Angelman syndrome Pipeline: Unmet needs call for a robust pipeline

The necessity of a robust Angelman syndrome pipeline is indispensable at the current moment because of a shortage of any approved therapy option available in the market.

The Angelman Syndrome current treatment landscape consists of symptomatic therapy options worth USD 330.2 million in 2017. 

Treatment entirely focuses on the management of symptoms and supporting the health and well-being of the affected child or adult. 

The management focusses on the therapies for the physical and neurological problems emphasising on the specific cognitive profiles and behavioural features of the condition. 

The Angelman syndrome treatment includes Speech therapy, Behaviour modification (to address hyperactivity, aggressive behaviour, sleep disorders, self-injury), Communication therapy (to assess speech development), Occupational therapy (to teach self-care), Physical therapy (to achieve functional mobility), Special education, Social skills training and Anti-epileptic medication to address the symptoms. 

Companies like Roche, Biogen, Ovid therapeutics, and Ionis, are among several companies that are finding potential cure taking forward Angelman syndrome market. 

Ovid Therapeutics is running Phase III clinical trials for its drug, Gaboxadol (OV101). The drug is a small molecule, which selectively activates GABAA receptors located on the surface of nerve cells and away from the synapse. OV101 is considered as the first compound that targets disrupted tonic inhibition. Also, it has been granted with Fast Track Designation and Orphan Drug Designation by US Food and Drug Administration.

In addition to this, Angelman syndrome pipeline also involves gene therapy candidates such as GTX-101 which is a therapy of GeneTx Biotherapeutics. It is an investigational antisense oligonucleotide designed to inhibit transcription of the UBE3A antisense transcript (UBE3A-AS) across the ordinarily silent paternal allele of UBE3A.

Another therapy by the same company named GTX-102 is also an antisense therapy; however, it silences gene expression by binding to the RNA molecule that regulates its expression. It is designed to target and inhibit the molecule that silences the paternal copy of the gene, called UBE3A antisense transcript (UBE3A-AS).

To tackle the current unmet needs in the field of Angelman syndrome treatment market and provide better treatment options, a robust Angelman syndrome pipeline is anticipated. Research for future Angelman syndrome treatment options is focused on gene therapies, topoisomerase inhibitors, minocycline, cannabidiols and PP2A inhibitor. With the ongoing research studies at both industry and university level, and increased in funding, the future of Angelman syndrome treatment seems promising.