Beta thalassemia is blood disorder caused by sequent variants in the beta-globin (HBB) gene that threaten normal production of hemoglobin. Lentiviral Autologous Beta-Globin Gene Therapy (Proposed Name: Zynteglo, formerly LentiGlobin) aims to reduce or eliminate the need for regular blood transfusions in patients with transfusion-dependent beta-t…
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.
Evkeeza (evinacumab-dgnb) is an ANGPTL3 (angiopoietin-like 3) inhibitor indicated as an adjunct to other low-density lipoprotein cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients, aged 12 years and older, with homozygous familial hypercholesterolemia (HoFH).
The eXciteOSA Daytime Therapy Device (formerly known as Snoozeal) is a noninvasive electrical muscle stimulation device that delivers electrical pulses through a mouthpiece that sits around the tongue. The system consists of a mouthpiece, a rechargeable control unit that connects to the mouthpiece via a USB interface, and a mobile app that allows the user to control and track…
Narsoplimab is an investigational, intravenous monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2) for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA).
Lupkynis (voclosporin) is an oral calcineurin-inhibitor immunosuppressant indicated in combination with a background immunosuppressive therapy regimen for the treatment of adult patients with active lupus nephritis (LN).
The Flowsense device is a wireless, noninvasive thermal flow sensor in development for the detection of flow in implanted cerebrospinal fluid shunts in patients with hydrocephalus.
Inolimomab (proposed name, Leukotac) is an immunotherapy monoclonal antibody targeting the interleukin-2 receptor (IL-2) in development for the treatment of acute graft versus host disease (aGVHD).
Humacyl is an off-the-shelf bioengineered human acellular vessel proposed for use as a hemodialysis access graft in patients with end stage renal disease (ESRD). This technology uses a process to decellularize donated human vascular smooth muscle cells, while preserving extracellular matrix proteins. This results in a tubular scaffold that is acellular at the time of implanta…
The bridge-enhanced anterior cruciate ligament (ACL) repair (BEAR) implant is a proprietary bio-engineered bridging scaffold made from bovine collagen. It is intended to facilitate healing of a complete ACL tear. It was developed as an alternative to the current standard reconstruction treatment for torn ACLs.
Berotralstat is an oral plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.