Title: LentiCure B.V.: Development of lentiviral gene therapy for Pompe disease for affordable and transparent pricing
Session: Next Generation Therapies: Presentations from Industry
Speaker:
Professor Pim Pijnappel, PhD
Erasmus University Medical Center
Abstract:
Dirk van Asseldonk1, W.W.M. Pim Pijnappel1-4, Giacomo Zundo2-4, Tessa Huizer2-4, Isabel Gordaliza Alaguero1-4, Bodil Willumsen4, Ans van der Ploeg2,4, Hannerieke van den Hout2,4, Michelle Kruijshaar2,4, Nadine van der Beek2,5, Pieter van Doorn2,5.
Enzyme therapy is effective in Pompe disease by also has a number of drawbacks including its invasive nature, the inability to stop disease progression, a heterogeneous response, the inability to reach the brain, the development of neutralizing antibodies that can interfere with treatment efficacy and safety, and a high price which threatens its reimbursement by national healthcare payers. It is therefore imperative to develop alternative treatment strategies.
We have developed hematopoietic stem and progenitor cell mediated-lentiviral gene therapy (HSPC-LVGT) in a mouse model for Pompe disease. In this form of gene therapy-fundamentally different from AAV gene therapy-, a classical bone marrow transplantation using cells from the patient him/herself is performed in which stem cells of the bone marrow are treated in the laboratory to permanently insert a copy of the GAA gene into the DNA of the stem cells. The stem cells are transplanted back into the patient, and will secrete the GAA protein to correct the brain. HSPC-LVGT can also provide immune tolerance to the GAA protein, thereby preventing the formation of neutralizing antibodies. In a mouse model for Pompe disease, we developed a modified version of HSPC-LVGT with improved efficacy that is able to completely correct the heart, muscles, and brain. HSPC-LVGT has an expected life-long efficacy after a single intervention. It has shown to be safe and effective in a number of diseases, and has resulted in approval for the treatment of metachromatic. leukodystrophy (LIbmeldy), a disease affecting the brain and closely related to Pompe disease.
Certain gene therapies have been developed for use in patients by the industry for very high prices, in the millions of dollars range. It is unknown how these prices have been established and what the actual costs have been. The high prices pose a challenge for healthcare payers to cover the costs. This has already led to certain patient groups to be expelled from eligibility or specific countries not granting reimbursement for a therapy. This problem is expected to get worse over time with the development of gene therapies for increasing number of disorders. These drawbacks have caused a change in the business strategy of companies and have already resulted in their withdrawal from the field of gene therapy for rare diseases.
For this reason, we have established LentiCure B.V. as a spin off company that is 100% owned by Erasmus MC. The aim of LentiCure is to bring gene therapies to patients for reasonable and transparent pricing. This public company aims to be funded by non-commercial parties, margins and profit will be in a socially acceptable range, used to maintain the company or to invest, e.g. in gene therapies for additional diseases. Finances are based on alternative sources including foundations and philanthropy. LentiCure in collaboration with Erasmus MC is currently preparing for the first in-human trial of HSPC-LVGT for Pompe disease. It aims to provide a platform for the development of lentiviral gene therapies for multiple rare genetic disorders.
Speaker Biography:
Dr. W.W.M. Pim Pijnappel is a professor at the Erasmus MC and has a broad expertise ranging from basic to clinical science. He performed his PhD research at the Hubrecht Laboratory (Utrecht, the Netherlands), and post-doctoral research at the EMBL Heidelberg and TU Dresden (Germany). His research group at the Center for Lysosomal and Metabolic Diseases at Erasmus MC in Rotterdam, the Netherlands, focuses on basic, translational, and clinical aspects of lysosomal storage disorders including Pompe disease and Hunter syndrome. The aim of his research is to obtain a better understanding of disease mechanisms, to develop novel treatment options, and to understand genotype/phenotype relationships. He has published more than 80 peer reviewed scientific articles and holds 11 patents on therapies and biomedical technology.
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Website: https://amda-pompe.org/
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