pharmaphorum

JANUARY 10, 2022

The company has also stated, at the same time as the study was announced, that it will look at developing mRNA technology in oncology and genetic diseases. At present, the biotech has 10 vaccine candidates at various stages of the preclinical and discovery phase, focused across three different areas of infectious diseases.

RNA 111

RNA Vaccination Vaccine Pharma Companies 111

pharmaphorum

APRIL 20, 2022

The potential of CRISPR technology has been a hot topic in the industry ever since it was first developed, but as trials progress further into the clinic, what therapeutic areas could be set to benefit? Cancer is a genetic disease, caused by certain changes in the way that genes control cell function, such as how they grow and divide.

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Gene Editing DNA Gene Genetics 52

pharmaphorum

FEBRUARY 17, 2022

The drug – which costs $125,000 at US list prices – can be used either alone or in combination with hydroxyurea, a well-established therapy for the disease. CRISPR Therapeutics and Vertex are also in the running with their gene-editing candidate CTX001, in phase 1/2 trials which are due to generate final results later this year.

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Drugs Gene Therapy Gene Editing Gene 59

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

pharmaphorum

SEPTEMBER 14, 2022

SparingVision has raised €75 million in second-round funding that will be used to fund clinical trials of gene therapies for ocular diseases retinitis pigmentosa (RP) and dry age-related macular degeneration (AMD). The current schedule is for trials to start in 2024, with first safety and efficacy data coming a year later.

Gene Therapy 52

Gene Therapy Gene Gene Sequencing Gene Editing 52

XTalks

DECEMBER 29, 2022

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. Given their precise targeting, gene therapies have largely been focused on oncology indications followed by rare genetic diseases.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

XTalks

DECEMBER 22, 2021

The gene-editing tool CRISPR edits DNA using specialized strands of guide RNA and endogenous cellular mechanisms of RNA interference. Double-stranded RNA is flagged by cells as foreign, triggering the cell’s RNA interference machinery to dice it up and destroy it. The deal is worth $1.5

Life Science 98

Life Science RNA Vaccination Vaccine 98

XTalks

JANUARY 6, 2025

For example, antisense oligonucleotide (ASO)-based therapies have gained traction, with 100 Phase I clinical trials initiated and around 25 percent of these advancing to Phase II or Phase III trials in recent years. 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