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.

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.

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.

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.

With CRISPR-Cas9 technology making its way toward clinical practice, laboratories are studying different gene-editing techniques, from base editors to prime editors, to correct mutations associated with various pathologies. Researchers at Tessera Therapeutics Inc. have been inspired by retrotransposons to develop a tool for editing DNA using RNA and reverse diseases such as phenylketonuria (PKU) or sickle cell disease (SCD).

Barring truly major surprises, exagamglogene autotemcel (Exa-cel, Vertex Pharmaceuticals Inc.) is on track to become the first approved CRISPR-based gene editing therapy. It is partly in expectation of Exa-cel’s approval that the European Hematology Association (EHA) and the European Society for Bone Marrow Transplantation hosted a session on “transplantation versus gene therapy in sickle cell disease.”

A proof of concept of ex vivo genetic modification of cells from patients and their transplantation in mice has demonstrated, for the first time, the therapeutic possibilities of prime editing in sickle cell disease (SCD).

Somatic human genome editing has made huge strides in the past five years, but the likely extremely high prices will be unsustainable. A global commitment to affordable, equitable access is urgently needed because the costs and infrastructure needs of this form of treatment are not manageable for either patients or health care systems.

Sickle cell disease (SCD) is the most common congenital disorder. The main symptoms of...