GEMMABio launches new company

Philadelphia, USA-based Gemma Biotherapeutics (GEMMABio) – a global genetic medicines company - today announced the launch of Rare Therapeutics (RareTx), a clinical-stage company focused exclusively on advancing gene therapies for ultra-orphan diseases. RareTx commences operations today with a focus on several promising treatments for lysosomal storage diseases.

The start-up will leverage public-private partnerships and regional centers of excellence it has access to as an affiliate of GEMMABio, which has established key partnerships in Brazil with Fiocruz and Abu Dhabi. GEMMABio raised $34 million in seed funding last year.

The company noted that traditional biopharmaceutical companies are not developing gene therapies for ultra-orphan diseases due to the limited markets and reimbursement challenges associated with one-time treatments for those conditions. Charging high prices for these one-and-done treatments is not a global solution, as it restricts access to a limited number of high-income individuals and countries. This widens a growing gap between what can be done and what the current system will support, leaving most rare disease communities without access to potential treatments.