FarmaKology-Scientists discover how caffeine protects against cardiovascular disease
Prime Medicine, Inc.
Prime Medicine was founded to bring the promise of gene editing to patients. We use Prime Editing, a next-generation technology that can “search and replace” to restore normal genetic function almost anywhere in the genome. Prime Editing searches to find the precise place in the genome to edit and replaces the segment of faulty DNA with a correct copy of DNA. A single Prime Editor can correct the individual mutations found across patients, meaning Prime Editing can potentially address more than 90 percent of known disease-causing genetic mutations.We envision a world where this technology can cure, halt, and ultimately prevent genetic diseases, providing lifelong benefit to patients. Prime Medicine offers the opportunity to shape the future of gene editing and transform how medicines are used to treat disease. Realizing the promise of Prime Editing requires a talented team with diversity of viewpoints and expertise. We’re building a group of dedicated, scientifically curious individuals who are passionate about helping patients live longer, healthier lives.
AstraZeneca’s Saphnelo (anifrolumab) has been approved in the European Union as an add-on therapy for the treatment of adult patients with moderate to severe, active autoantibody-positive systemic lupus erythematosus (SLE), despite receiving standard therapy. Saphnelo is the first biologic for SLE approved in Europe with an indication that is not restricted to patients with a high degree of disease activity. SLE is a serious and complex autoimmune condition that can affect any organ, and patients often experience inadequate disease control, long-term organ damage and poor health-related quality of life.1-3 There are approximately 250,000 people with SLE in Europe, and most are women who are diagnosed between the ages of 15 and 45.
Hillstream BioPharma Receives Orphan Drug Designation for HSB-1216 for the Treatment of Uveal Melanoma
Hillstream BioPharma Inc., a biotechnology company developing novel therapeutic candidates targeting ferroptosis, an emerging new anti-cancer mechanism resulting in iron mediated cell death for drug resistant and devastating cancers, announced the FDA granted Orphan Drug Designation to HSB-1216 for Uveal Melanoma (UM). Hillstream continues to build an attractive oncology pipeline and expand its oncology pipeline for devastating diseases with this significant milestone achievement for HSB-1216.
European Commission Approves Oxbryta® (voxelotor) for the Treatment of Hemolytic Anemia in Patients with Sickle Cell Disease Age 12 Years and Older
Global Blood Therapeutics, Inc. today announced the European Commission has granted Marketing Authorization for Oxbryta® (voxelotor) for the treatment of hemolytic anemia due to sickle cell disease in adult and pediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide (hydroxyurea). Oxbryta, a once-daily, oral treatment, is the first medicine approved in Europe that directly inhibits sickle hemoglobin (HbS) polymerization, the molecular basis of sickling and destruction of red blood cells in SCD.
BioNTech introduces first modular mRNA manufacturing facility to promote scalable vaccine production in Africa
BioNTech SE has taken a next step to improve vaccine supply in Africa. The company has introduced its approach to establishing scalable vaccine production by developing and delivering turnkey mRNA manufacturing facilities based on a container solution. At a high-level meeting at BioNTech’s new manufacturing facility in Marburg and at the invitation of kENUP Foundation, the company presented the container solution named “BioNTainer” to key partners.