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NTLA-2002

NTLA-2002 is a lipid nanoparticle encapsulating single guide RNA (G012267) targeting the human KLKB1 gene and messenger RNA (mRNA000042) encoding Cas9. … It is an investigational therapy designed to knock out the target gene kallikrein B1 (KLKB1) to reduce plasma kallikrein activity, thereby preventing hereditary angioedema attacks.[L45738]

mRNA-3927

mRNA-3927 is an investigational mRNA therapy that consists of two mRNAs that encode for the alpha and beta subunits of the mitochondrial enzyme propionyl-CoA carboxylase (PCC) encapsulated within a lipid

Acetic acid C-11

Acetic acid C-11 is under investigation in clinical trial NCT01953965 (Look at Way the Heart Functions in People With Pulmonary Hypertension (PH) Who Have Near Normal Right Ventricle (RV) Function and

Mexazolam

[A184910] The use of benzodiazepines in the treatment of anxiety is generally a second line measure.[A184910] … [A184910] Mexazolam's structure is similar to that of other benzodiazepines such as [oxazolam] and [cloxazolam].

3-amino-5-phenylpentane

These are compounds containing a phenylpropylamine moiety, which consists of a phenyl group substituted at the third carbon by a propan-1-amine. 3-amino-5-phenylpentane targets the proteins cathepsin K … 3-amino-5-phenylpentane is a solid. This compound belongs to the phenylpropylamines.

PR-104

GlycoPEG-GCSF

GlycoPEG-GCSF is a long-acting GlycoPEGylated granulocyte colony stimulating factor for the treatment of chemotherapy-induced neutropenia.

LetibotulinumtoxinA

[L42330] It is a 900 kDa multimeric complex comprising a 150 kDa toxin, a 130 kDa non-toxic non-haemagglutinating protein, and various other haemagglutinins. … [A249920] LetibotulinumtoxinA is a type A botulinum neurotoxin produced from fermentation of _Clostridium botulinum_ strain CBFC26.

Vedolizumab

Vedolizumab is a recombinant humanized IgG1 monoclonal antibody directed against the human lymphocyte α4β7 integrin, a key mediator of gastrointestinal inflammation implicated in diseases like ulcerative … [A261576] Vedolizumab is administered by IV infusion over a period of 30 minutes; after the first dose, it is given again at two and six weeks and then every 8 weeks thereafter.

Alfaxalone

Alfaxalone, also known as alphaxalone or alphaxolone, is a neuroactive steroid and general anaesthetic.