Conference Day Two

Hanson wade have taken the decision to cancel this meeting. Please do accept our apologies for any inconvenience or disappointment this will cause. Please register your interest if you would like updates on the meeting or topic.

08:00 am | Pre-Conference Coffee & Networking

Optimizing Enzyme Replacement and mRNA Therapy

9:00 am Engineered Enzymes As Next-Generation ERTs


  • While enzyme replacement therapies (ERTs) help many patients with genetic disorders, the enzymes being replaced often have sub-optimal properties as drugs – poor stability in body fluids, low uptake in target cells and tissues, and high immunogenicity
  • We describe our CodeEvolver® protein engineering platform that incorporates high-throughput screening, next-gen sequencing, and artificial intelligence to iteratively evaluate large protein diversity space in order to select therapeutic enzymes with dramatically improved properties
  • We present case studies demonstrating the application of this technology toward the engineering of therapeutic enzymes that are highly stable and active within the gastro-intestinal tract or have improved properties for systemic correction of lysosomal dysfunction

9:30 am Single ERT to Address Calcification Disorders with Overlapping Phenotype

  • Yves Sabbagh SVP & Chief Scientific Officer, Inozyme Pharma


  • ENPP1 and ABCC6 Deficiency are rare genetic calcification disorders with high unmet medical need and no approved therapy
  • Both disorders have low circulating levels of pyrophosphate, a potent inhibitor of calcification, leading to vascular calcification and other complications affecting soft tissues and the skeleton
  • Using an ERT approach with ENPP1-Fc protein, proof of concept was achieved in pre-clinical models specific to both diseases with increased PPi levels and prevention of vascular and soft tissue calcification supporting the evaluation of the clinical candidate INZ-701 as a therapeutic agent

10:00 am Panel Discussion: Innovating ERT Modalities to Expand the Array of Options Available to Patients


  • Look back at what advancement have been made in the field and where the new opportunities could lie
  • Analyze case studies of innovations that have been successful and unsuccessful

10:00 am | Speed Networking & Morning Coffee Break

Collaboration within the field as well as with Large Pharma Companies & Patient Advocacy Groups to Advance Research & Reduce Unmet Clinical Needs Around the World

11:00 am Effective Developer & Provider Collaboration to Bring Novel Therapeutics For IEM to Patients


  • Role of medical affairs throughout the therapeutic product development life cycle
  • Tools for advancing engagement providers during therapeutic development
  • Effective health care provider collaboration during IEM gene therapy development

11:30 am Panel Discussion: Keeping the Patient in the Centre of the Conversation: Improving Collaboration Between Pharma, Biotech & Patient Advocacy Groups

  • Jodie Gillon Chief Patient Officer, Abeona Therapeutics
  • Terri Klein President & Chief Executive Officer, National MPS Society
  • Kent Christopherson Senior Director, Medical Affairs, Orchard Therapeutics
  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation


  • Address patient access and recruitment in an increasingly competitive landscape to more effectively source the right patient for the right trial
  • Reviewing patient recruitment pathways to more confidently access the right patient populations for the right trials
  • Evaluating challenges encountered in small patient number trials

12:00 pm | Lunch Break

Preclinical Development of Novel IEM Therapeutics

1:30 pm Evolution of Genome Editing: In Vivo Gene Insertion using CRISPR-Based Therapies

  • Sean Burns Senior Director - Disease Biology, Intellia Therapeutics

2:00 pm Modulation of Endogenous Gene Expression in Urea Cycle Disorders


  • Novel approaches to upregulate gene expression
  • Potential to work across multiple UCDs
  • Early discovery data suggests therapeutically meaningful impact in late onset patients

2:30 pm | Afternoon Coffee Break

Tissue, Organ & Systemic Therapeutic Delivery

3:30 pm The Development History of Pabinafusp Alfa (JR-141)


  • The development history of world-first approval of novel intravenous enzyme replacement therapy for the treatment of CNS manifestations and somatic symptoms for Hunter syndrome
  • Efficacy data on CNS symptoms, somatic and biomarkers, and safety profile in PI/II, PII, PIII studies of JR-141.
  • Future clinical development plan in the US, EU and Brazil.

4:00 pm Cell-Type Selective Targeted Delivery of a Recombinant Lysosomal Enzyme for Enzyme Therapies

  • Kate Cygnar Associate Director - Genome Engineering Technologies, Regeneron Pharmaceuticals


  • Antibodies or antibody fragments can be linked to lysosome enzymes to improve delivery to tissues and cell types of interest
  • Such molecules can be delivered as a purified protein, or via gene therapy delivery methods
  • Pre-clinical data for antibody-lysosomal alpha glucosidase (GAA) fusion proteins under study for Pompe disease will be discussed

4:30 pm | End of Main Agenda