Inborn genetic diseases Archives

Blood clot made of red blood cells, platelets and fibrin protein strands. Thrombus

News & Features

Bluebird Tightens Sickle Cell Race with BLA for Gene Therapy

Malorye Branca -

April 25, 2023

News & Features

Brain Blood Vessel Disease Halted by Bone Marrow Transplant

Chris Anderson -

February 3, 2023

Sickle Cell disease - 3D Illustration of sickled blood cells on a dark background

Molecular Dx

“Spleen-on-a-Chip” Could Help Develop Better Therapies for Sickle Cell Disease

Helen Albert -

January 31, 2023

Illustration of a human head and neck showing a cut-through to the brain on the left hand side on a background picture of fluorescent neurons and with a DNA helix to the right hand side to represent genetic brain and neurological disorders such as the movement disorder ataxia.

Molecular Dx

Tandem Repeat Expansion Mutation Behind Late-Onset Movement Disorder Identified

Helen Albert -

December 15, 2022

News & Features

bluebird Nets Unanimous ‘Yes’ for CALD Gene Therapy Application

Malorye Branca -

June 10, 2022

Molecular Dx

Map of Human Blood Stem Cell Development Created

Helen Albert -

April 13, 2022

News & Features

Centogene and Agios Expand Rare Blood Disease Partnership

Malorye Branca -

November 5, 2021

News & Features

Vertex Taps Mammoth’s CRISPR Tech in Gene Therapy Deal Worth up to $695M

Alex Philippidis -

October 26, 2021

News & Features

Base Editor Effectively Treats Sickle-Cell Disease in Mice

Kevin Mayer -

June 7, 2021

Genetic engineering, GMO and Gene manipulation concept. Hand is inserting sequence of DNA. 3D illustration of DNA.

News & Features

Vertex Sweetens Deal with CRISPR Therapeutics by $900M for Blood Disorders Gene Therapy Candidate

Chris Anderson -

April 20, 2021

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DeepMind’s Next-Gen Protein Structure Predictor AlphaFold 3 Released

AI Improves Prostate Cancer Recurrence Prediction

Targeting Iron Uptake Shows Promise for Treating Asthma and Other Allergic…

Zenas Nets $200M to Advance mAb for Autoimmune Diseases

Altering CAR Proteins Improves T Cells’ Ability to Attack Solid Tumors

Inborn genetic diseases

Bluebird Tightens Sickle Cell Race with BLA for Gene Therapy

Brain Blood Vessel Disease Halted by Bone Marrow Transplant

“Spleen-on-a-Chip” Could Help Develop Better Therapies for Sickle Cell Disease

Tandem Repeat Expansion Mutation Behind Late-Onset Movement Disorder Identified

bluebird Nets Unanimous ‘Yes’ for CALD Gene Therapy Application

Map of Human Blood Stem Cell Development Created

Centogene and Agios Expand Rare Blood Disease Partnership

Vertex Taps Mammoth’s CRISPR Tech in Gene Therapy Deal Worth up to $695M

Base Editor Effectively Treats Sickle-Cell Disease in Mice

Vertex Sweetens Deal with CRISPR Therapeutics by $900M for Blood Disorders Gene Therapy Candidate

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