Delandistrogene moxeparvovec is a single-infusion gene therapy for the treatment of Duchenne muscular dystrophy.
Emerging Technology Reports
The Emerging Technology Report provides regular updates about emerging and potentially disruptive technologies that are likely to have a significant impact in healthcare. This “early warning” report monitors new health technologies from 6 months to 2 years within a regulatory submission and tracks those as they go through clinical trial development and the regulatory approval process.
For each emerging technology, our analysts describe the technology and intended use, its prospects relative to the regulatory process and FDA information, competing technologies, relevant clinical guidelines, high-level look at the evidence such as published abstracts in the medical literature and available meeting abstracts, and available clinical trials.
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.