,000 from Alliance for Cancer Gene Therapy to advance a promising immunotherapy approach to fight lung cancer and other solid tumor cancers.

1 Mar 2021

Catalent to Acquire Delphi Genetics and Launch US Plasmid Manufacturing Site to Establish Global pDNA Development and Manufacturing Capabilities

Catalent and Delphi Genetics today announced that they have entered into a final and definitive agreement whereby Catalent will acquire 100% of the shares of Delphi Genetics.

26 Feb 2021

Catalent to Acquire Delphi Genetics and Launch US Plasmid Manufacturing Site to Establish Global pDNA Development and Manufacturing Capabilities

Catalent and Delphi Genetics today announced that they have entered into a final and definitive agreement whereby Catalent will acquire 100% of the shares of Delphi Genetics.

26 Feb 2021

Canadian pilot study uses gene therapy to successfully treat patients with Fabry disease

Results of a world-first Canadian pilot study on patients treated with gene therapy for Fabry disease show that the treatment is working and safe.

25 Feb 2021

A new combination therapy for treating rare, fatal Sanfilippo syndrome in children

A study aiming to develop a new therapeutic technique could bring a revolution in our approach to treating rare, fatal Sanfilippo syndrome, a disorder that affects children as young as 2 years old and leads to childhood dementia and premature death.

24 Feb 2021

Novel vectors reduce the risks associated with ocular gene therapies

Strategies based on the use of gene therapy to mitigate the effects of mutations that cause blindness are undergoing rapid development.

22 Feb 2021

UC San Diego researchers launch clinical trial to test gene therapy for Alzheimer’s disease

Researchers at University of California San Diego School of Medicine have launched a first-in-human Phase I clinical trial to assess the safety and efficacy of a gene therapy to deliver a key protein into the brains of persons with Alzheimer's disease (AD) or Mild Cognitive Impairment (MCI), a condition that often precedes full-blown dementia.

18 Feb 2021

Gene Therapy News and Research

Further Reading

First cell-based gene therapy approved by the FDA for treating multiple myeloma

Mogrify wins Hewitsons Award for Innovation in Business and Price Bailey Award for Business of the Year at CambridgeshireLive Business Excellence Awards 2020

Novel gene therapy reprograms glioma cells into functional neurons

First model of early human embryo generated from skin cells

Alliance for Cancer Gene Therapy provides new opportunity to develop cancer cell and gene therapies

Novel cancer-fighting gene therapy enters phase I clinical trial

Beneficial effects of gene therapy can be seen in SCID-X1 patients decades after treatment

UVA researcher developing innovative gene therapy to help children with Rett syndrome

New approach could treat infertility caused by abnormal GnRH secretion

Phase 2 clinical trial is now open at Columbia to test gene therapy for dry macular degeneration

New academic spin-out based in Finland introduces a nasal spray vaccine against COVID

Discovery offers a precise route to treat inflammation associated with neuropathic pain

PICI awards $3 million to support early career cancer researchers

ACGT awards $500,000 to advance immunotherapy approach for treating solid tumors

Catalent to Acquire Delphi Genetics and Launch US Plasmid Manufacturing Site to Establish Global pDNA Development and Manufacturing Capabilities

Catalent to Acquire Delphi Genetics and Launch US Plasmid Manufacturing Site to Establish Global pDNA Development and Manufacturing Capabilities

Canadian pilot study uses gene therapy to successfully treat patients with Fabry disease

A new combination therapy for treating rare, fatal Sanfilippo syndrome in children

Novel vectors reduce the risks associated with ocular gene therapies

UC San Diego researchers launch clinical trial to test gene therapy for Alzheimer’s disease

Event guide: 9th Gene Therapy Development Summit 2026 eBook

The Critical Role of Sample Storage in Drug Discovery and Development

Advancing Neuroscience Research Through Collaboration and Patient-Centered Innovation

The impact of vibration on microscopy

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