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.
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.
CYB003 (Cybin Inc.) Deuterated Psilocybin Analogue for Major Depressive Disorder
CYB003 (Cybin Inc.) is an oral deuterated psilocybin analogue proposed for use in conjunction with supportive psychotherapy for treatment of patients with moderate to severe major depressive disorder who have had an inadequate response to an antidepressant medication.
FSYX Ocular Pressure Adjusting Pump (Balance Ophthalmics Inc.) for Open-Angle Glaucoma
The FSYX Ocular Pressure Adjusting Pump (Balance Ophthalmics Inc.) is an investigational negative pressure eye goggle intended for adjunctive therapy to reduce nocturnal intraocular pressure in patients with open-angle glaucoma and normal daytime intraocular pressure.
Fidanacogene Elaparvovec-dzkt (Beqvez; Pfizer Inc.) for Hemophilia B
Fidanacogene elaparvovec-dzkt (Beqvez; Pfizer Inc.) is a single-infusion gene therapy for the treatment of adults with moderate to severe hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated vi…
Ventura Interatrial Shunt (V-Wave Ltd.) for Heart Failure
The Ventura Interatrial Shunt (V-Wave Ltd.) is an investigational transcatheter interatrial shunt proposed for treatment of chronic heart failure.
Rusfertide (Protagonist Therapeutics Inc./Takeda Pharmaceutical Co. Ltd.) for Polycythemia Vera
Rusfertide (formerly PTG-300; Protagonist Therapeutics Inc./Takeda Pharmaceutical Co. Ltd.) is a subcutaneously injected hepcidin mimetic for the treatment of erythrocytosis associated with polycythemia vera.
Varipulse Pulsed Field Ablation (Biosense Webster Inc.) for Atrial Fibrillation
Varipulse is an investigational pulsed-field ablation system in development for treatment of paroxysmal atrial fibrillation.
Letetresgene Autoleucel (Adaptimmune Therapeutics PLC) for Advanced Synovial Sarcoma
Letetresgene autoleucel is an investigational autologous engineered T-cell therapy for the treatment of HLA-A*02 positive patients with advanced synovial sarcoma expressing the NY-ESO-1 antigen.
ReCET (Endogenex Inc.) Duodenal Mucosal Regeneration for Type 2 Diabetes
ReCET (Endogenex Inc.) is an investigational endoscopic procedure that uses pulsed electric fields to promote duodenal mucosal regeneration for treatment of type 2 diabetes that is poorly controlled with oral medications and/or insulin.
Relyvrio (Sodium Phenylbutyrate/Taurursodiol; Amylyx Pharmaceuticals Inc.) for Amyotrophic Lateral Sclerosis (ALS)
Relyvrio (sodium phenylbutyrate/taurursodiol, formerly AMX0035; Amylyx Pharmaceuticals Inc.) is an oral fixed-dose combination drug indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults. As of April 4, 2024, Relyvrio has been withdrawn from the market.