pharmaphorum

MARCH 10, 2022

18 months after signing its first partnering deal with Genentech, Scenic Biotech has raised $31 million in first-round financing that it will use to take its own genetic modifier-targeted medicines into clinical trials. The post Scenic Bio raises $31m for genetic modifier platform appeared first on.

Genetics 98

Genetics Gene Sequencing Gene Clinical Trials 98

pharmaphorum

JUNE 23, 2022

The deal comes a year after Intellia and partner Regeneron reported promising results from the first clinical trial of a drug used to edit the genomes of cells within the body, in patients with rare disease ATTR amyloidosis. billion in potential milestones if the project advances through development and onto the market.

Gene Editing 97

Gene Editing In-Vivo Gene Sequencing Gene 97

pharmaphorum

APRIL 23, 2021

billion in milestone payments in the deal, which will focus on using small-molecule compounds to “fine tune” the effects of genetic medicines in the body. The approach relies on the use of drug responsive domains (DRDs) on the gene-editing medicine, which can be used to either increase or decrease protein expression.

Gene Editing 52

Gene Editing Genetics Drugs Gene 52

pharmaphorum

FEBRUARY 22, 2021

Bluebird Bio’s decision to hit pause on the launch of Zynteglo for beta thalassaemia after two cases of cancer were seen in a clinical trial could see fears over the safety of viral vectors used to deliver gene therapies resurface. . million in Europe.

Gene Therapy 52

Gene Therapy Gene Gene Sequencing Gene Expression 52

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