As February 28 is Rare Disease Day, we have three guests on the podcast today all relevant to rare diseases. And they are Dan Mandell, CEO and co-founder of GRO Biosciences; Stefano Portolano, chief executive officer, Azafaros; and from Replay, Adrian Woolfson, executive chairman, president, and co-founder, and Lachlan MacKinnon, CEO and co-founder.
Rare Disease Day
Taking place on February 28 each year, the event raises awareness and looks to generate change for the 300 million people worldwide living with a rare disease, as well as their families and carers.
This year, the day will be marked by more than 600 events in 106 countries.
Replay and The University of Texas MD Anderson Cancer Center create Syena
Replay, a genome writing company reprogramming biology by writing and delivering big DNA, and The University of Texas MD Anderson Cancer Center, have launched Syena, a new oncology-focused product company pioneering T-cell receptor (TCR) natural killer (NK) cell therapies (TCR-NKs).
Building on the intellectual property and technology from MD Anderson and Replay, Syena has the potential to create the next generation of cell therapy.
Azafaros is a clinical stage company founded in 2018 with a deep understanding of rare genetic disease mechanisms, and a compound library from Leiden University.
Its aim is to build a pipeline of disease-modifying therapeutics to offer patients and their families new treatment options. The company’s lead clinical-staged program is AZ-3102, a small molecule, orally available, brain penetrant azasugar, with the potential to treat GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) and Niemann-Pick disease type C (NP-C).
This week, the company announced it has been granted two Rare Pediatric Disease Designations (RPDD) by the United States Food and Drug Administration (FDA) for the treatment of GM1 and GM2 gangliosidoses, Orphan Medicinal Product Designation (OMPD) by the European Medicines Agency (EMA) for the treatment of GM2 gangliosidosis, and an Innovation Passport by the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for the treatment of GM1 and GM2 gangliosidoses.
GRO Biosciences is leveraging groundbreaking science to expand the amino acid alphabet and deliver on protein therapeutics.
The U.S. company is transforming treatments for increasingly prevalent chronic medical conditions including autoimmune and metabolic diseases to improve the lives of patients. GRObio is applying its platform to advance partnered and collaborative programs, as well as its own pipeline of protein therapeutics bearing unique NSAA (non-standard amino acid) chemistries.
The company’s NSAA therapeutics feature previously unattainable capabilities including unprecedented duration of action and precise regulation of the immune system.