Related Research Articles
Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells.
A cancer vaccine, or oncovaccine, is a vaccine that either treats existing cancer or prevents development of cancer. Vaccines that treat existing cancer are known as therapeutic cancer vaccines or tumor antigen vaccines. Some of the vaccines are "autologous", being prepared from samples taken from the patient, and are specific to that patient.
Glioblastoma, previously known as glioblastoma multiforme (GBM), is the most aggressive and most common type of cancer that originates in the brain, and has a very poor prognosis for survival. Initial signs and symptoms of glioblastoma are nonspecific. They may include headaches, personality changes, nausea, and symptoms similar to those of a stroke. Symptoms often worsen rapidly and may progress to unconsciousness.
Metachromatic leukodystrophy (MLD) is a lysosomal storage disease which is commonly listed in the family of leukodystrophies as well as among the sphingolipidoses as it affects the metabolism of sphingolipids. Leukodystrophies affect the growth and/or development of myelin, the fatty covering which acts as an insulator around nerve fibers throughout the central and peripheral nervous systems. MLD involves cerebroside sulfate accumulation. Metachromatic leukodystrophy, like most enzyme deficiencies, has an autosomal recessive inheritance pattern.
Choroideremia is a rare, X-linked recessive form of hereditary retinal degeneration that affects roughly 1 in 50,000 males. The disease causes a gradual loss of vision, starting with childhood night blindness, followed by peripheral vision loss and progressing to loss of central vision later in life. Progression continues throughout the individual's life, but both the rate of change and the degree of visual loss are variable among those affected, even within the same family.
Monoclonal antibodies (mAbs) have varied therapeutic uses. It is possible to create a mAb that binds specifically to almost any extracellular target, such as cell surface proteins and cytokines. They can be used to render their target ineffective, to induce a specific cell signal, to cause the immune system to attack specific cells, or to bring a drug to a specific cell type.
Ramucirumab is a fully human monoclonal antibody (IgG1) developed for the treatment of solid tumors. This drug was developed by ImClone Systems Inc. It was isolated from a native phage display library from Dyax.
Nelotanserin is a drug developed by Arena Pharmaceuticals which acts as an inverse agonist on the serotonin receptor subtype 5-HT2A and was under development for the treatment of insomnia. It was shown to be effective and well tolerated in clinical trials, but development was halted in December 2008 because the substance did not meet the trial's effectiveness endpoints. Research continues on newer analogues which may potentially be more successful.
Cellectis is a French biopharmaceutical company. It develops genome-edited chimeric antigen receptor T-cell technologies for cancer immunotherapy. It has offices in Paris, New York City, and Raleigh, North Carolina.
Gene therapy for osteoarthritis is the application of gene therapy to treat osteoarthritis (OA). Unlike pharmacological treatments which are administered locally or systemically as a series of interventions, gene therapy aims to establish sustained therapeutic effect after a single, local injection.
Many variants of herpes simplex virus have been considered for viral therapy of cancer; the early development of these was thoroughly reviewed in the journal Cancer Gene Therapy in 2002. This page describes the most notable variants—those tested in clinical trials: G207, HSV1716, NV1020 and Talimogene laherparepvec. These attenuated versions are constructed by deleting viral genes required for infecting or replicating inside normal cells but not cancer cells, such as ICP34.5, ICP6/UL39, and ICP47.
Sio Gene Therapies, Inc. was a clinical-stage pharmaceutical company that developed gene therapies to treat neurological disorders. The company was headquartered in New York City and was incorporated in Basel, Switzerland. The company was founded by former hedge fund analyst and 2024 Republican Party presidential primary candidate Vivek Ramaswamy in October 2014 as a wholly owned subsidiary of Roivant Sciences, which was itself founded in May 2014.
Resamirigene bilparvovec is an experimental gene therapy medication studied as a treatment for X-linked myotubular myopathy (XLMTM), a severe and usually fatal genetic disorder affecting mainly male infants and caused by a mutation in the MTM1 gene. The drug consists of a MTM1 transgene encapsulated in a viral vector from the adeno-associated virus class (AAV8) and is administered to affected children as an intravenous infusion.
Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B. Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX. The gene is expressed in the liver to produce Factor IX protein, to increase blood levels of Factor IX and thereby limit bleeding episodes. Hemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting Factor IX, a protein needed to produce blood clots to stop bleeding.
DSP-0038 is a serotonin receptor modulator that acts as a 5-HT2A receptor antagonist and 5-HT1A receptor agonist. It is under development for psychosis related to Alzheimer's disease. The drug was developed using artificial intelligence that enabled it to proceed from discovery to Phase I trial in one year, compared to an industry average of 4–6 years.
EXS4318 is a selective PKC theta inhibitor that is potentially first-in-class medication. Developed by Exscientia, it was licensed to Bristol Myers Squibb. In 2023, it entered clinical trials in humans.
VERVE-101 and VERVE-102 are an experimental gene therapy developed by Verve Therapeutics that targets the PCSK9 gene and is intended to reduce blood cholesterol levels. It works on the same protein as the cholesterol-lowering drugs known as PCSK9 inhibitors but, unlike them, is permanent. It works via base editing, a form of CRISPR gene editing. It is one of the first gene therapies that could be beneficial to a wider segment of the population, in contrast to earlier gene therapies that were developed to treat a rare genetic disorder. Both treatments use the same RNA gene editing technology, but they use a different lipid nanoparticle delivery vehicle.
DB-OTO is an experimental gene therapy for otoferlin-related hearing loss developed by Regeneron Pharmaceuticals. It is delivered via adeno-associated virus.
FBX-101 is an experimental gene therapy delivered via adeno-associated virus rh10 vector to the GALC gene. It is developed by Forge Biologics to treat Krabbe disease.
SPK-3006 is an experimental gene therapy developed for Pompe disease by Spark Therapeutics. It is delivered via adeno-associated virus and is intended to increase alpha-glucosidase production in the liver.
References
- ↑ Philippidis, Alex (1 January 2022). "StockWatch: Astellas Hones Gene Therapy Focus with Taysha Stake: Tricida shares crater after failed Phase III trial; Vaxcyte bolsters challenge to Pfizer with clinical success; ESSA triples after acing prostate cancer study". GEN Edge. 4 (1): 809–815. doi:10.1089/genedge.4.1.137. S2CID 253331384.
- ↑ Philippidis, Alex (1 January 2023). "StockWatch: Seven Analysts Cut Price Target for Taysha on FDA Advice, Job Cuts: Gene therapy developer says call for randomized placebo-controlled trial is 'unfeasible'; Evelo falls on clinical miss; HillStream jumps on Dana-Farber deal". GEN Edge. 5 (1): 128–132. doi:10.1089/genedge.5.1.28. S2CID 257006849.
- ↑ Philippidis, Alex (1 October 2020). "Sarepta Duchenne Muscular Dystrophy Candidate Faces U.S. Food and Drug Administration Assay Request, Patent Suit". Human Gene Therapy. 31 (19–20): 1031–1033. doi:10.1089/hum.2020.29137.bfs. PMID 33074027. S2CID 224780854.
- ↑ Philippidis, Alex (1 January 2023). "Taysha Faces Stock Delisting, Scraps $75M Durham Manufacturing Facility: Company aims to preserve capital while anticipating second-half clinical milestones for key programs, with Q1 results expected Thursday". GEN Edge. 5 (1): 362–367. doi:10.1089/genedge.5.1.70. S2CID 258737047.
- ↑ Philippidis, Alex (1 January 2023). "StockWatch: Illumina Results Leave Investors, Analysts Anxious: Taysha zooms on $150M PIPE financing; Alaunos plunges on job cuts; Design nosedives on patient 'reactions'; Phase IIb trial failure sinks Galecto; Zynerba quadruples on up-to-$200M buyout by Harmony". GEN Edge. 5 (1): 549–556. doi:10.1089/genedge.5.1.106. S2CID 265960414.