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, GLP toxicology studies and cGMP manufacturing. 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 an orphan drug designation and rare pediatric disease designation from the FDA.
JLK-247rp
JLK-247rp is an expansion of JLK-247, the rp stands for Retinitis Pigmentosa (RP). RP is a common symptom of Sanfilippo syndrome, however some people with HGSNAT mutations present with RP as adults and do not have cognitive disabilities. Phoenix Nest is investigating the impact of JLK-247 given as an injection in the eye.
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
JLK-247 has received funding support from:
NIH/NINDS 1R44NS147802: 07/14/2026-06/30/2028, Manufacturing and Analytical Development for JLK-247 Gene Therapy, $2,984,658
NIH/NINDS 1R44NS129393: 09/01/2023 - 08/31/2025, Development of Gene Replacement Therapy for Sanfilippo Syndrome type C, $2,990,505
JGJ Gene Research Inc: 10/05/2025-10/05/2027, Natural History Study to Determine the Disease Phenotype in the retina of the P304L Mouse, Time-frame of Disease Progression, and Biomarkers for Therapeutic Efficacy, $368,300
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
JLK-447 has received funding support from:
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
Vanicre Les Maladies Lysosomales: 02/14/2025, A Combination Retrospective and Prospective Natural History Study of Subjects with Sanfilippo Syndrome Type IIIC (MPS IIIC), $340,000
Vanicre Les Maladies Lysosomales: 02/08/2023, A Combination Retrospective and Prospective Natural History Study of Subjects with Sanfilippo Syndrome Type IIIC (MPS IIIC), $877,000
JLK-448 A.K.A. C-RARE has received funding support from:
Sanfilippo Sud and ISSA members: 02/28/2023-TBD, C-RARE development for JLK-448 International Remote only Prospective Observational Study, $120,000
AVP-6 has received funding support from:
Cure Sanfilippo Foundation: 05/06/2026-05/06/2028, Neuroprotective Peptides for Treatment of Sanfilippo Disease, $497,000
Cure Sanfilippo Foundation: 01/04/2021 - 03/31/2023, Neuroprotective Peptides for Treatment of Sanfilippo Disease, $207,000
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
Partnering/Licensing Opportunities:
If interested in licensing or partnering with us please contact Jill Wood at 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