(PharmaNewsWire.Com, July 06, 2017 ) Market Research Hub (MRH) has recently publicized a new study to its vast repository, which is titled as “Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) - Pipeline Review, H1 2017”. This research study outlays comprehensive information on the Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type; that are being developed by companies and universities. Presently, the molecules developed by companies in Preclinical and Discovery stages are 4 and 1 respectively. Additionally, the report covers products from therapy areas metabolic disorders which include indications of Phenylketonuria (PKU).
Initially, the report starts by presenting the overview of Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) along with its therapeutic development on products under development and therapeutic assessment. Phenylalanine 4 hydroxylase is an enzyme that catalyzes the hydroxylation of the aromatic side-chain of phenylalanine to generate tyrosine. The tyrosine is generated by the action of phenylalanine hydroxylase and it is essential for the synthesis of various neurotransmitters which act on the nervous system and also administer key functions like respiration and heart rate. This enzyme works with a molecule called tetrahydrobiopterin (BH4) to carry out this chemical reaction. Tyrosine is also used in making several types of hormones and certain chemicals that transfer signals to the brain and a pigment called melanin, which gives hair and skin their color.
It has been analyzed that, a deficiency in PAH can lead to phenylketonuria (PKU), which is an autosomal recessive genetic disorder characterized by an inability to metabolize phenylalanine, resulting in elevated Phe levels in the bloodstream that can lead to mental retardation, if left untreated. Due to this the therapeutic assessment is vital, which covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages.
Furthermore, the study offers detailed drug profiles for the pipeline products which embraces, expressive MoA, R&D brief, product description, licensing and collaboration details & other major developmental activities. It primarily includes CDX-6114, SYN-200, DTX-501, Recombinant Phenylalanine-4-Hydroxylase Replacement for Phenylketonuria and Small Molecules to Activate Phenylalanine Hydroxylase for Phenylketonuria.
Finally, it also reviews also reviews key players involved in Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) targeted therapeutics development with respective active and dormant or discontinued projects. At present, leading players are Codexis Inc, Dimension Therapeutics Inc, Synthetic Biologics Inc and Others.
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