Delandistrogene moxeparvovec is a single-infusion gene therapy for the treatment of Duchenne muscular dystrophy.
Delandistrogene Moxeparvovec (Sarepta Therapeutics Inc.) for Duchenne Muscular Dystrophy
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Horizon Scanning
It is a challenge for health plans and clinicians to know what emerging technologies are in the preapproval phase and what their clinical, financial, and risk impacts might be. This is where we come in.The Hayes Horizon Scanning Solution identifies innovative and potentially disruptive health technologies that are in various stages of the regulatory approval process. Our Research Analysts continually investigate new technologies that are on the horizon or in the early stages of clinical investigation, as well as the testing of existing technologies that may be seeking approval for new clinical applications. Hayes Horizon Scanning helps you track emerging technologies before you need to make decisions. Accessed through the Hayes Knowledge Center, Hayes Horizon Scanning addresses the following fundamental questions to help our clients forecast emerging technologies:
- Is this emerging technology new or replacing an existing technology?
- Where is this technology in the FDA regulatory process?
- When will this technology be commercially available in the market?
- What is the projected use, cost, and financial impact of this technology?
Exagamglogene Autotemcel (Vertex/CRISPR) for Transfusion-dependent Beta Thalassemia
Exagamglogene autotemcel is an investigational gene therapy delivered by autologous hematopoietic stem cell transplantation for the treatment of transfusion-dependent beta thalassemia.
Exagamglogene Autotemcel (Vertex/CRISPR) for Sickle Cell Disease
Exagamglogene autotemcel (Vertex Pharmaceuticals and CRISPR Therapeutics) is an investigational gene therapy delivered by autologous hematopoietic stem cell transplantation for the treatment of sickle cell disease.
Respiratory Syncytial Virus Vaccine, Adjuvanted (Arexvy; GSK plc) for Prevention of Lower Respiratory Tract Disease due to Respiratory Syncytial Virus in Older Adults
Arexvy (respiratory syncytial virus vaccine, adjuvanted; GSK plc) is a vaccine indicated for active immunization for the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus in individuals 60 years of age and older.
Atrial Flow Regulator (Occlutech) for Heart Failure
The Atrial Flow Regulator (Occlutech) is a transcatheter interatrial shunt under investigation for the treatment of heart failure with preserved or reduced ejection fraction.
Vowst (Fecal Microbiota Spores, Live-brpk; Seres Therapeutics Inc.) for Prevention of Recurrent Clostridioides Difficile Infection
Vowst (fecal microbiota spores, live-brpk; Seres Therapeutics Inc.) is an oral capsule containing fecal microbiota spores indicated to prevent the recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older following antibacterial treatment for recurrent CDI (rCDI).
Pentosan Polysulfate Sodium (Paradigm Biopharmaceuticals Ltd.) for Knee Osteoarthritis
Pentosan polysulfate sodium (proposed name, Zilosul) is a subcutaneously injected semisynthetic glucosaminoglycan under investigation for treatment of knee osteoarthritis.
Tofersen (Qalsody; Biogen) for Amyotrophic Lateral Sclerosis
Tofersen (Qalsody; Biogen) is an intrathecally administered antisense oligonucleotide indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS).
Lovotibeglogene Autotemcel (Bluebird Bio) for Sickle Cell Disease
Lovotibeglogene autotemcel is an investigational gene therapy delivered by autologous hematopoietic stem cell transplantation for the treatment of sickle cell disease.
Lecanemab (Leqembi; Esai Co. Ltd. and Biogen) for Alzheimer Disease
Lecanemab (Leqembi) is an intravenously administered anti-amyloid beta (Aβ) protofibril antibody indicated for the treatment of Alzheimer disease.