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SIG-001

SIG-001 is a cultured human retinal pigment epithelial cell line (ARPE-19) genetically modified with a non-viral vector to express B domain deleted human factor VIII protein, encapsulated within two-layer

Microcrystalline cellulose

A polysaccharide with glucose units linked as in CELLOBIOSE. It is the chief constituent of plant fibers, cotton being the purest natural form of the substance. … As a raw material, it forms the basis for many derivatives used in chromatography, ion exchange materials, explosives manufacturing, and pharmaceutical preparations.

RG-7795

Doxecitine

It is an essential component of the deoxyribonucleotide pool required for DNA synthesis and repair. … Doxecitine is a synthetic form of the naturally occurring pyrimidine deoxyribonucleoside deoxycytidine.

Lasofoxifene

It gained approval by European Commission in March 2009. … It was initially developed as Oporia by Pfizer as a treatment for postmenopausal osteoporosis and vaginal atrophy, in which were both rejected for approval by FDA.

Plasma protein fraction (human)

[L2262] The proteins in PPFh are stabilized with sodium caprylate and acetyltryptophan and it contains some electrolytes such as sodium, potassium and chloride.[L2261] … It is also stated that no more than 1% of the total protein should be gamma globulin.[L2260] PPFh is a sterile, frozen solution of solvent/detergent treated human plasma.

Buformin

It was withdrawn from the market in most countries due to a high risk of causing lactic acidosis. … Buformin is an anti-diabetic drug of the biguanide class, chemically related to metformin and phenformin.

Adagrasib

[A254052] In a phase I/IB clinical study that included patients with KRAS^G12C-mutated advanced solid tumors (NCT03785249), adagrasib exhibited anti-tumor activity. … KRAS mutations are highly common in cancer and account for approximately 85% of all RAS family mutations.

Plecanatide

It should not be used in children less than six years of age, and should be avoided in patients six years to 18 years of age … Plecanatide is a drug approved in January 2017 by the FDA for the treatment of chronic idiopathic constipation (CIC).

Beremagene geperpavec

[L46916] DEB is caused by mutations in the _COL7A1_ gene that encodes collagen VII (COL7), a major component of the anchoring fibrils for dermal–epidermal cohesion. … Developed by Krystal Biotech, it was first approved by the FDA on May 19, 2023, for the treatment of wounds associated with dystrophic epidermolysis bullosa (DEB).