SCD

Portland-based Hemex Health announced Monday a software-based upgrade that expands the age range for sickle cell disease (SCD) tests on its portable, low-cost Gazelle platform from 6 months and up to all ages, including newborns.

In other SCD news, San Francisco’s Global Blood Therapeutics said Monday that voxelotor—its oral, once-daily treatment for the disease—has notched Promising Innovative Medicine designation from British regulators.

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Continuing its breakneck business pace, gene-editing specialist Graphite Bio—a Stanford spinout that just launched in September 2020—filed for an IPO last week.

San Francisco-based Graphite secured U.S. FDA clearance in December 2020 for Phase 1/2 trials of GPH101, its investigational sickle cell therapy.

Boston-based Vertex Pharmaceuticals and Swiss biotech CRISPR Therapeutics announced a significant amendment on Tuesday to their collaboration on a potential sickle cell disease (SCD) cure.

Vertex will pay CRISPR $900 million upfront (plus up to $200 million in milestones) to expand its interest in CTX001, an investigational CRISPR/Cas9-based gene editing therapy that the companies say may provide a cure to both SCD and transfusion-dependent beta-thalassemia (TDT).

In other gene…

Portland-based Hemex Health announced Monday that its Gazelle Malaria one-minute point-of-care test outperforms current rapid diagnostic tests (RDTs) and is “nearly as accurate” as expert microscopy in detecting P. vivax malaria.

The press release referenced trial results from Brazil published last week in Malaria Journal: compared to optical microscopy, the battery-powered Gazelle test achieved 96.2% sensitivity versus 83.9% sensitivity for RDTs, according to the article. (Both…

Novartis announced Wednesday a collaboration with the Gates Foundation to develop an accessible in vivo gene therapy for sickle cell disease (SCD) that could potentially be administered once, directly to a patient, without the need to modify the cells in a lab. The collaboration aims to provide affordable and practical gene therapy for low-resource settings, the press release said.

In other SCD news, Massachusetts-based Bluebird Bio announced Tuesday that it has temporarily suspended…

Fast out of the starting gate, new gene editing entrant Graphite Bio announced last week that it has already won the go-ahead for Phase 1/2 trials of its sickle cell gene editing therapy, GPH101. The San Francisco-based company also announced this week that former Novartis CEO Joe Jimenez and former Celgene executive Perry Karsen have joined its board of directors.

Graphite Bio launched in September 2020.

Hemex Health released initial results at this week’s American Society of Hematology annual meeting showing that its combined anemia and sickle cell disease (SCD) test achieved 100% sensitivity and over 92.3% specificity for anemia (with 100% accuracy for hemoglobin variants) in 46 patients.

The Portland-based company expects to launch the combo test (through a software upgrade) on its low-cost, compact Gazelle platform in mid-2021 in selected countries, according to the press release…

An investigational CRISPR-Cas9-based sickle cell disease treatment from Intellia Therapeutics has received Gates Foundation funding, the Massachusetts-based biotech revealed Wednesday.

Co-founded by Jennifer Doudna—who earned the 2020 Nobel Prize in chemistry for her genome editing work—Intellia is developing a platform that delivers CRISPR-Cas9 intravenously, “potentially avoiding the need for bone marrow transplantation surgery,” according to the company’s press release.

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Portland-based Hemex Health announced this week that its design for a “one-minute, $0.25” non-invasive Sickle cell, Malaria, and Anemia Rapid Test (SMART) has won $100,000 with its third-place finish in the NIH Technology Accelerator Challenge.

SMART, designed in collaboration with other researchers, will build on Hemex’s existing minimally invasive Gazelle platform and will screen for the three diseases using an optical finger sensor provided by Dublin-based Medtronic, according to…

San Francisco gene-editing company Graphite Bio launched today with $45 million in Series A funding. The Stanford spinout’s press release said its lead sickle cell disease program aims to correct the sickle β globin gene’s single nucleotide point mutation.

In other news, Massachusetts-based GreenLight Biosciences today announced $3.3 million of Gates Foundation funding to develop mRNA-based gene therapies for sickle cell disease and other challenges as well as a gene-editing platform…