Exagamglogene autotemcel (Casgevy; Vertex Pharmaceuticals and CRISPR Therapeutics) is an autologous genome-edited hematopoietic stem cell-based gene therapy indicated for the treatment of patients aged 12 years and older with transfusion-dependent beta thalassemia.
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.
Automated Low-Flow Ascites Pump (alfapump; Sequana Medical NV) for Refractory Ascites
The automated low-flow ascites pump (alfapump; Sequana Medical NV) is an implantable wireless battery-powered pump for treatment of recurrent or refractory ascites due to liver cirrhosis.
Edison System (HistoSonics) for Histotripsy of Renal Tumors
The Edison System (HistoSonics) is a nonthermal focused ultrasound system with real-time image guidance under investigation for the noninvasive mechanical destruction of stage I primary solid renal tumors via histotripsy.
Eplontersen (Wainua; Ionis Pharmaceuticals Inc./AstraZeneca) for Hereditary Transthyretin-mediated Amyloid Polyneuropathy
Eplontersen (Wainua; Ionis Pharmaceuticals Inc./AstraZeneca) is an injectable transthyretin-directed antisense oligonucleotide indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Filsuvez (Birch Triterpenes) Topical Gel (Chiesi Global Rare Diseases) for Epidermolysis Bullosa
Filsuvez (birch triterpenes) topical gel (formerly Oleogel-S10; Chiesi Global Rare Diseases) is indicated for the treatment of wounds associated with dystrophic and junctional epidermolysis bullosa in adult and pediatric patients 6 months of age and older.
Globe Pulsed Field Mapping and Ablation System (Kardium Inc.) for Atrial Fibrillation
The Globe Pulsed Field Mapping and Ablation System is an investigational pulsed-field ablation system for treatment of atrial fibrillation.
PulseSelect Pulsed Field Ablation (PFA) System (Medtronic Inc.) for Atrial Fibrillation
The PulseSelect system (Medtronic Inc.) is a pulsed field ablation (PFA) system indicated for cardiac electrophysiological mapping (stimulation and recording) and for treatment of drug refractory, recurrent, symptomatic paroxysmal atrial fibrillation or persistent atrial fibrillation (episode duration less than 1 year).
Lovotibeglogene Autotemcel (Lyfgenia; Bluebird Bio) for Sickle Cell Disease
Lovotibeglogene autotemcel (Lyfgenia; Bluebird Bio Inc.) is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events.
Exagamglogene Autotemcel (Casgevy; Vertex/CRISPR) for Sickle Cell Disease
Exagamglogene autotemcel (Casgevy; Vertex Pharmaceuticals and CRISPR Therapeutics) is an autologous, genome-edited, hematopoietic stem cell-based gene therapy indicated for the treatment of sickle cell disease (SCD) in patients aged 12 years and older with recurrent vaso-occlusive crises (VOCs).
Prademagene Zamikeracel (Abeona Therapeutics Inc.) for Recessive Dystrophic Epidermolysis Bullosa
Prademagene zamikeracel (pz-cel; formerly EB-101) (Abeona Therapeutics Inc.) is a gene-corrected epidermal sheet under investigation for the treatment recessive dystrophic epidermolysis bullosa.