GENE ONLINE|News &
Opinion
Blog

.

Зеркало официального сайта всегда доступно для входа на Мостбет, даже при ограничениях. Скачать Mostbet – это шаг к удобству и азартным развлечениям.

Если вы ищете место для увлекательных ставок, обратите внимание на Joycasino, где лучшие предложения для игроков ждут вас.
2022-04-27| Trials & Approvals

CRISPR Biotech Editas Obtains FDA Rare Pediatric Disease Label

by Fujie Tham
Share To

Cambridge, MA-based Editas Medicine recently announced that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease designation to EDIT-301, an investigational, gene-edited medicine for treating beta thalassemia. Beta thalassemia is a blood disorder that reduces hemoglobin production, the protein responsible for carrying oxygen.

Following their first designation for treating sickle cell disease, this second FDA label will lead to initiation of Phase I/II study of EDIT-301 in transfusion-dependent beta thalassemia patients this year.

Related Article: Landmark CRISPR Trial Shows Promise Against Fighting Blindness, but Is It a Cure?

 

Editas’ EDIT-301

 

EDIT-301 is an experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). This therapeutic consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin genes (HBG1, HBG2) promoters, with potential to provide a one-time, persistent treatment benefit for people living with severe sickle cell disease and transfusion-dependent beta thalassemia.

Currently, Editas boasts 10 pipelines revolving around in-vivo gene-edited medicines and ex-vivo gene-edited cell medicines, with research and development efforts focused on diseases which there are currently few or no treatments available. Under its Ex Vivo Gene Edited Cell Medicines catalog, Editas is in partnership with Bristol-Myers Squibb to develop the next generation of ex-vivo gene-edited cell medicines for cancer immunotherapy. 

Editas Medicine was featured as one of GeneOnline’s 10 top gene therapy companies to look out for in 2021.

©www.geneonline.com All rights reserved. Collaborate with us: [email protected]
Related Post
BIO Asia-Taiwan 2024 Conference has Officially Started, With International Leaders in the Industry Gathering Despite the Storm
2024-07-25
Wistar and Accelerated Biosciences Partner to Develop Novel hTSCs Platform for Immunotherapies
2024-07-15
Revolutionizing Treatment: ASGCT’s Clinical Trials Spotlight on Immunotherapy, Cancer Vaccines, and Auditory Diseases
2024-05-09
LATEST
Novo Nordisk Bets Big on CagriSema With A Heavyweight Move Planned for 2026
2025-02-07
Major Win – FDA Grants Fast Track Designation to Adicet Bio’s ADI-001 for Lupus Disease
2025-02-07
First-of-its-Kind Diagnostic Aid Uses a Single Hair Strand to Detect Autism Risk in Babies
2025-02-06
Teladoc Dials Up Virtual Care with $70 Million Acquisition of Catapult
2025-02-06
Silicon Valley in the Crosshairs as EU Readies to Strike U.S. Big Tech
2025-02-06
Johnson & Johnson Under Fire Again as Legal Action Over Alleged Talc-Cancer Link Moves to the UK
2025-02-05
MediBuddy Partners with Japan’s ELECOM to Launch Smart Health IoT Devices in India
2025-02-05
EVENT
2025-02-05
Precision Medicine World Conference 2025
Santa Clara, California
2025-02-19
Healthcare Conference Taipei 2025
Taipei, Taiwan
2025-03-17
BIO-Europe Spring 2025
Milan, Italy
2025-04-21
World Vaccine Congress 2025
Washington, U.S.A
2025-04-25
AACR Annual Meeting 2025
Chicago, U.S.A
Scroll to Top