BioSpace

SEPTEMBER 27, 2023

The New England Journal of Medicine study suggests that the 2022 death of a Duchenne muscular dystrophy patient was due to an adverse effect of recombinant AAV used to deliver the gene therapy.

Gene Editing 124

Gene Editing Gene Gene Therapy Medicine 124

Drug Discovery World

DECEMBER 6, 2023

Gene editing company Eligo Bioscience has announced a successful $30 million Series B funding round, led by Sanofi Ventures. “We We feel this reflects the strong support for our vision and confirms the potential of Eligo to create a novel class of transformative genetic medicines.”

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. The post AstraZeneca pays record 660% premium for gene editing company LogicBio appeared first on.

Gene Editing 64

Gene Editing Gene Genome Genomics 64

Medical Xpress

DECEMBER 21, 2022

Using the CRISPR-Cas9 gene editing system, UT Southwestern researchers corrected mutations responsible for a common inherited heart condition called dilated cardiomyopathy (DCM) in human cells and a mouse model of the disease.

Gene Therapy 98

Gene Therapy Gene Editing Gene Research 98

Drug Discovery World

SEPTEMBER 6, 2022

By Dr. Deborah Phippard, Chief Scientific Officer of Precision for Medicine and Anshul Mangal, President & General Counsel of Project Farma. . In recent years the advanced therapy industry has been the most significant disruptor in medicine. ViaCyte, Beam, and Verve are all due to begin gene editing trials this year.

Gene Therapy 52

Gene Therapy Gene Gene Editing Manufacturing 52

Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

Gene Therapy 52

Gene Therapy FDA Approval Gene Gene Editing 52

pharmaphorum

OCTOBER 13, 2022

Gene therapies and research into them have grown immensely in recent years, offering more novel tools in regenerative medicine to fight disease, including rare diseases and genetic disorders. The move marks a continuation in its ongoing commitment to exploring gene-editing technology.

Gene Editing 52

Gene Editing Gene Gene Therapy Genetics 52

Drug Discovery World

SEPTEMBER 25, 2023

CRISPR gene editing companies Integrated DNA Technologies (IDT) and Aldevron have inked a new global distribution agreement to expand CRISPR products for cell and gene therapy customers. The post Partners working to realise the future of genomic medicine appeared first on Drug Discovery World (DDW).

Genome 52

Genome Genomics Gene Editing Medicine 52

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., “Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients.”

Gene Therapy 40

Gene Therapy Gene Gene Editing In-Vivo 40

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 52

pharmaphorum

AUGUST 24, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2 The post Vertex builds in gene editing yet again, with $1.2bn Arbor deal appeared first on.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Scienmag

AUGUST 18, 2020

In Nature Communications, Fred Hutch scientists demonstrate in a mouse model how gene therapy could cure an infection that afflicts billions of people Infectious disease researchers at Fred Hutchinson Cancer Research Center have used a gene editing approach to remove latent herpes simplex virus 1, or HSV-1, also known as oral herpes.

Gene Therapy 45

Gene Therapy Gene Gene Editing Scientist 45

Scienmag

JANUARY 19, 2021

publishers New Rochelle, NY, January 19, 2021–Gene editing therapies, including CRISPR-Cas systems, offer the potential to correct mutations causing inherited retinal degenerations, a leading cause of blindness. Credit: Mary Ann Liebert, Inc.,

Gene Editing 101

Gene Editing Genome Genomics Gene Therapy 101

Roots Analysis

FEBRUARY 27, 2024

The global genome editing market is anticipated to grow at a CAGR of 12.6% How is the genome editing market landscape evolving: Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques.

Genome 40

Genome Genomics Gene Gene Editing 40

The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Selim Corbacioglu from the University of Regensburg, relies on CRISPR-Cas9 gene editing to switch off BCL11A.

Gene Therapy 52

Gene Therapy Gene Gene Editing Genetics 52

BioSpace

NOVEMBER 17, 2022

Editas Medicine is pausing its ocular gene therapy program after demonstrating a favorable safety profile and seeking a potential partner to develop EDIT-101, the company announced Thursday.

Gene Therapy 42

Gene Therapy Gene Gene Editing Medicine 42

Drug Discovery World

DECEMBER 21, 2023

In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Other biotechnologies coming to the fore this year were antibody-drug conjugates and CRISPR gene editing. Read the article – Expert view: What’s next for cell and gene therapy?

Gene Therapy 59

Gene Therapy Gene Antibody FDA Approval 59

Drug Discovery World

JUNE 22, 2023

“This is part of our long-term business strategy of leveraging exosome-mediated delivery to create and enable genetic medicines such as gene therapy and genome editors”.

Gene Therapy 52

Gene Therapy Gene Editing Drug Delivery Gene 52

Drug Discovery World

MAY 24, 2023

This exceeds the current, but growing, global supply of plasmid DNA for genetic medicine, and dramatically increases Touchlight’s capacity to meet growing demand for DNA from biopharmaceutical companies. Touchlight has contracts to supply many top biopharmaceutical companies, including Pfizer and Lonza.

DNA 52

DNA Gene Editing Gene Therapy Genetics 52

Drug Discovery World

OCTOBER 6, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

Drugs 52

Drugs Gene Editing Genome Genomics 52

XTalks

JANUARY 26, 2024

Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment. By editing these genes, researchers can effectively neutralize their cancer-promoting effects.

Research 118

Research Gene Editing Gene Genetics 118

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

Drug Discovery World

OCTOBER 10, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

Gene Editing 59

Gene Editing Genome Genomics Genetics 59

Drug Discovery World

APRIL 15, 2024

Melissa Little, CEO of the Novo Nordisk Foundation Centre for Stem Cell Medicine “In the stem cell space, 2023 has seen significant increases in the delivery of pluripotent stem cell-derived cell types into first-in-human clinical trials. Gene therapy will move to ex vivo gene modification and the delivery of gene-modified cell types.

Gene Editing 59

Gene Editing Clinical Trials Clinical Trials In-Vivo 59

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

Delveinsight

FEBRUARY 25, 2021

Positioning of Gene and Cell Therapies in Neurosciences. Decades of technological progress and careful research, Gene and cell therapy (GCT), which were previously viewed as the ‘future’ of medicine, are now all set to become a reality. Gene and cell therapies hold the potential to provide a one-off cure.

Gene 52

Gene Gene Therapy Gene Editing Development 52

Drug Discovery World

JANUARY 9, 2023

The findings are published in Science Translational Medicine. . Using gene engineering, we are repurposing cancer cells to develop a therapeutic that kills tumour cells and stimulates the immune system to both destroy primary tumors and prevent cancer.” . CRISPR-Cas9 gene editing .

Scientist 52

Scientist Gene Editing Gene Engineer 52

Pharmaceutical Technology

JANUARY 10, 2023

Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP.

Gene Editing 130

Gene Editing Gene Gene Therapy Genetics 130

Drug Discovery World

DECEMBER 20, 2022

Personalised and precision medicines will dominate pipelines. In its expected biotech trends for 2023, financial services company Nasdaq emphasises personalised medicine as a potential area for huge growth. . In summary, 2023 could be a very exciting year for life sciences.

Drugs 52

Drugs Gene Editing Clinical Trials Clinical Trials 52

Pharmaceutical Technology

JUNE 15, 2023

Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo gene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD).

Gene Editing 130

Gene Editing Gene Gene Therapy In-Vivo 130

Drug Discovery World

SEPTEMBER 6, 2022

The cell and gene therapy industry has experienced exponential growth in the last three years and as funding for the field continues to break records there are no signs of a slowdown. With 2,261 ongoing clinical trials in regenerative medicine, the FDA expects to approve 1 between 10 and 20 new cell and gene therapies a year by 2025.

Gene Therapy 52

Gene Therapy Gene Manufacturing Clinical Trials 52

The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

Manufacturing 52

Manufacturing Gene Therapy Trials Gene 52

Drug Discovery World

JULY 10, 2023

Artificial intelligence (AI), gene editing, synthetic biology and more, are beginning to prove their usefulness in drug design and development, taking therapies away from the one-size-fits-all approach, towards more definable targets that operate more effectively. There are myriad ways that this is being enabled.

Gene Therapy 52

Gene Therapy Gene Scientist Gene Editing 52

XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies. Here are four life sciences trends to look out for in 2023.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

XTalks

JANUARY 4, 2024

Pfizer has kickstarted the new year with its first-ever gene therapy approval, awarded by Health Canada to the company’s Beqvez (fidanacogene elaparvovec) for the treatment of hemophilia B. There is a significant focus on developing gene therapies as longer-term solutions for the disease.

Gene Therapy 111

Gene Therapy Gene Gene Editing Sales 111

pharmaphorum

FEBRUARY 17, 2022

Oxbryta is the first medicine approved in Europe that directly prevents sickle haemoglobin (HbS) polymerisation, the molecular basis of sickling and destruction of red blood cells in SCD, and has been tipped to become a $1 billion-plus blockbuster at peak. The post GBT claims EU approval for sickle cell drug Oxbryta appeared first on.

Drugs 59

Drugs Gene Therapy Gene Editing Gene 59

Drug Discovery World

APRIL 3, 2023

As the industry’s awareness of alternatives to traditional antibodies continues to grow, there is increasing demand for these alternatives, which we are seeing particularly within the therapeutic sector, to enable targeted therapeutics and enable the emerging gene therapy market. DS: The company now has clients across big pharma.

Antibody 52

Antibody Gene Therapy Gene In-Vitro 52