At daedalus BIOTECH, our goal is to develop the first therapeutic for Primary Sclerosing Cholangitis (PSC), an inflammatory disease of the bile duct that is both rare and fatal. By establishing collaborations with leading researchers and laboratories, daedalus BIOTECH is focused on target optimization to treat PSC.
Coupled with the use of Lipid Nanoparticles (LNPs) to deliver our therapeutic payload to the target site, daedalus BIOTECH has embarked on a five year journey to develop a therapeutic from bench-top to patients with PSC.
PSC is a complex, rare inflammatory disease of bile ducts characterized by progressive fibrosis and stricturing. The clinical course of PSC is highly variable but generally progressive resulting in liver failure and the potential of malignant transformation.
At present, there are no effective therapies that alter disease course and prevent death or the need for liver transplantation. Although rare with a prevalence of 1:10,000, the complications, co-morbidities and malignant potential of PSC represent a significant burden for patients and health systems.
The cause of PSC is unknown but involves a complex interaction of genetic, epigenetic, environmental and microbiome exposures with the immune system.
Current therapeutic pipelines focus on four potential pathways1. Bile Acid Modification: "Toxic Bile Hypothesis."
2. Intestinal Microbiome Modification: "Leaky Gut Hypothesis."
3. Immunomodulation: "Aberrant Gut lymphocyte Hypothesis."
4. Anti-fibrotic Therapies: reflecting fibrosis as the final common pathway that correlates with the burden of disease.
Lipid Nanoparticle (LNP) delivery technology has been developed over the past 30 years and is a key component of the successful mRNA-based COVID vaccines.
A non-viral delivery system has the advantages of a reduced immune response, larger therapeutic payloads, multi-dosing capabilities, design flexibility and ease of manufacturing.
daedalus BIOTECH’s targeted therapeutic will use a novel LNP for delivery to the liver. By adjusting and modifying the four main LNP components (ionizable cationic lipids, phospholipids, cholesterol and PEG-lipids) we will tune our novel LNP to target multiple hepatic cell types (e.g. hepatocytes, cholangiocytes, portal myofibroblasts, etc.) and open the door for new therapeutic options.
“LNP's will be going into millions of arms over the course of this year,” says University of British Columbia nanoparticle scientist Pieter Cullis. “What was a fringe field back in the 1980s has turned into something that is mainstream now."
Source: C & En March 8, 2021
In Greek mythology Daedalus was the master craftsman, the creator of the labyrinth in which he was imprisoned but ultimately escaped with the wings he invented from feathers and wax. He was the father of Icarus, who in escaping with Daedalus, did not heed his father's warnings and flew too close to the sun.
Daedalus’ knowledge, power of technology, and wisdom to use it properly is the inspiration for daedalus BIOTECH as we explore and develop the first therapeutic to treat PSC.
Pieter R. Cullis, Ph.D. FRSC, Director, Life Sciences Institute, University of British Columbia; Chair, Personalized Medicine Initiative; Professor, Department of Biochemistry and Molecular Biology, Director, NanoMedicines Research Group, UBC. Dr. Cullis and co-workers have been responsible for fundamental advances in the generation, loading and targeting of lipid nanoparticle (LNP) systems for intravenous delivery of small molecule drugs and macromolecular drugs such as small interfering RNA (siRNA). This work has contributed to three drugs that have been approved by regulatory agencies in the U.S. and Europe for the treatment of cancer and its complications. Dr. Cullis has co-founded ten biotechnology companies, has published over 300 scientific articles and is an inventor on over 60 patents.
Brendan has been a physician and digital pioneer for the past 25 years. Trained at Yale and McGill, he has had a parallel career as an innovator and entrepreneur. Through his lifestyle medicine practice, Brendan's deep passion is to use the latest science to help people change their behaviours and optimize their health. In 2020 he was diagnosed with PSC (Primary Sclerosing Cholangitis). In the course of his research, to make sense of his diagnosis, Brendan turned to his network. A conversation with Pieter Cullis sparked the realization of the potential for LNP technologies to power novel approaches to the tremendous unmet therapeutic need of PSC. Daedalus Biotech was formed in 2021 with the mission to bring a new therapeutic to market for PSC.
Paul has been dedicated his career to the life sciences. Initially as a researcher at McMaster University, he then moved to the commercialisation of life sciences in pharmaceuticals and biotechnology companies. Paul's career includes business leadership roles in Canada, USA, UK and Europe. Adding to his global Corporate experience have been Start-ups and Not-for-Profits (NPO). Rare Diseases, including PSC, continue to inspire and motivate actions that successful navigation of the development and regulatory environments brings, namely, therapeutic for people with Rare Disease.
Dr. Kulkarni obtained his PhD from the University of British Columbia and has over 10 years of academic and industry experience in the nanoparticle drug delivery field. He has published over 25 peer-reviewed articles in prestigious journals. Dr. Kulkarniís research has focused on the role of the various lipid components in LNP and the biophysics that governs particle formation. His work has contributed to clinical translation, including scale-up and manufacturing of LNP systems in accordance with GLP and GMP regulations. Dr. Kulkarni is a leader in the design and development of lipid nanoparticle (LNP) formulations of small molecule and nucleic acid therapeutics.
Dr. Ramji is a Clinical Associate Professor at the University of British Columbia, practicing at the St. Paulís hospital site. He completed his Internal medicine and Gastroenterology fellowship training at the University of British Columbia, and thereafter undertook a Hepatology fellowship at the University of Toronto.
His area of clinical and research interest includes viral hepatitis, NASH and PSC. His center is involved in investigator-initiated studies, and Clinical Studies in phase IIa to phase IV. Other research interests include implementation science for identification and linkage to care of persons with liver disease.
Dr. Ramji also coordinates multiple provincial education programs including the Liver Forum and GP Liver Forum. He coordinates the BC HCV Network, is on the governing board for the Canadian Association for the Study of the Liver (CASL), is on the Steering committee for the national CanHepC clinical group, Canadian HBV network and a co-founder of the Canadian Nash Network (CanNASH). He has over 60 peer-reviewed papers, is a co-author for the Canadian Guidelines in HCV and HBV, and a reviewer for multiple scientific journals.
Allen has over fifteen years of multi-industry and multifunctional executive experience across various regions. He is involved in advising and collaborating with scientific researchers and founders to create and scale impactful technology ventures. He holds an MBA from the University of Oxford and a BComm from the University of British Columbia. He has also completed technology studies programs at the Stanford Centre for Professional Development and Singularity University.
John is an experienced entrepreneur, having built & sold several companies and co-led 2 global-enterprise turn arounds, but his real passion is for value-strategy. More specifically, helping passionate leaders harness the forces of disruption to create transformative new products and services that maximize both impact and value.
John’s strategy practice is split between one-on-one support for a handful of transformative CEO’s (Chief Strategy Officer role) and working with start-ups (well over 200 at this point) to help them hone their insight, vision and story so they can attract the talent & resources they need to succeed.
