Pipeline
PHOENIX NEST BIOTECH
Pipeline
PRODUCT
APPROACH
JLK-247
Gene therapy
ALL-027
Enzyme replacement
AVP-6
Peptide
INDICATION
MPS IIIC
MPS IIID
MPS A-D
DISCOVERY
PRECLINICAL
CLINICAL
JLK-247
In house program, provisional patent filed.
A one-time intrathecal administration of AAV9 vector-mediated gene replacement therapy for the treatment of Sanfilippo Syndrome Type C or MPS IIIC.
ALL-027
Licensed from Lundquist Institution.
An intracerebroventricular enzyme replacement therapy for treatment of Sanfilippo Syndrome Type D or MPS IIID.
AVP-6
In house program, provisional patent filed.
A peptide, intranasal therapy to treat synaptic dysfunction in neurodegenerative diseases.
Funding Support
ALL-027 has been supported by these NIH grants:
NIH/NINDS 1R41NS089061-01: 09/01/2014 – 08/31/2015 Development and in vitro validation of therapy for mucopolysaccharidosis III, $223,102
NIH/NINDS 2R42NS089061-02/03: 06/01/20 - 09/29/2018 Development and in vitro validation of therapy for mucopolysaccharidosis III, $1,495,008
NIH/NINDS 2R44NS089061-04/05: 09/30/2018 - 08/31/2022, Development and validation of therapy for mucopolysaccharidosis III, $5,612,276
ALL-127 has been supported by:
NIH/NINDS 1UB1NS122644-01: 09/01/2021 - 08/31/2024, Evaluation of clinical outcomes assessment (COA) and potential biomarkers to facilitate Interventional trial for Mucopolysaccharidosis IIID Patients, $3,419,934
JLK-247 has been supported by Cure Sanfilippo Foundation:
CSF Grant 05/01/2020 - 05/01/2022: Gene therapy for Sanfilippo Type C using scAAV9, $379,539
CSF Grant 04/01/2021 – 05/01/2021: Gene therapy for Sanfilippo Type C using scAAV9, $125,000
Partnering Opportunities:
We are open to new opportunities to collaborate. If interested, please contact jwood@phoenixnestbiotech.com
PRODUCT
JLK-247
APPROACH
Gene therapy
INDICATION
MPS IIIC
DISCOVERY
PRECLINICAL
CLINICAL
PRODUCT
ALL-027
APPROACH
Enzyme replacement
INDICATION
MPS IIID
DISCOVERY
PRECLINICAL
CLINICAL
PRODUCT
LA-027
APPROACH
Enzyme replacement
INDICATION
MPS IIID
DISCOVERY
PRECLINICAL
CLINICAL