AVP-6
Sanfilippo syndrome (MPS III A-D) - Neuroprotection
DiscoveryActive
Key Facts
Indication
Sanfilippo syndrome (MPS III A-D) - Neuroprotection
Phase
Discovery
Status
Active
Company
About Phoenix Nest
Phoenix Nest is a private, pre-revenue biotech pioneering therapies for Sanfilippo syndrome, a fatal pediatric neurodegenerative disorder. Its pipeline includes gene therapy, enzyme replacement, and a neuroprotective peptide program, all supported by over $17 million in non-dilutive grants from the NIH and patient foundations. The company is characterized by a patient-founder ethos, a strong academic partnership network, and programs advancing through preclinical and early clinical study stages. Its mission is driven by an inherent sense of urgency to address these unmet medical needs.
View full company profile