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.
ThirtyFiveBio aims to treat gastrointestinal cancer by targeting a so-called orphan receptor called GPR35. The biotech startup is revealing its science at the American Association of Cancer Research annual meeting.
Hear executives from Quantum Health, Surescripts, EY, Clinical Architecture and Personify Health share their views on digital transformation in healthcare.
The FDA approved Airsupra, an asthma rescue inhaler that administers two different medications, one that opens up airways and another that treats inflammation. The inhaler stems from a partnership between AstraZeneca and Avillion.
AstraZeneca is jumping into the chase for cell therapies that address solid tumors by acquiring Neogene Therapeutics. The startup’s technology can be used to discover and develop novel targets that may be able to broaden the scope of cell therapies for cancer.
Ochre Bio’s Series A financing will support development of RNA therapies for chronic liver disease. Longer term, the company aims to develop therapies that regenerate organs in patients, potentially avoiding the need for organ transplants.
Neurocrine Biosciences is acquiring Diurnal Group, a U.K-based biotech that has commercialized two endocrine disorder drugs. Neurocrine’s top-selling product is a blockbuster drug for an involuntary muscle movement disorder.
Closing cancer health equity gaps require medical breakthroughs made possible by new funding approaches.
Pfizer and Merck have each won FDA approvals in the past year for new pneumococcal vaccines, but GSK aims to top both of them with its $2.1 billion acquisition of Affinivax. That biotech’s Phase 3-ready vaccine candidate addresses more bacterial strains than any pneumococcal vaccines currently available.
Sanofi’s new alliance with artificial intelligence biotech Exscientia spans up to 15 small molecule drugs in cancer and immunology. If drugs from the partnership reach the market, Exscientia could earn as much as $5.2 billion in milestone payments.
Sanofi is committing up to $60 million to Gyroscope Therapeutics, a clinical-stage biotech whose lead program could become the first gene therapy for a particular vision loss disorder. The deal also gives the pharma giant right of first refusal on that gene therapy in certain geographies.
Exscientia’s IPO and private placement raised $464 million to support its artificial intelligence-based approach to discovering and developing new drugs. In addition to partnerships with pharmaceutical companies, an alliance with the Bill & Melinda Gates Foundation is focused on developing antiviral drugs for future pandemics.
The future looks bright for the integrated specialty pharmacy model – for health systems, providers, and most importantly, for patients.
Roche is partnering with Adaptimmune Therapeutics to develop allogeneic cell therapies based on Adaptimmune’s technology. In addition to these “off-the-shelf” products, the partners will also develop a personalized allogeneic cell therapy designed specifically for a patient’s cancer.
An AstraZeneca antibody drug for Covid-19 has clinical data showing the drug reduced the risk of infection by 77%. With the results, the pharmaceutical giant pulls ahead of other companies developing injectable versions of antibody drugs for Covid-19 prevention.
Alexion Pharmaceuticals drug Ultomiris is already approved to treat a rare blood disorder, but the company hoped the antibody’s approach could also work in amyotrophic lateral sclerosis. Despite failing in ALS, Alexion, recently acquired by AstraZeneca, brings additional clinical-stage programs for rare diseases.
An AstraZeneca lupus drug has been awarded FDA approval for treating the most common form of the autoimmune disorder. The regulatory decision gives AstraZeneca a product that can compete head-to-head with GlaxoSmithKline’s lupus drug, Benlysta.
A NeRRE Therapeutics drug that failed a clinical trial for refractory or unexplained chronic cough is getting a second shot as a potential treatment for chronic cough in patients with idiopathic pulmonary fibrosis, a rare lung disorder. The biotech raised £20M to proceed with Phase 2 testing.