Vertex Pharmaceuticals gains a Phase 3-ready Alpine Immune Sciences drug with potential applications in a wide range of immunological disorders. The lead indication for the Alpine drug, povetacicept, is the chronic kidney disease IgA nephropathy.
The FDA approved Vertex Pharmaceuticals’ gene therapy Casgevy for treating beta thalassemia, an inherited blood disease that leads to low levels of functioning hemoglobin. Last month, the one-time treatment won its first FDA nod for treating sickle cell disease.
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.
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.
Vertex Pharmaceuticals aims to use CRISPR Therapeutics’ gene-editing technology to develop type 1 diabetes cell therapies that don’t prompt the immune system to reject them. This approach could compete with a Sana Biotechnology program expected to reach its first test in humans this year.
Vertex Pharmaceuticals already has a cell therapy candidate for type 1 diabetes but the company says acquiring rival ViaCyte will provide it with complementary assets and technologies. The two companies already share a partner in common: both have therapeutic candidates that use the gene-editing capabilities of CRISPR Therapeutics.
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.
Vertex Pharmaceuticals is collaborating with Obsidian Therapeutics to develop up to five new gene editing therapies. Obsidian’s technology precisely controls protein expression, a capability the partners plan to apply to several undisclosed serious diseases.
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.
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.
Analyst reports over the past few weeks have looked into what effects the Covid-19 pandemic could have on drugmakers' sales, examining multiple factors. But virtual launches and telemedicine could mitigate some market challenges.
We will highlight Build My Health's revenue practice management tools, which could help physician practices add up to $250,000 to their practices.
The company, part of BioSig Technologies, aims to run a Phase II study of the drug, merimepodib, which had previously failed in a Phase IIb HCV study run by Vertex Pharmaceuticals. The company hopes the drug's mechanism could prove effective against SARS-CoV-2.
A recruiter in the industry said trial-monitoring staff report having their hours cut, while CRAs are grounded, and others have reported being laid off on social media. Several drugmakers have wound down clinical trial operations as the pandemic has overwhelmed a growing number of hospitals.
The biotech company said it is well-stocked to supply its approved drugs for cystic fibrosis to patients. Meanwhile, it plans to implement virtual visits and ship drugs to participants in its ongoing trials, while pausing enrollment and delaying initiation in some.
Shares of the company rose 9 percent on the news that the FDA had approved Trikafta. The drug will be an option for patients using the company's other approved CF drugs.
The Boston-based company said it would acquire Semma Therapeutics, which is developing a stem cell-derived therapy and device for the disease. Preclinical data were announced in July, and clinical trials are planned for 2020.