Rocket Pharmaceuticals received the rare pediatric disease priority review voucher with the accelerated FDA approval for Kresladi in March. The non-dilutive capital from the voucher’s sale will support a pipeline that includes a gene therapy in pivotal clinical testing for the rare genetic disorder Danon disease.
Rocket Pharmaceuticals received FDA approval for Kresladi, a gene therapy developed to treat leukocyte-adhesion deficiency type 1, an inherited immunodeficiency that can become fatal to infants and young children. Kresladi is the first gene therapy for this ultra-rare disease and the first commercial product for Rocket.
In an interview, Kyan Health Co-Founder and Chief Commercial Officer Konstantin Struck discussed how Kyan gives mid-market and enterprise employers access to premium workforce mental healthcare, at a price point that is affordable.