Elivaldogene autotemcel (eli-cel) is a lentiviral-based gene therapy for treatment cerebral adrenoleukodystrophy (CALD).
Elivaldogene Autotemcel for Cerebral Adrenoleukodystrophy
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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.
TissueGene-C for Knee Osteoarthritis
TissueGene-C is an injectable solution of normal and genetically modified allogeneic human chondrocytes in development for the treatment of knee osteoarthritis.
Betibeglogene Autotemcel (Proposed Name: Zynteglo) for Beta Thalassemia
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…
Evkeeza (Evinacumab-dgnb) for Homozygous Familial Hypercholesterolemia
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).
Paradise Renal Denervation System for Hypertension
The Paradise Renal Denervation System 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 proposed for the treatment of hypertension.
eXciteOSA Device for Mild Obstructive Sleep Apnea
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…
Breyanzi (Lisocabtagene Maraleucel) for Large B-Cell Lymphoma
Breyanzi (lisocabtagene maraleucel [liso-cel]) is an autologous chimeric antigen receptor (CAR) T-cell therapy, a novel type of immunotherapy in which a patient’s own immune cells are genetically re-programmed to target and attack cancer cells. It is indicated for the treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphoma after 2 or more lines of …
Aducanumab for Alzheimer Disease
Aducanumab is an investigational human monoclonal anti-amyloid beta antibody in development for the treatment of Alzheimer disease.
Narsoplimab for Stem Cell Transplant-Associated Thrombotic Microangiopathy
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) for Lupus Nephritis
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).