The first gene therapies for sickle cell disease are now approved: Casgevy from Vertex Pharmaceuticals Casgevy and Lyfgenia from Bluebird Bio. Despite the same-day approval, key differences give one of these therapies commercialization advantages over the other.
Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is now approved in the United Kingdom for treating the blood disorders sickle cell disease and beta thalassemia. It’s the first regulatory approval in the world for a CRISPR-based therapy.
Munck Wilson Mandala Partner Greg Howison shared his perspective on some of the legal ramifications around AI, IP, connected devices and the data they generate, in response to emailed questions.
Sickle cell disease is among the Beam Therapeutics programs spared, and here the company needs to show differentiation from genetic medicines under FDA review from Vertex Pharmaceuticals and Bluebird Bio. Meanwhile, Beam aims to find partners for its research in allogeneic cancer cell therapies and hepatitis B.
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.
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.
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.
At the Abarca Forward conference earlier this year, George Van Antwerp, managing director at Deloitte, discussed how social determinants of health and a personalized member experience can improve medication adherence and health outcomes.
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.
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.
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.
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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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.
CVS Caremark recently launched efforts to better prevent, detect and treat sickle cell disease, HIV and cardiovascular disease. CVS Caremark CMO Sree Chaguturu shared how the PBM launched this effort at the engagement track at HLTH, hosted by MedCity News
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.
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.
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.