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Home » sickle cell disease

Articles Tagged with ''sickle cell disease''

Graphite’s nula-cel perseveres as Kamau emerges from stealth

Dec. 8, 2023
By Amanda Lanier
Following a strategic transaction with Graphite Bio Inc., Kamau Therapeutics is emerging from stealth with sickle cell treatment nulabeglogene autogedtemcel (nula-cel). Kamau received an option to acquire all of Graphite’s genome editing assets, including a platform technology that integrates precision DNA repair using homology directed repair and CRISPR/Cas9, as well as the autologous CRISPR/Cas9 gene corrected CD34+ cell product nula-cel, which offers a potential cure for sickle cell disease derived from the patient's cells.
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Sickle cells

US FDA approves first CRISPR-based gene therapy Casgevy for sickle cell; Lyfgenia gets nod, too

Dec. 8, 2023
By Karen Carey and Mari Serebrov
Both Vertex Pharmaceuticals Inc.’s Casgevy (exagamglogene autotemcel, exa-cel) and Bluebird Bio Inc.’s Lyfgenia (lovotibeglogene autotemcel, lovo-cel) received U.S. FDA approval Dec. 8, providing 16,000 American sickle cell patients who have recurring vaso-occlusive events with access to the first cell-based gene therapies.
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Red blood cells, DNA

Historic MHRA exa-cel win bagged, Crispr/Vertex await FDA nod

Nov. 16, 2023
By Randy Osborne
With a landmark U.K. approval in hand for Casgevy (exagamglogene autotemcel [exa-cel]) to treat sickle cell disease and transfusion-dependent beta thalassemia, Crispr Therapeutics AG and partner Vertex Therapeutics Inc. are turning their attention to the PDUFA dates set by the U.S. FDA for the treatment in both conditions.
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Sickle cell illustration

Adcom: Don’t sacrifice progress for perfection

Nov. 1, 2023
By Mari Serebrov
Had it been asked to, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee would have voted Oct. 31 to recommend approval of Vertex Pharmaceutical Inc. and Crispr Therapeutics AG’s exagamglogene autotemcel, or exa-cel, as a one-time transformative treatment for severe sickle cell disease in individuals 12 and older.
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Sickle cell disease 3D illustration

No red flags flying ahead of adcom on Vertex’s SCD therapy

Oct. 27, 2023
By Lee Landenberger
It’s just a discussion next week of Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG’s sickle cell disease (SCD) therapy, with no vote expected from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee. The FDA isn’t questioning the drug’s efficacy as much as it is concerned about the effects of off-target editing.
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Hepatitis B virus
Drug Design, Drug Delivery & Technologies

Precision Biosciences outlines pipeline progress

Sep. 12, 2023
Precision Biosciences Inc. uses its proprietary Arcus platform to develop in vivo gene editing therapies and has outlined new data from its wholly owned and partnered pipeline.
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Sickle cells

With hold lifted, Fulcrum restarts phase Ib of FTX-6058 in sickle cell disease

Aug. 22, 2023
By Karen Carey
Shares of Fulcrum Therapeutics Inc. shot up 38.5% on Aug. 22 following news that the U.S. FDA had lifted the clinical hold on the company’s phase Ib sickle cell disease candidate, FTX-6058.
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Sickle cell disease

Trinity Biotech gets FDA nod for hemoglobin diagnostic system

Aug. 9, 2023
By Shani Alexander
Trinity Biotech plc. received U.S. FDA 510(k) clearance for its lab-based hemoglobin diagnostic system, the Premier Resolution system, which the company hopes will allow it to regain its market leading position in hemoglobin variant detection. The Premier Resolution system is an automated analyzer which quantifies fetal hemoglobin and hemoglobin A2 and detects more than 200 hemoglobin variants. The device is a modern successor to the company’s Ultra system which once held a leading position in the U.S. hemoglobin variant diagnostic market.
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Hematologic

Scribe Therapeutics and Sanofi collaborate on in vivo therapies for sickle cell disease

July 18, 2023
Scribe Therapeutics Inc. has announced an expanded collaboration with Sanofi SA, under which Sanofi receives an exclusive license to use Scribe’s CRISPR X-Editing (XE) genome editing technologies for the development of in vivo therapies, including for sickle cell disease.
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Sickle cell illustration
Genetic/Congenital

Base editing could be more potent than CRISPR-Cas9 for treating hemoglobinopathies

July 10, 2023
By Helen Albert
Research led by St. Jude Children’s Research Hospital and Harvard University shows base-editing approaches could be more effective than CRISPR-Cas9 gene-editing approaches for treating conditions such as sickle cell disease and β-thalassemia. Writing in the July 3, 2023, issue of Nature Genetics, the researchers compared three base-editing approaches with two CRISPR-Cas9 approaches to increasing levels of fetal hemoglobin in CD34+ hematopoietic stem and progenitor cells, and found one of the base-editing approaches was the most potent.
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