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Home » sickle cell disease

Articles Tagged with ''sickle cell disease''

Mast cell releasing histamine during allergic response

EHA 2022: Best in class? Cogent soars on early data in mastocytosis

June 10, 2022
By Jennifer Boggs
Analysts have already started tagging Cogent Biosciences Inc.’s bezuclastinib as potentially best in class, after the company presented impressive, though early stage, data at the European Hematology Association Congress in Vienna demonstrating promising efficacy and a possibly differentiating safety profile for the selective KIT D816V inhibitor in advanced systemic mastocytosis.
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Third sacker: Bluebird plots major-league layoffs to stay in gene therapy game

April 5, 2022
By Randy Osborne
Bluebird Bio Inc. became the latest in a spate of gene therapy firms to disclose restructuring plans, as the company aims to save $160 million over the next two years, saying goodbye to about a third of its workforce. It’s the other shoe to drop after Cambridge, Mass.-based Bluebird rattled Wall Street with phraseology in the firm’s fourth-quarter earnings report March 4 that expressed “substantial doubt” regarding whether operations could go on.
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Wall Street MAD about Fulcrum’s interim phase I data in sickle cell

Aug. 10, 2021
By Randy Osborne
Fulcrum Therapeutics Inc. shares (NASDAQ:FULC) closed at $18.77, up $10.44 or 125%, on word of positive interim results from a phase I trial in healthy adult volunteers with oral FTX-6058 for sickle cell disease (SCD). The firm has “already achieved maximal target engagement [MTE] at all three doses,” said Christopher Morabito, the company’s chief medical officer. “I don’t think we’ll exceed that.”
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WHO setting up global framework to guide genome editing research

July 12, 2021
By Nuala Moran
LONDON – The World Health Organization (WHO) is to set up a channel for confidential reporting of illegal, unregistered, unethical or unsafe human genome editing research, as part of a new governance framework it is proposing to develop.
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Sickle cell illustration

Neutigers to study use of wearables and AI to improve sickle cell outcomes

June 24, 2021
By Meg Bryant
Princeton University spinout Neutigers Inc. is launching a study to explore the use of artificial intelligence (AI) and everyday wearables to flag early symptoms of sickle cell anemia vaso-occlusive crisis (VOC) before they get worse and land patients in the hospital. The aim is to reduce deaths and facilitate interventions to address the entire continuum of care for patients with the inherited red blood cell disorder, Adel Laoui, founder and CEO, told BioWorld.
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Researcher in lab using Gazelle

Hemex’s newborn screening for sickle cell disease is more than a baby step forward

June 14, 2021
By Annette Boyle
Hemex Health Inc.’s newborn screening for sickle cell disease substantially reduces the labor involved for parents and providers in testing for the potentially fatal condition. The test had previously been able to test infants 6 weeks and older on the company’s Gazelle platform.
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Red blood cells, DNA

Holds on four Bluebird gene therapy studies are released by the FDA

June 7, 2021
By Lee Landenberger
The FDA has lifted clinical holds on four studies from Bluebird Bio Inc., following recent similar actions with other gene therapy programs. Two of the studies concern phase I/II and phase III clinical trials of the gene therapy Lentiglobin (BB-1111) in treating sickle cell disease. The remaining two studies are phase III clinical trials of betibeglogene autotemcel gene therapy, which share a vector with Lentiglobin, for treating transfusion-dependent beta-thalassemia.
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CRISPR bakin’: Intellia on sizzle as Wall Street crunches available data

April 22, 2021
By Randy Osborne
Advances lately in the genome-editing space include Beam Therapeutics Inc. publication in The CRISPR Journal details of its work with inlaid base editors, which the firm is applying in the BEAM-102 program for sickle cell disease. IBEs’ predictable, shifted editing window lets researchers go after disease-causing mutations that canonical base editors cannot reach, Beam said, and do the job with high efficiency and few off-target effects on the genome. The hottest news due in the near-term future from the sector will spill from Intellia Therapeutics Inc., of Cambridge, Mass., which is due to roll out first-in-human data with a systemic CRISPR-based genome editing therapy, NTLA-2001, in hereditary transthyretin amyloidosis.
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Test tubes with blood samples

Vertex and CRISPR in a $1.1B deal to develop sickle cell, beta-thalassemia therapy

April 20, 2021
By Lee Landenberger
In biotech and biopharma’s third-largest ever up-front development and commercialization deal, Crispr Therapeutics AG will receive an initial $900 million in an amended deal with Vertex Pharmaceuticals Inc. to lead the development, manufacturing and commercialization of gene editing therapy CTX-001 for sickle cell disease and transfusion-dependent beta-thalassemia.
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Sickle cell illustration

Bluebird seeking to resume SCD trials, clear concerns about vector

March 10, 2021
By Michael Fitzhugh
Just weeks after two unexpected cases of blood cancer landed trials of its investigational gene therapies for sickle cell disease (SCD) and beta-thalassemia on FDA-issued clinical holds, Bluebird Bio Inc. said it's talking to regulators about their resumption after what RBC analyst Luca Issi called a "partial exoneration" of the BB-305 lentiviral vector shared between the medicines.
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