Delveinsight

JANUARY 14, 2021

Technologists are getting better at coding biology, and venture firms are flooding a new generation of startups with cash to commercialize their technology bringing in the next wave of genetic innovation. Verve Therapeutics unveils its lead program for treating genetically high cholesterol.

Gene Editing 40

Gene Editing Gene Genetics DNA 40

XTalks

NOVEMBER 3, 2023

CRISPR works as genetic scissors to edit parts of the genome. The CRISPR-Cas9 gene editing system was first discovered to be endogenous in bacteria. Given the system’s powerful ability to make cuts in genes in a precise manner, the system is being leveraged in gene therapies.

Gene Therapy 59

Gene Therapy Gene Gene Editing In-Vivo 59

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

XTalks

DECEMBER 29, 2022

The life sciences and healthcare are among the biggest industries globally, and their significance was particularly highlighted during the past couple of years by the COVID-19 pandemic. Given the hyperfocus on the life sciences thanks to COVID, consumers appear to be more autonomous and vocal about their medical demands and choices.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

Pharmaceutical Technology

FEBRUARY 13, 2023

Gene therapies are an attractive way of targeting the underlying genetic mutations, but traditional approaches cannot be used while targeting mutations for Dravet Syndrome, forcing researchers to develop new ways. Meanwhile, others are researching the link between gene variation and different responses to treatments.

Research 264

Research Gene Gene Therapy Genome 264

XTalks

DECEMBER 22, 2021

If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the life sciences. Here is a look at some life science trends to expect in the coming year, including key themes and topics in the pharmaceutical, biotechnology and medical device industries to keep an eye out for in 2022.

Life Science 98

Life Science RNA Vaccination Vaccine 98

pharmaphorum

AUGUST 31, 2022

One area of the gene therapy space that could become a major treatment modality is CRISPR technology. The key to why this technology could become central to gene editing is due to the specificity with which it can edit DNA and that it is also a relatively cheap process, compared with existing technologies.

Gene Therapy 98

Gene Therapy Gene Gene Editing DNA 98

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself.

DNA 98

DNA Gene Gene Silencing Genome 98

XTalks

JANUARY 4, 2024

Hemophilia B, also known as congenital Factor IX (FIX) deficiency or Christmas disease, is a rare genetic bleeding disorder caused by a deficiency or defect in the FIX clotting protein. Pfizer said that according to the World Federation of Hemophilia, more than 38,000 people worldwide were living with hemophilia B in 2021.

Gene Therapy 111

Gene Therapy Gene Gene Editing Sales 111

XTalks

DECEMBER 13, 2023

Casgevy’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the US. In 2022, bluebird won approvals for two gene therapies — Skysona for the treatment of the rare neurological disorder cerebral adrenoleukodystrophy (CALD) and Zynteglo for beta-thalassemia.

Gene Therapy 98

Gene Therapy Gene FDA Approval In-Vivo 98

XTalks

MARCH 28, 2024

For CRC, treatment options that are targeting genetic mutations such as BRAF , KRAS and HER2 have been developed. Building Cancer Awareness and Empowering Métis Youth: Featuring Jordyn Playne, President, Métis Nation of Ontario Youth Council – Xtalks Life Science Podcast Ep.

Gene Editing 59

Gene Editing Life Science Clinical Trials Clinical Trials 59

Pharmaceutical Technology

MAY 26, 2023

Tripling its production capacity, Touchlight can now manufacture 8kg plasmid DNA, a key component for mRNA gene therapies and vaccines. As per the company, dbDNA is suitable for rapid, scalable manufacture of GMP DNA and can incorporate gene sequences of sizes ranging from 500bp to 20kb, which are typically unstable as pDNA.

DNA 130

DNA Manufacturing Gene Gene Therapy 130

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself.

DNA 52

DNA Gene Gene Silencing Genome 52

XTalks

NOVEMBER 30, 2023

More Investigational Therapies for HIV Are on the Way American Gene Technologies, a pioneering biotech company headquartered in Rockville, Maryland, has released promising results from its Phase I trial of AGT103-T, a gene therapy designed for individuals with chronic HIV disease.

FDA Approval 111

FDA Approval Gene Therapy Gene Clinical Trials 111

XTalks

SEPTEMBER 4, 2024

The HELIOS-B study’s findings are timely as new gene-editing therapies, like the one being studied in MedStar Health’s Phase III MAGNITUDE trial for ATTR-CM, are also being explored. The MAGNITUDE trial uses CRISPR/Cas9 technology to target the genetic source of the disease, potentially offering a one-time treatment.

Trials 52

Trials RNA Protein Gene Editing 52

XTalks

FEBRUARY 11, 2021

This has ushered in a new era of genomics that is fostering rapid, detailed and personalized insights into human genetics. Xtalks is celebrating the International Day of Women and Girls in Science with a special overview of notable female scientists who have made revolutionary contributions to the field of genetics.

Genetics 119

Genetics DNA Genome Genomics 119

XTalks

SEPTEMBER 21, 2020

A study from the University of California San Francisco (UCSF) has revealed that women with Alzheimer’s do better than men with the disease due to a genetic advantage conferred by their extra X chromosome. Genetic Sex Differences in Disease.

Gene 89

Gene Protein Gene Expression Scientist 89

XTalks

NOVEMBER 29, 2024

Kristin Yarema, President of Cell Therapy at Poseida Therapeutics – Xtalks Life Science Podcast Ep. 129 Through this acquisition, Roche secures proprietary technologies such as Poseida’s Cas-CLOVER gene editing platform and in-house good manufacturing practice (GMP) manufacturing capabilities.

Gene Therapy 100

Gene Therapy Gene Gene Editing Genetic Engineering 100

XTalks

NOVEMBER 7, 2024

Casgevy: A Game-Changer for Beta Thalassemia CRISPR Therapeutics and Vertex are transforming blood disorder treatment with their gene-editing solution Casgevy (exagamglogene autotemcel [exa-cel]). This one-time gene therapy tackles transfusion-dependent beta-thalassemia (TDT), a disorder causing severe anemia.

Gene Therapy 105

Gene Therapy Gene Editing Gene FDA Approval 105

XTalks

DECEMBER 24, 2020

The pandemic propelled the life science and healthcare sectors onto center stage, and they rose to the occasion against the most unprecedented health challenge in recent times. While COVID-19 has undoubtedly been the biggest story in the life science industry in 2020, it was a busy and positive year in many other areas.

Life Science 111

Life Science Vaccination Vaccine Gene 111

XTalks

JANUARY 6, 2025

Gene Editing CRISPR-Cas9 and related gene-editing technologies continue to advance, and today they are widely used to study and develop therapeutic approaches for a broad range of human diseases. Furthermore, CRISPR/Cas9 presents a promising avenue for overcoming genetic diseases in the near future.

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111

XTalks

FEBRUARY 11, 2025

Gene therapy has shown promise in treating cancers that are particularly difficult to manage such as neuroblastoma and Wilms tumor using CAR-T cell therapy, CRISPR-Cas9 gene editing and RNA-based interventions to target genetic drivers of disease.

Gene Therapy 66

Gene Therapy Clinical Trials Clinical Trials Trials 66