Pipeline

PHOENIX NEST BIOTECH

Pipeline

PRODUCT

APPROACH

JLK-247

Gene therapy

LA-027

Enzyme replacement

AVP-6

Small molecule

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. 

 

LA-027 

Licensed from Lundquist Institution, Patented.

An intracerebroventricular enzyme replacement therapy for treatment of Sanfilippo Syndrome Type D or MPS IIID. 

 

AVP-6 

In house program, provisional patent filed.

A small molecule, intranasal therapy to treat synaptic dysfunction in neurodegenerative diseases.

Funding Support

LA-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

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

LA-027

APPROACH

Enzyme replacement

INDICATION

MPS IIID

DISCOVERY

PRECLINICAL

CLINICAL

PRODUCT

LA-027

APPROACH

Enzyme replacement

INDICATION

MPS IIID

DISCOVERY

PRECLINICAL

CLINICAL