The Batten Disease drugs in development market research report provides comprehensive information on the therapeutics under development for Batten Disease, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. GlobalData’s report assesses key aspects of the companies and drugs in development for Batten Disease. Buy the report here.

The report also covers the descriptive pharmacological action of the therapeutics and the latest news and press releases. Additionally, the report provides an overview of the key players involved in therapeutic development for Batten Disease and features dormant and discontinued products.

GlobalData tracks 29 drugs in development for Batten Disease by 20 companies/universities/institutes. The top development phase for Batten Disease is preclinical with 16 drugs in that stage. The Batten Disease pipeline has 21 drugs in development by companies and eight by universities/ institutes. Some of the companies in the Batten Disease pipeline products market are: Recursion Pharmaceuticals, Polaryx Therapeutics and Neurogene.

The key targets in the Batten Disease pipeline products market include Palmitoyl Protein Thioesterase 1 (Palmitoyl Protein Hydrolase 1 or CLN1 or PPT1 or EC, Peroxisome Proliferator Activated Receptor Alpha (Nuclear Receptor Subfamily 1 Group C Member 1 or NR1C1 or PPARA), and Tripeptidyl Peptidase 1 (Tripeptidyl Aminopeptidase or Lysosomal Pepstatin Insensitive Protease or Cell Growth Inhibiting Gene 1 Protein or TPP1 or EC

The key mechanisms of action in the Batten Disease pipeline product include Peroxisome Proliferator Activated Receptor Alpha (Nuclear Receptor Subfamily 1 Group C Member 1 or NR1C1 or PPARA) Agonist with three drugs in IND/CTA Filed. The Batten Disease pipeline products include seven routes of administration with the top ROA being Oral and six key molecule types in the Batten Disease pipeline products market including Small Molecule, and Gene Therapy.

Batten Disease overview

Batten disease, a rare genetic disorder, belongs to a group of progressive degenerative neurometabolic disorders known as the neuronal ceroid lipofuscinoses. Symptoms of Batten disease usually become apparent between five and 15 years of age. Symptoms include vision loss, lack of muscle coordination, mental retardation or decreasing mental function, emotional disturbances, seizures, muscle spasms, deterioration of muscle tone, and movement problems.

For a complete picture of Batten Disease’s pipeline drug market, buy the report here.

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s pipeline drugs offers detailed profiles of pharmaceutical drugs in all stages of pre-clinical and clinical development, from discovery through to pre-registration. Coverage is limited to novel human medicinal drugs and biosimilars seeking market approval proprietary and is one of two primary repositories of pharmaceutical drug information offered by GlobalData through its Pharmaceutical Intelligence Center.