XTalks

DECEMBER 20, 2023

CRISPR Gene Editing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason. Given its ability to precisely edit genes, there is tremendous promise for the revolutionary technology to offer cures for genetic disorders and to treat various diseases.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

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

pharmaphorum

JANUARY 10, 2022

With the two approvals acting as a validation of the technology, larger companies are prepared to make larger investments to ensure that they are at the forefront of developments in RNA-focused therapies.

RNA 111

RNA Vaccination Vaccine Pharma Companies 111

XTalks

SEPTEMBER 4, 2024

The HELIOS-B results build on earlier success from the HELIOS-A trial, which led to the US Food and Drug Administration (FDA) approval of vutrisiran (Amvuttra) for polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (hATTR).

Trials 52

Trials RNA Protein Gene Editing 52

XTalks

MARCH 28, 2024

For CRC, treatment options that are targeting genetic mutations such as BRAF , KRAS and HER2 have been developed. Gene Editing and CAR T-Cell Therapy Genetic engineering technologies, such as CRISPR-Cas9, are providing newer potential avenues for cancer treatment.

Gene Editing 59

Gene Editing Life Science Clinical Trials Clinical Trials 59

XTalks

DECEMBER 22, 2021

RNA therapeutics hold promise as powerful treatments for diseases, including those that are genetic-based. The gene-editing tool CRISPR edits DNA using specialized strands of guide RNA and endogenous cellular mechanisms of RNA interference. RNA Therapeutics. The deal is worth $1.5

Life Science 98

Life Science RNA Vaccination Vaccine 98

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. In 2020, 31 out of 53 novel drug approvals were for rare or orphan diseases​​.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

pharmaphorum

NOVEMBER 2, 2021

Clinical interest in bsAbs gained momentum following the promising data and market success of blinatumomab (BLINCYTO®, Amgen), a fragment-based bispecific T cell engager (BiTE), which first received FDA approval in 2014 and EMA approval in 2015.

Antibody 52

Antibody Immune Response Production Engineer 52

XTalks

DECEMBER 29, 2022

As of December 16, 2022, there are seven US Food and Drug Administration (FDA) approved gene therapies. In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. In 2023, a number of gene therapies are expected to get the FDA green light.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize.

Life Science 111

Life Science Vaccination Vaccine Gene 111

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

JANUARY 6, 2025

Notably, the ASO drug Vitravene (fomivirsen) received US Food and Drug Administration (FDA) approval for treating cytomegalovirus (CMV) retinitis in AIDS patients. Additionally, CRISPR genome-wide screening holds great potential for identifying key disease-associated genes and uncovering novel therapeutic targets.

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111