pharmaphorum

APRIL 19, 2021

French genomic medicines firm SparingVision has agreed to buy GAMUT Therapeutics, a biotech specialising in gene therapies for inherited eye diseases such as retinitis pigmentosa (RP) that could compete against Roche/Spark’s Luxturna in a wider patient group. . million ($52.7

Gene Therapy 105

Gene Therapy Gene Production Genome 105

BioSpace

JULY 27, 2023

The British drugmaker’s rare disease subsidiary Alexion will take on a number of Pfizer’s preclinical gene therapy programs and technologies to advance its genomic medicines pipeline.

Gene Therapy 76

Gene Therapy Gene Genome Genomics 76

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

BioPharma Reporter

JUNE 23, 2022

BIO, the worldâs largest trade association for the biotech industry, has responded to the US Food and Drug Administration's draft guidance on developing human gene therapy products that incorporate genome editing (GE) of human somatic cells.

Gene Therapy 59

Gene Therapy Genome Genomics Gene 59

pharmaphorum

SEPTEMBER 12, 2022

Alongside drugs and therapeutics, data collection and technology enhancements have redefined the traditional healthcare experience, especially within oncology, and genomic profiling has become a significant factor in allowing for personalised care. Tailored oncology.

Genome 105

Genome Genomics Clinical Trials Clinical Trials 105

Pharmaceutical Technology

JUNE 16, 2023

Benefiting from Abu Dhabi’s improved genomics capabilities, the deal seeks to expand research into genomic medicine and genetic diseases to provide patients in the UAE and beyond with improved access to new tools and treatments.

Life Science 130

Life Science Genetic Disease Genome Genomics 130

Pharmaceutical Technology

SEPTEMBER 9, 2022

It is expected to expedite the development of a wide range of new therapeutics using the small non-coding area of the genome that is not explored. The company discovered mutations in small RNA genes that are connected to microvascular dysfunction in a previous study.

RNA 246

RNA Development Gene Therapy Gene 246

BioSpace

DECEMBER 11, 2023

Backed by ARCH Venture Partners and Fujifilm, as well as technology licensed from MIT, Tome is looking to create curative cell and integrative gene therapies.

Gene Therapy 104

Gene Therapy Genome Genomics Medicine 104

Pharmaceutical Technology

MARCH 8, 2023

Its focus areas include human systems, precision diagnostics, data sciences, next-generation biomanufacturing, and genomic medicines. This Beacon’s focus will be on product solutions that help address the bottlenecks impacting manufacturing yield and quality of cell therapies. The University of Pennsylvania Immunotherapy Richard W.

Gene Therapy 244

Gene Therapy Manufacturing Gene Production 244

Pharmaceutical Technology

MARCH 28, 2023

Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.

Gene Editing 235

Gene Editing DNA Gene Gene Therapy 235

pharmaphorum

SEPTEMBER 26, 2022

The past twelve months have culminated in an unprecedented level of excitement, investment, and clinical progress within the gene therapy field. This year’s agenda includes a novel track designed for quality control and process development groups working in gene therapy. Download the full event guide to find out more.

Gene Therapy 52

Gene Therapy Gene Development Bioassay 52

BioTech 365

FEBRUARY 10, 2021

Genethon and WhiteLab Genomics Join Forces to Enhance Gene Therapy Through Artificial Intelligence Genethon and WhiteLab Genomics Join Forces to Enhance Gene Therapy Through Artificial Intelligence PARIS–(BUSINESS WIRE)–WhiteLab Genomics, a specialist in artificial intelligence applied to gene and cell therapies, … Continue reading (..)

Gene Therapy 40

Gene Therapy Genome Genomics Gene 40

Worldwide Clinical Trials

JANUARY 24, 2024

Newborn Sequencing is a research initiative that allows for a baby’s full genome to be sequenced at birth, diagnosing their genetic diseases. Genetic counselors are critical in this pathway, especially as more is learned about the human genome and relevant incidental findings.

Genetics 189

Genetics Trials Clinical Trials Clinical Trials 189

pharmaphorum

NOVEMBER 12, 2020

UCB has made a new foray into the gene therapy space, buying fellow Belgium-based company Handl to get control of its adeno-associated virus (AAV) capsid delivery platform and two research programmes in neurodegenerative diseases. The post UCB revs up its gene therapy drive with Handl acquisition appeared first on.

Gene Therapy 52

Gene Therapy Gene Manufacturing Contract Manufacturing 52

BioPharma Reporter

MARCH 13, 2023

The extremely high costs of gene therapies are unsustainable, and a global commitment to affordable, equitable access to these treatments is urgently needed, concluded the organising committee of a conference on human genome editing.

Gene Therapy 52

Gene Therapy Gene Genome Genomics 52

pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. bluebird bio’s Philip Gregory.

Gene Therapy 59

Gene Therapy Gene Trials Gene Expression 59

Medical Xpress

NOVEMBER 21, 2022

In a new study published in Circulation: Genomics & Precision Medicine, Mayo Clinic researchers designed and developed the first suppression-replacement KCNH2 gene therapy for correcting both long QT syndrome (LQTS) and short QT syndrome (SQTS).

Gene Therapy 55

Gene Therapy Gene Genome Genomics 55

BioTech 365

MAY 31, 2021

WhiteLab Genomics and the Accelerator for Technological Research in Genomic Therapies Announce Their Collaboration on Artificial Intelligence for the Bioproduction of Gene Therapies WhiteLab Genomics and the Accelerator for Technological Research in Genomic Therapies Announce Their Collaboration on Artificial Intelligence … Continue reading → (..)

Genome 40

Genome Genomics Gene Therapy Gene 40

BioSpace

NOVEMBER 20, 2023

will receive $50 million over two years from Flagship to study somatic genomics with an eye to finding new targets for gene therapies. Quotient Therapeutics, co-located in Cambridge, Mass. and Cambridge, U.K.,

Gene Therapy 87

Gene Therapy Genome Genomics Gene 87

BioSpace

JUNE 24, 2024

Exsilio Therapeutics emerged from stealth on Tuesday with a platform that leverages mRNA technology to develop redosable genomic medicines for a range of complex diseases.

Gene Therapy 66

Gene Therapy Genome Genomics Gene 66

pharmaphorum

APRIL 27, 2021

Dosing can start once again in uniQure’s phase 3 trial of its haemophilia B gene therapy, after the FDA concluded that a case of liver cancer seen in the study was unlikely to be caused by the treatment. . The post FDA lifts hold on uniQure gene therapy after cancer case review appeared first on.

Gene Therapy 52

Gene Therapy Gene Genome Genomics 52

BioTech 365

OCTOBER 27, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: ElevateBio cuts a deal for Life Edit, adding genome engineering tech to support gene therapy pipeline.ElevateBio cuts a deal for Life Edit, adding genome engineering tech to … Continue reading →

Gene Therapy 40

Gene Therapy Engineer Genome Genomics 40

STAT News

JANUARY 30, 2023

The act also created an industry that didn’t exist in the United States before its enactment, enabling the formation of companies to develop and commercialize therapies for rare diseases. The human genome was sequenced in 2003. That said, a lot has changed in science and drug development since 1983.

RNA 111

RNA Gene Therapy Drugs Gene 111

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

Pharmaceutical Technology

MAY 17, 2023

The platform based on Scribe CRISPR by Design applies holistic engineering to convert bacterial immune systems into key genome editing technologies targeting the patients’ specific requirements. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Genetics 130

Genetics Medicine In-Vivo Gene Therapy 130

Pharmaceutical Technology

JULY 29, 2022

Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression. This treatment activates the OTC gene so that ammonia can be removed from the blood. This gene therapy will give the instructions to produce the enzyme arginine so that it can be hydrolysed to ornithine.

RNA 245

RNA Gene Expression Gene Gene Therapy 245

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna Genomics general manager and chief scientific officer Eric Huang said: “Through our collaboration with Life Edit, we hope to harness the power of gene editing technologies as part of our broader research and development engine, helping to advance our mission and deliver on the promise of mRNA.”

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

Gene Therapy 59

Gene Therapy Gene Gene Editing In-Vivo 59

Scienmag

JULY 12, 2021

Research team from the Universities of Göttingen and British Columbia investigates evaluations of breeding technology A research team from the University of Göttingen and the University of British Columbia (Canada) has investigated how people in five different countries react to various usages of genome editing in agriculture.

Genome 71

Genome Genomics Research Gene Therapy 71

XTalks

MARCH 21, 2025

Lee Honigberg on the Future of Blood-Based Biomarker Assays GeneDX GeneDX recently integrated AI (Multiscore) into its interpretation platform, improving speed and accuracy in analyzing complex genomic data. 10x Genomics 10x Genomics reported $707 million in 2024 revenue.

Genome 59

Genome Genomics FDA Approval Trials 59

BioSpace

OCTOBER 26, 2021

With the complete acquisition of Life Edit, ElevateBio will be able to integrate that company’s genome editing capabilities with its cell and gene therapies.

Gene Editing 76

Gene Editing Gene Gene Therapy Genome 76

Cloudbyz

JULY 31, 2024

This revolution is enabling the growth of innovative biomarker-based precision medicine and cell and gene therapy, transforming both clinical research and post-market care. These therapies involve modifying a patient’s cells or genes to treat or prevent diseases, with the promise of long-lasting effects.

Gene Therapy 52

Gene Therapy Gene Medicine In-Vitro 52

BioTech 365

NOVEMBER 17, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Test of adeno-associated viral vectors with gene therapy in dogs with hemophilia produces evidence of genomic changes.A

Gene Therapy 40

Gene Therapy Genome Genomics Gene 40

The Pharma Data

SEPTEMBER 21, 2020

. A biotech company spun out of the University of Edinburgh will utilise research to develop new gene therapies to target different types of cancer. The funding will be used to develop a pipeline of ‘highly selective’ gene therapies targeting cancer stem cells in a variety of solid tumour types.

Gene Therapy 52

Gene Therapy Gene Development Genome 52

Pharmaceutical Technology

SEPTEMBER 29, 2022

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Development 130

Development In-Vivo Genome Genomics 130

Pharmaceutical Technology

DECEMBER 14, 2022

GSK has entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets. The alliance will merge the PRISM oligonucleotide platform of Wave and the capabilities of GSK in genetics and genomics. By Cytiva Thematic.

Life Science 162

Life Science Development Gene Therapy Genetics 162

BioSpace

OCTOBER 20, 2020

Paris-based SparingVision, a genomic medicine company focused on ocular diseases, raised €44.5 million (approximately $52.2 million) in a financing round.

Gene Therapy 52

Gene Therapy Genome Genomics Gene 52