Development for Non-Oncology Indications
What are the Cell and Gene Therapies of the Future?
Development in Neurology and Ophthalmology
Development in Stem Cell Therapeutics and Regenerative Medicine
Development in Cell & Gene Therapy to Treat Rare and Difficult Indications
Development in Cell Therapy for Solid Tumours
Development in Allogeneic Cellular Therapies

Development for Non-Oncology Indications

27 Jan 2022
Sartorius Stedim Biotech
Charles River

13:30 Chairperson’s Opening
Matthew M. Hewitt, Executive Director, Scientific Solutions Cell & Gene Therapy, Charles River Laboratories

13:35 A Differentiated Approach to Gene Therapy for Sickle Cell Disease

  • Aruvant is focused on developing gene therapies for rare diseases
  • ARU-1801 is in a phase 1/2 clinical trial in patients with severe sickle cell disease
  • Unlike other gene therapies, ARU-1801 has shown engraftment using only reduced intensity conditioning—a potential advantage for patients, providers and hospitals
  • Initial data has shown improvement, and in recent cases full resolution, of the primary symptom of SCD

Will Chou, Chief Executive Officer, Aruvant

13:50 NK Lineage Specific Differentiation of Human Induced Pluripotent Stem Cells for Clinical-Scale Allogenic Cell Therapies

  • NK-lineage specific differentiation utilizing human pluripotent stem cells
  • Scalable feeder-free 3D spheroid culture system mimicking NK development in vivo
  • Pure and potent cytotoxic NK cells ideal for future allogeneic cell therapies

Allen Feng, Co-Founder and Chief Scientific Officer, HebeCell Corp

14:05 Small Mobile Stem (SMS) Cells: A New Single Agent, Multitargeted Approach To Tissue Regeneration

  • Small Mobile Stem (SMS) cells are adult stem cells derived from human peripheral blood that demonstrate extensive proliferation, motility, and resilience
  • Enables allogenic cell therapy based upon favorable immunogenicity and meaningful interaction with cells key to tissue regeneration
  • Animal preclinical data confirm lung regeneration and the formation of blood vessels. IND Application in process for COPD

Abdulkader Rahmo, Chief Scientific Officer, SMSbiotech

14:20 Democratization of hematopoietic stem cell-based cellular therapies

  • Hematopoietic stem cell transplant (a.k.a. bone marrow transplant) cures >70 blood diseases
  • Major challenges with current hematopoietic stem cell transplants.
  • Garuda’s technology has a potential to democratize and transform the field of hematopoietic stem cell transplant and related cellular therapies.

Dhvanit Shah, Founder, President and Chief Executive Officer, Garuda Therapeutics

14:35 Speaker Q&A and Panel Discussion


Matthew Hewitt
Senior Director, Scientific Services, Cell and Gene Therapy
Charles River
Allen Fang
Co-Founder and Chief Scientific Officer
HebeCell Corp
Will Chou
Chief Executive Officer
Abdulkader Rahmo
Chief Scientific Officer
Dhvanit Shah
Founder, President and Chief Executive Officer
Garuda Therapeutics