sickle cell disease
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Consumer / Employer, Health Tech
New App Aims To Support Teens with Sickle Cell Disease
HPC International launched Pinpoint, an app that helps teens manage their sickle cell disease. It allows them to learn about the disease, track their pain and communicate with their physicians.
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FDA Halts Fulcrum Trial, the Latest Setback in a Week of Sickle Cell Stumbles
Fulcrum Therapeutics offered few details about the clinical hold the FDA placed on its Phase 1b test of its sickle cell disease drug. Graphite Bio, Intellia Therapeutics, and Sangamo Therapeutics also encountered setbacks to their sickle cell programs.
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Payer’s Place: Dawn Maroney
Dawn Maroney, President, Markets of Alignment Health and CEO of Alignment Health Plan, to discuss how they are using technology to provide better service and care to consumers.
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Serious Side Effect Sidelines Gene-Edited Sickle Cell Therapy from Graphite Bio
Graphite Bio voluntarily paused a Phase 1/2 test of its gene-edited therapy for sickle cell disease after the first patient in the study developed a serious blood complication. The setback will also delay plans to reach the clinic with another genetic medicine in its pipeline.
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Novo Nordisk to buy sickle cell disease biotech Forma Therapeutics in $1.1B deal
Novo Nordisk announced that it will buy Forma Therapeutics, a clinical-stage biotech focused on rare hematologic diseases, in an deal valued at $1.1 billion. The deal is the second billion-dollar sickle cell disease deal the pharmaceutical industry has seen in the last 30 days — Pfizer forked over $5.4 billion in August to buy Global Blood Therapeutics.
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Pfizer to buy sickle cell disease biotech Global Blood Therapeutics in $5.4B deal
Pfizer’s own R&D efforts in sickle cell disease have fallen short, so the pharmaceutical giant is turning to M&A to gain a presence in the blood disorder. The acquisition of Global Blood therapeutics brings the commercialized drug Oxbryta plus a pipeline of other therapeutic candidates.
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Novartis picks Precision Bio to bring in vivo gene editing meds to blood disorders
Novartis is paying Precision Bio $75 million up front to begin a partnership aiming to develop in vivo gene-editing therapies for serious genetic blood disorders, such as sickle cell disease. One of the goals of the alliance is to make genetic medicines more globally accessible.
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Imara’s two trial flops in blood disorders dim drug’s prospects in heart failure
The lead drug candidate of Imara failed two separate mid-stage clinical trials evaluating it in rare, inherited hemoglobin disorders. Imara had been planning to advance the small molecule to yet another Phase 2 study in heart failure, but in light of the twin trial failures, the biotech is now weighing “strategic options.”
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Sanofi’s overhaul of genomic medicines strategy spells end of Sangamo alliance
A Sanofi strategy shift is leading to the end of a gene-editing alliance with Sangamo Therapeutics. Though the partnership has produced encouraging early clinical data with a sickle cell therapy highly personalized to the patient, Sanofi now plans to focus on universal approaches to genomic medicines.
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Applying Remote Patient Monitoring to Surgery Prep and Recovery, Oncology and Women’s Health
Join us to learn about the latest trends in remote monitoring and how to extend its benefits beyond chronic conditions to more patients – all while using fewer staff resources.
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With sickle cell disease study in sight, Graphite Bio draws up a $238M IPO
Gene-editing company Graphite Bio’s $238 million IPO was the biggest in biotech this week as it works toward human tests of a therapy that corrects the genetic mutation behind sickle cell disease. Elevation Oncology and Monte Rosa Therapeutics also made their stock market debuts, raising cash for clinical trials.
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Vertex to pay CRISPR Therapeutics $900M for bigger share of gene-editing alliance
Vertex Pharmaceuticals is amending its collaboration with CRISPR Therapeutics, paying $900 million now for chance to grab a bigger share of a gene-editing therapy’s profits later—if the treatment is approved. Clinical trials evaluating the CRISPR-based therapy are underway in sickle cell disease and beta thalassemia.
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Global Blood Therapeutics inks deal for two sickle cell disease drugs from Sanofi
In acquiring the two early-stage Sanofi drugs for sickle cell disease, Global Blood Therapeutics aims to complement Oxbryta, its FDA-approved treatment for the rare blood disorder. Meanwhile, Sanofi can turn its full sickle cell efforts on a gene-editing therapy partnered with Sangamo Therapeutics.
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Bluebird scrambles to find out if its gene therapy caused cancer in clinical trial
Cancer is a known risk of lentiviral-based gene therapies. Though bluebird says its gene therapy is engineered to avoid that risk, the company is suspending two clinical trials pending the outcome of its inquiry.
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Presentations at EHA highlight gene therapy approaches to beta-thalassemia, sickle cell disease
Phase III data on bluebird bio’s Zynteglo, as well as Vertex and CRISPR Therapeutics’ CTX001, showed patients achieving transfusion independence via two gene therapy approaches.
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FDA approves Global Blood Therapeutics’ drug targeting root cause of sickle cell disease
The agency gave accelerated approval to GBT’s Oxbryta (voxelotor) for sickle cell disease in patients aged 12 and older. The drug works by inhibiting sickle hemoglobin polymerization.