Publications-Presentations-Press

PHOENIX NEST BIOTECH NEWS & EVENTS

Publications

Publications relevant to Phoenix Nest programs; authored, funded, or in collaboration with employees of Phoenix Nest and or Jonah’s Just Begun-Foundation to Cure Sanfilippo.

Pan X, Taherzadeh M, Bose P, Heon-Roberts R, Nguyen ALA, Xu T, Pará C, Yamanaka Y, Priestman DA, Platt FM, Khan S, Fnu N, Tomatsu S, Morales CR, Pshezhetsky AV. Glucosamine amends CNS pathology in mucopolysaccharidosis IIIC mouse expressing misfolded HGSNAT. J Exp Med. 2022 Aug 1;219(8):e20211860. doi: 10.1084/jem.20211860. Epub 2022 Jun 15. PMID: 35704026; PMCID: PMC9204472.

Ludwig J, Sawant OB, Wood J, Singamsetty S, Pan X, Bonilha VL, Rao S, Pshezhetsky AV. Histological characterization of retinal degeneration in mucopolysaccharidosis type IIIC. Exp Eye Res. 2023 Apr;229:109433. doi: 10.1016/j.exer.2023.109433. Epub 2023 Feb 27. PMID: 36858249; PMCID: PMC10103010.

Viana GM, Priestman DA, Platt FM, Khan S, Tomatsu S, Pshezhetsky AV. Brain Pathology in Mucopolysaccharidoses (MPS) Patients with Neurological Forms. J Clin Med. 2020 Feb 1;9(2):396. doi: 10.3390/jcm9020396. PMID: 32024172; PMCID: PMC7073982.

Cheng KW, Wang F, Lopez GA, Singamsetty S, Wood J, Dickson PI, Chou TF. Evaluation of artificial signal peptides for secretion of two lysosomal enzymes in CHO cells. Biochem J. 2021 Jun 25;478(12):2309-2319. doi: 10.1042/BCJ20210015. PMID: 34032266.

Wang F, et al. (2020). Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α-N-Acetylglucosamine-6-Sulfatase in Neonatal Mice. Mol Pharm. 2021 Jan 4;18(1):214-227. doi: 10.1021/acs.molpharmaceut.0c00831. Epub 2020 Dec 15. PMID: 33320673

Heon-Roberts R, Nguyen ALA, Pshezhetsky AV. Molecular Bases of Neurodegeneration and Cognitive Decline, the Major Burden of Sanfilippo Disease. J Clin Med. 2020 Jan 27;9(2):344. doi: 10.3390/jcm9020344. PMID: 32012694

Martins C, et al. (2019). Molecular characterization of a large group of Mucopolysaccharidosis type IIIC patients reveals the evolutionary history of the disease. Hum Mutat. 2019 Aug;40(8):1084-1100. doi: 10.1002/humu.23752. Epub 2019 Jun 22. PMID: 31228227

Carla Martins, et al. (2015) Neuroinflammation, mitochondrial defects and neurodegeneration in mucopolysaccharidosis III type C mouse model. Brain 2015 Feb;138(Pt 2):336-55. doi: 10.1093/brain/awu355. Epub 2015 Jan 6. PMID: 25567323

Presentations & Press

October 30, 2024

News Release - Phoenix Nest Initiates Natural History Study of Sanfilippo Syndrome type C

May 2021

ASGCT 24th Annual Meeting | “Patient Advocates Role in Advancing Gene Therapy”

October 2020

Parents using viral video to overcome the single obstacle in the way of saving their son’s life: $3 million

February 2018

WORLD Annual Conference | Mission Hide and Help-Sanfiippo Awareness Campaign

July 2016

PatientCrossroads (now Invitae) Announces Expansion of the Mucopolysaccharidosis (MPS) Patient Insight Network | Launch of ConnectMPS a Patient Registry

May 2013

Help for Patients with Orphan Diseases

January 10, 2023

News Release- Phoenix Nest announces the launch of ALL-127, a Natural History Study of Sanfilippo Syndrome type D

February 2021

A Retrospective Natural History Study of Mucopolysaccharidosis IIIC

February 2019

WORLD Annual Conference | Evaluation of Potential Chaperone Therapy for Mucopolysaccharidosis

February 2017

WORLD Annual Conference | How our Approach for Sanfilippo Could be a Prototype for Industrializing Rare Disease Drug Discovery and Development

February 2016

WORLD Annual Conference | Sanfilippo Patient Registry and Natural History Study

July 2012

Phoenix Nest -a bespoke virtual biotech-announces its launch in the wake of new law signed by President Obama

October 2021

News Release- Phoenix Nest awarded $3.4 million NIH Grant

October 2020

Columbus Children’s Foundation and Phoenix Nest Bio Partner to
Accelerate Gene Therapy for Sanfilippo Syndrome Type C

February 2019

ASHG Annual Conference | Development of Intrathecal Enzyme Replacement Therapy for Sanfilippo Syndrome type D (Mucopolysaccharidosis IIID)

September 2016

IMPS Bi-Annual International MPS Conference | ConnectMPS.org a patient registry

July 2013

Taconic Initiates Custom Model Generation Project to Support Sanfilippo Syndrome Research.