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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Gene Therapy Gene Development Genetics 298

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

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Gene Therapy Gene Gene Editing Genetics 238

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.

Genetics 176

Genetics Gene Scientist Research 176

Drug Discovery World

JULY 19, 2024

Dr Gonzalez-Aseguinolaza said: “This is a tremendous honour, and I am very pleased that the RFS Society recognises my dedication and work in the field of gene therapy. Since 2014, Dr Gonzalez-Aseguinolaza has been head of the Gene Therapy Program at the Centro Investigación Médica Aplicada (CIMA), Universidad de Navarra, in Spain.

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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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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.

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Gene Therapy Gene Development Manufacturing 256

Worldwide Clinical Trials

OCTOBER 24, 2023

Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. These insights were shared through several sessions, in presentation tracks specifically dedicated to “Community and Capacity Building” and “Becoming a Research Ready Organization.”

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Gene Gene Editing Gene Therapy Clinical Trials 130

STAT News

FEBRUARY 22, 2023

Moderna is aiming to build a gene editing franchise powered by some of the same technologies used in its COVID-19 vaccines. The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions.

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Gene Editing Gene Genetic Disease Research 126

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 242

Worldwide Clinical Trials

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

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Gene Editing Gene Gene Therapy In-Vivo 189

STAT News

FEBRUARY 16, 2023

With hundreds of genes thought to be linked to obesity, the challenge is sifting through them all to determine which ones increase the risk of downstream complications like heart disease and diabetes. In a study published Thursday in Nature Genetics, researchers took the first steps in finding a potential candidate specifically in women.

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Drug Discovery World

OCTOBER 5, 2023

A research team at the Francis Crick Institute and Great Ormond Street Hospital (GOSH)/UCL Great Ormond Street Institute of Child Health have identified potential treatments for children with rare genetic conditions. The researchers have shown that problems with calcium underlie these progressive diseases.

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Genetics Research Gene Doctors 59

Pharmaceutical Technology

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Immuno-oncology in Pharmaceuticals: Gene therapy delivery using viral vectors. Sanofi is one of the leading patent filers working with viral vectors.

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Gene Therapy Gene Genome Genomics 246

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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Gene Therapy Gene Medicine Genetics 245

Pharmaceutical Technology

MAY 25, 2023

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.

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Gene Therapy Gene Gene Editing Manufacturing 243

Pharmaceutical Technology

MARCH 24, 2023

Moderna has entered a strategic partnership with Generation Bio for the development of non-viral genetic medicines. Moderna’s biological and technical expertise will be combined with core technologies of the non-viral genetic medicine platform from Generation Bio.

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Genetics Medicine Development In-Vivo 130

Scienmag

SEPTEMBER 18, 2020

Two active genetics strategies help address concerns about gene-drive releases into the wild Credit: Ana Silva In the past decade, researchers have engineered an array of new tools that control the balance of genetic inheritance.

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Genetics Gene Engineer Research 86

AuroBlog - Aurous Healthcare Clinical Trials blog

MARCH 17, 2024

Similar genetic changes in two different types of brain cells may contribute to cognitive impairment in schizophrenia and aging. US researchers examined gene expression in more than a million brain cells collected post-mortem from 191 donors.

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Gene Expression Gene Genetics Research 185

Drug Discovery World

JUNE 19, 2024

New research has uncovered how different people respond to sepsis based on their genetics, which could lead to the development of targeted therapies. This provides insight into how an individual’s genetic makeup influences how they respond to sepsis and who would benefit from targeted therapies.

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Genetics Immune Response Genome Genomics 64

AuroBlog - Aurous Healthcare Clinical Trials blog

AUGUST 7, 2023

New research from ETH Zürich in Switzerland could see future wearable devices (with perhaps a few implants and a touch of genetic engineering) boost our health directly. Fitness trackers help you stay healthy by keeping count of your steps and monitoring your heart rate, driving you on to hit those cardio goals.

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Genetic Engineering Gene Engineer Genetics 204

Pharmaceutical Technology

JANUARY 5, 2023

Capsida Biotherapeutics and Eli Lilly and Company ’s wholly owned subsidiary Prevail Therapeutics have announced a partnership for the development of non-invasive gene therapies for central nervous system (CNS) diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. By Cytiva Thematic.

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Gene Therapy Gene Development Engineer 237

Scienmag

SEPTEMBER 29, 2021

Describing a previously unknown genetic condition that affects children, researchers at University of California San Diego School of Medicine and Rady Children’s Institute for Genomic Medicine say they also found a potential method to prevent the gene mutation by administering a drug during pregnancy.

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Genetics Research Genome Genomics 84

Drug Discovery World

JULY 9, 2024

Researchers at the Francis Crick Institute have mapped all the possible outcomes of changes to a tumour-suppressing gene called VHL. It is the first step in a huge research endeavour to unpick the outcomes of tens of thousands of genetic variations in genes associated with cancer.

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Gene Mapping Gene Genome Genomics 59

Medical Xpress

MARCH 1, 2023

Researchers have discovered how a gene that increases the risk of developing genetic heart disease functions, paving the way for new treatments.

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Gene Research Genetics Development 75

Scienmag

MAY 6, 2021

Jude Children’s Research Hospital scientists have developed a highly efficient method to address a major challenge in biology–identifying the genetic ‘switches’ that regulate gene expression. Jude Children’s Research Hospital St. Credit: St.

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Olympian Clinical Research

SEPTEMBER 27, 2023

While the exact cause of vitiligo remains a subject of ongoing research, genetics has emerged as a significant factor in understanding its onset and progression. In this deep dive, we’ll explore the genetic links associated with vitiligo and address the burning question: Is vitiligo hereditary?

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Genetics Gene Clinical Research Research 52

Medical Xpress

JANUARY 19, 2023

Several genes that cause sarcoma have been identified in the first comprehensive genetic map of sarcomas, generated by research led by Omico, the Garvan Institute of Medical Research and UNSW Sydney.

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Medical Xpress

MAY 16, 2023

However, how the brain has developed over the course of evolution and which genes are responsible for the high cognitive abilities is still largely unclear. The better our understanding of the role of genes in brain development, the more likely it will be that we will be able to develop treatments for serious brain diseases.

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Pharmaceutical Technology

JUNE 4, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Transcription factors for genetically modified cells. However, not all innovations are equal and nor do they follow a constant upward trend.

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Genetics Gene Expression DNA Antibody 130

Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. A key part of the IBC’s evaluation is assessing the risks posed by the engineered genetic materials.

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Engineer Genetics Clinical Research Research 52

Drug Discovery World

MARCH 20, 2023

Researchers have identified two previously unknown genes linked to schizophrenia and newly implicated a third gene as carrying risk for both schizophrenia and autism. The investigators found the two risk genes, SRRM2 and AKAP11, by comparing the gene sequences of people with schizophrenia to those of healthy controls.

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Gene Research Genetics Gene Sequencing 52

Drug Discovery World

JANUARY 10, 2024

New understanding of a gene that is linked to some forms of dementia and other age-related diseases gives scientists fresh hope that action can be taken against these diseases long before the onset of symptoms. The researchers studied a family affected by both frontotemporal dementia and motor neurone disease.

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Gene Research Gene Therapy Life Science 59

Drug Discovery World

AUGUST 7, 2023

Evox Therapeutics has agreed a research collaboration and option agreement with the Icahn School of Medicine at Mount Sinai (Icahn Mount Sinai) in New York, NY, US. The collaboration will work on developing exosome-encapsulated AAV (exoAAV) vectors as a novel gene delivery technology aimed at improving treatments for heart disease.

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Gene Therapy Gene Research In-Vivo 52

Drug Discovery World

JANUARY 19, 2024

UK scientists have uncovered the interplay between cancer-driving genetic mutations and inherited genetic variants in a rare type of blood cancer. The study describes how inherited genetic variants can influence whether a spontaneous mutation in a particular gene increases the risk of developing this rare blood cancer.

Genetics 59

Genetics Gene DNA Development 59

BioSpace

JUNE 23, 2022

The Boston University School of Medicine has unraveled details about a known genetic component for the development of Alzheimer’s disease (AD): the APOE4 gene.

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Genetics Gene Research Medicine 79

Medical Xpress

FEBRUARY 20, 2023

Researchers have identified 395 genes that are differently expressed in people with type 2 diabetes. One of the genes proved to be very strongly associated with impaired insulin secretion. Now, researchers want to investigate if it is possible to use the genetic CRISPR/Cas9 scissors to correct the gene's activity.

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Insulin Gene Genetics Research 98

Pharmaceutical Technology

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Genetically modified animal models. Regeneron Pharmaceuticals is one of the leading patent filers in the generation of genetically modified animal models.

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