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The principle is simple. The promise is enormous.

DNA begets RNA, and RNA begets protein: that is the simplified central dogma of biology. At BioMarin, we are committed to developing breakthrough medicines that intervene at every level of the central dogma. When we opened our doors over two decades ago, our central scientific modality was enzyme replacement therapy, which we leverage to provide synthetic copies of a missing protein in some of the world’s rarest genetic conditions with high unmet medical needs. That targeted approach has resulted in the development of breakthrough treatments for five intractable conditions.

Today, BioMarin remains at the forefront of molecular medicine. By interrogating the human genome, we have designed breakthrough technologies that go deeper than protein or RNA to address the root cause of genetic disease: DNA. BioMarin is investigating the use of adeno-associated virus (AAV) gene therapy to deliver short strands of DNA to cells, which the body can use as templates for creating the proteins it needs, using its own, native machinery. This therapeutic platform leverages decades of research and scientific advancements and has the potential to treat multiple conditions.

Current Collaborations

Furthering our commitment to developing promising gene therapy candidates, our preclinical collaboration with DiNAQOR AG expands our gene therapy pipeline to include DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM), one of the most common genetic heart diseases. Up to 60% of HCM cases are genetic in origin, and an estimated 40% of those cases are due to mutations in MYBPC3, the gene that encodes cardiac myosin-binding protein C.

BioMarin’s platform expertise doesn’t stop at gene therapy. Our preclinical collaboration with Deep Genomics will utilize artificial intelligence (Deep Genomics’ AI Workbench) to identify oligonucleotide drug candidates for rare diseases with high unmet need, further expanding our portfolio of capabilities to enable therapeutic efforts that target not just DNA and protein, but RNA, as well.

With expanding platform expertise in multiple arenas, BioMarin’s commitment to finding the best approach for each unique condition has never been more clear.

Learn more about our gene therapy manufacturing

Our Unique Approach to Gene Therapy

BioMarin sets a gold standard in the production of investigational gene therapies, as one of very few therapeutic development companies with its own manufacturing facility. This intensive level of involvement allows for continuous process improvements, helping us establish high specifications in purity and potency. The safety and efficacy of BioMarin’s investigational gene therapies are currently under assessment for multiple conditions.

Our dedication is evidenced by the technical expertise we have built in the field of gene therapy, with many of the original pioneering minds in AAV gene therapy contributing as part of the BioMarin team. We continue our research to optimize gene therapy approaches, including novel capsid technologies that are designed to enhance the most beneficial attributes of this treatment method.