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
JLK-247 is an experimental gene replacement therapy for treating patients with Sanfilippo syndrome Type C. Consisting of a self-complementary AAV9 vector-mediated intrathecal delivery of a functional copy of the HGSNAT gene aiming to restore the enzyme activity in tissues that are deficient in HGSNAT and prevent the accumulation of heparan sulfate which results in the severe neurological damage.
Phoenix Nest was awarded NIH/NINDS grants to fund vector manufacturing and GLP toxicology studies. Nonclinical proof-of-concept data supports AAV9 gene therapy. Dose-response studies were conducted at University of Texas at Southwestern, under Dr. Stephan Gray. Toxicology studies are underway.
JLK-247 has received FDA Orphan drug designation.
In house program, provisional patent filed.
ALL-027
ALL-027 is an experimental enzyme replacement therapy for treating patients with Sanfilippo syndrome type D. Intracerebroventricular delivery of a recombinant GNS enzyme aims to restore the enzyme activity in tissues that are deficient in GNS and prevent the accumulation of heparan sulfate which results in the severe neurological damage.
Phoenix Nest received NIH/NINDS grant funding and completed dose-response studies and generated a GMP-grade cell bank in CHO cell line for enzyme production.
AVP-6
AVP-6 is a synthetic peptide for intranasal administration for a nose-to-brain delivery that is expected to target synaptic dysfunction. This experimental therapy is disease agnostic and is being developed to improve Sanfilippo syndrome subtypes A, B, C and D pathology related to neuronal synaptic transmission.
In house program, provisional patent filed.​​
Funding Support
ALL-027 has received funding support from:
​​​
NIH/NINDS R41, R42, R44NS089061: 09/01/2014 - 06/30/2024, Development and in vitro validation of therapy for Mucopolysaccharidosis III $7,271,886
ALL-127 has received funding support from:
​
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 received funding support from:
​
Cure Sanfilippo Foundation: 05/01/2020 - 05/01/2022, Gene therapy for Sanfilippo Type C using scAAV9, $379,539
Cure Sanfilippo Foundation: 04/01/2021 - 05/01/2021, Gene therapy for Sanfilippo Type C using scAAV9, $125,000
​
NIH/NINDS 1R44NS129393: 09/01/2023 - 08/31/2025, Development of Gene Replacement Therapy for Sanfilippo Syndrome type C $2,990,505
NIH/NINDS R44NS137892: 07/01/2024 - 06/30/2027, Identification of Potential Biomarkers and Clinical Tools for use in Mucopolysaccharidosis IIIC patients, $2,994,701
​
AVP-6 has received funding support from:
​
Cure Sanfilippo Foundation: 01/04/2021 - 03/31/2023, Neuroprotective Peptides for Treatment of Sanfilippo Disease, $207,000
​
Partnering/Licensing Opportunities:
​
If interested in licensing ALL-127 please contact Jill Wood at jwood@phoenixnestbiotech.com
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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