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).
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?
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.
Aortix Percutaneous Mechanical Circulatory Support Device (Procyrion Inc.)
Aortix is an investigational percutaneous mechanical circulatory support device in development for the treatment of chronic heart failure patients on medical management who have been hospitalized for acute decompensated heart failure with worsening renal function.
Sonpiretigene Isteparvovec (Nanoscope Therapeutics Inc.) for Retinitis Pigmentosa
Sonpiretigene isteparvovec is an investigational gene therapy for retinitis pigmentosa, administered as an intravitreal injection.
ADAMTS13, Recombinant-krhn (Adzynma; Takeda Pharmaceutical Co. Ltd.) for Congenital Thrombotic Thrombocytopenic Purpura
Adzynma (Takeda Pharmaceutical Co. Ltd.) is a human recombinant “A disintegrin and metalloproteinase with thrombospondin motifs 13” (rADAMTS13) indicated for prophylactic or on demand enzyme replacement therapy in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura.
Rebonuputemcel Injectable Discogenic Cell Therapy (IDCT; DiscGenics Inc.) for Degenerative Disc Disease
Rebonuputemcel Injectable Discogenic Cell Therapy (IDCT; DiscGenics Inc.) is an allogeneic progenitor cell therapy for the treatment of symptomatic early-to-moderate lumbar degenerative disc disease at a single level from L3 to S1.
Paradise Ultrasound Renal Denervation System (ReCor Medical Inc.) for Resistant Hypertension
The Paradise Ultrasound Renal Denervation (uRDN) System (ReCor Medical Inc.) uses ultrasound energy delivered via a balloon-cooled catheter to perform targeted circumferential denervation to the renal afferent and efferent sympathetic nerves. The system is indicated to reduce blood pressure as an adjunctive treatment in hypertension patients in whom lifestyle modifications an…
Nuvella System (Neuspera Medical Inc.) for Urinary Urge Incontinence
The Nuvella System (Neuspera Medical Inc.) is an investigational integrated sacral nerve stimulator system for treatment of urinary urge incontinence.
Aurora EV-ICD System (Medtronic Inc.) for Ventricular Tachyarrhythmias
The Aurora EV-ICD System (Medtronic Inc.) is an extravascular implantable cardioverter-defibrillator (ICD) indicated for the automated treatment of patients who have experienced, or are at significant risk of developing, life-threatening ventricular tachyarrhythmias through the delivery of antitachycardia pacing, cardioversion, and defibrillation therapies.
WiSE Cardiac Resynchronization Therapy System (EBR Systems Inc.)
The WiSE CRT system is a wireless left ventricular pacing system that works with an implanted right ventricular pacemaker and/or defibrillator for patients in whom cardiac resynchronization therapy (CRT) is indicated.