Huntingtons Disease Pipeline Drugs Assessment: Clinical Trails Analysis, Player Profiles, Collaborations, Key Targets, Geographic Focus, and Data Publications, 2019 - 2030

Huntingtons diseaseis a fatal genetic disorder which causes progressive breakdown of nerve cells in brain.  It will deteriorate the physical and mental abilities of a person. HD is known as the quintessential family disease because every child of a parent with HD has a 50/50 chance of carrying the faulty gene.Huntington's disease usually causes movement, cognitive and psychiatric disorders.  General symptoms are involuntary jerkin, muscle stiffness or rigidity, impaired gait and posture, Lack of impulse control, Slowness in processing thoughts or ''finding'' words, Insomnia. Huntingtons Diseases is diagnosed by genetic testing, brain scans etc. Treatment of Huntingtons disease includes occupational therapy, Physical therapy, Psychotherapy, medications like anti-psychotic drugs, anti-depressants, Mood-stabilizing drugs

Key Developments: 

• In September 2017,Uniqure NVs AMT-130 was granted orphan drug designation by U.S.FDA for Huntingtons disease AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA which silences the defective huntingtin (HTT) gene. In March 2016, uniQure published preclinical results of AMT-130, indicating that a one-time administration can block the mutant HTT gene that causes Huntingtons disease
• In May 2017, Stealth BioTherapeutics Inc. (Stealth), initiated CHALLENGE-HD, a Phase 1/2 trial evaluating SBT-20 in patients with early stage Huntington's disease. SBT-20 is an investigational tetrapeptide aimed at improving mitochondrial function by restoring the physical and biochemical properties of dysfunctional mitochondria.