Bio Pharma Dive

MAY 16, 2023

Founded in early 2021, the company is using optogenetics to design treatments that could work across disease genotypes and in the later stages of vision loss.

Gene Therapy 324

Gene Therapy Genotype Gene 324

Pharmaceutical Technology

AUGUST 18, 2022

A custom-made, one-dose gene therapy, Zynteglo is indicated for such patients who need red blood cells (RBCs) transfusions on a regular basis. 0 genotypes. A genetic blood ailment, beta-thalassemia is caused by beta-globin gene mutations and results in a substantial decline or lack of production of adult haemoglobin.

Gene Therapy 246

Gene Therapy Gene Genotype In-Vivo 246

BioTech 365

JUNE 11, 2021

betibeglogene autotemcel (beti-cel) One-Time Gene Therapy for ?-thalassemia thalassemia Continues to Demonstrate Durable Efficacy Across Pediatric and Adult Patient Populations and All Genotypes in Data Presented at EHA2021 Virtual betibeglogene autotemcel (beti-cel) One-Time Gene Therapy for ?-thalassemia

Genotype 40

Genotype Gene Therapy Gene 40

pharmaphorum

DECEMBER 9, 2020

bluebird bio has presented long-term data from its Zinteglo one-time gene therapy for the blood disorder beta-thalassaemia, as the company continues talks with payers in Europe to bring the ultra-pricey treatment to market. The post Bluebird trumpets long-term data from beta-thalassaemia gene therapy appeared first on.

Gene Therapy 59

Gene Therapy Gene Genotype Marketing 59

BioTech 365

DECEMBER 5, 2020

Long-Term Data for bluebird bio’s betibeglogene autotemcel (beti-cel) Gene Therapy Show Patients Across Ages and ?-thalassemia

Genotype 40

Genotype Gene Therapy Gene 40

Pharmaceutical Technology

DECEMBER 15, 2022

Therefore, there is also a significant need for new therapies that target the underlying cause of CFD that may offer a cure for patients living with severe disease, or for patients with genotypes such as dysfibrinogenemia and hypodysfibrinogenemia, who are often ineligible for treatment with HFCs.

Marketing 130

Marketing Production Genotype Development 130

XTalks

JANUARY 21, 2025

Using cervical or self-collected vaginal specimens, the test works with the BD Viper LT and BD COR systems to detect individual genotypes, such as HPV 16, 18 and 31, and grouped results for other types. Companion diagnostic for Pfizers hemophilia B gene therapy Beqvez (idanacogene elaparvovec-dzkt).

FDA Approval 105

FDA Approval Manufacturing Development RNA 105