BioMarin Pharmaceutical Inc.

A biologic is a type of medicine that is made using a living organism, such as a cell. These complex molecules can perform specialized tasks in the body to target the underlying mechanisms of complex conditions. Our scientists are harnessing biologics to research innovative therapeutic options for people living with genetically defined conditions. For example, in metabolic disorders such as phenylketonuria (#PKU), the presence of certain gene variants can disrupt enzyme production and interfere with essential cellular processes. Research into biologics offers a way for scientists to pursue targeted solutions for PKU and other rare conditions.

The BioMarin Women’s Alliance (BWA) invited members to reflect on their time at BioMarin, and discuss how recognizing moments such as Women's History Month promotes a culture of belonging. Read insights from Serena Tamura, PhD, Diana Harrington Peroni, and Lubna Mustafa, and follow along for more on our celebration of Women's History Month.

Our team left #ACMGMtg25 inspired and grateful for the chance to connect with members of the scientific, medical and patient communities who share our dedication to improving the lives of those with genetically defined conditions. We’re encouraged by the progress in #PKU and skeletal conditions, including #achondroplasia, that was highlighted at the meeting. We remain determined to continue following the science to expand our impact for patients who are counting on us. Read more in our press release: https://bit.ly/4ibHrEZ

We announced data in #achondroplasia and #PKU today at #ACMGMtg25, underscoring the impact of our efforts to help people living with genetically defined conditions. Read our press release to learn more: https://bit.ly/4ibHrEZ

The BioMarin Women’s Alliance (BWA) Ireland chapter celebrated Women’s History Month with a fireside chat featuring Geraldine Cregan, Vice President, Shanbally Operations, and Mary Leamy, Vice President, People, Global Technical Operations. This event marked the first gathering since the chapter's formation in October. Co-chairs Emma OLeary and Katie Creedon led the discussion, exploring Ger and Mary’s career paths, significant decisions and professional achievements. The panelists offered insights on navigating the industry as women leaders and responded to attendees’ questions with honesty and openness. Thanks to Ger and Mary for sharing their experiences and congrats to BWA Ireland on the official launch of the new chapter.

"It’s been an incredible journey these past two decades as our research has not only helped enable advancements in achondroplasia, but opened the door for clinical trials in other skeletal conditions as well." Dr. Ravi Savarirayan, Group Leader of Skeletal Biology at Murdoch Children's Research Institute (MCRI) in Melbourne, Australia, has been at the forefront of research advancing care for children with skeletal conditions for more than 20 years. During a recent visit to our headquarters in San Rafael, Dr. Savarirayan spoke to our employees about why he has dedicated his career to improving the lives of people with skeletal dysplasias, progress over the past two decades and where he hopes to see the field go next. As we join the medical genetics community at #ACMGMtg25 this week, read an interview with Dr. Savarirayan to learn more about what he shared with us: https://bit.ly/4i69Ps5

Peptides are small chains of amino acids that act as messengers and building blocks throughout the body. Scientists and doctors can leverage peptides to interact with specific biological pathways in a targeted way. Take C-type natriuretic peptide (CNP), for example. CNP is key to activating pathways that promote bone growth. Understanding how CNP functions has unlocked new insights into developing medicines for people with genetic skeletal conditions. We’re looking forward to discussing these advancements this week at #ACMGMtg25.

As Executive Director of Talent Acquisition, Johanna Lucas has spent more than a decade bringing outstanding individuals to BioMarin, helping build the teams needed to deliver breakthrough medicines for genetically defined conditions. Johanna is the first employee to be interviewed in our new series highlighting “The DNA of BioMarin” – our people. Drawing inspiration from the four bases that constitute DNA – A (adenine), T (thymine), C (cytosine) and G (guanine) – we’ve asked employees to reflect on the role the following concepts play in their everyday work: Aspiration, Translation, Connection and Gratitude. Learn more about Johanna’s career journey and her role in helping create a workplace and culture that lead to a profound impact for those counting on us: https://bit.ly/4huFItb #DNAofBioMarin

“Accessibility means access for all, adapted to the needs of everyone.” We were proud to support Rebeca Costa for the second year in her mission to make Rio de Janeiro’s iconic Carnival more inclusive and accessible for members of the dwarfism community and people with disabilities. Some of the improvements Rebeca championed this year included sign language, audio description for those who are visually impaired, accessible bathrooms, ramps, designated space for wheelchair users and stair-climbing chairs. We applaud Rebeca’s dedication to making this one-of-a-kind experience available to people of all abilities. Learn more about how Rebeca – a lawyer, influencer, consultant, podcast host and advocate – is using her voice and drawing on her lived experience to make her community more inclusive: https://bit.ly/4bGFzBE

The BioMarin Women’s Alliance (BWA) hosted a fireside chat with Cristin Hubbard, Executive Vice President and Chief Commercial Officer, as part of our recognition of Women’s History Month. The conversation with BWA co-chair Carolina Amador, Ph.D., covered a range of topics through the lens of Cristin’s career journey, including the natural ebb and flow of progress and setbacks, the two-way street of mentorship, and the importance of supporting women’s health at home and in the workplace. BWA also incorporated educational segments into the event that highlighted progress toward workplace equity, as well as the significant disparities that still exist. As we recognize International Women’s Day, we’re thankful to BWA for organizing this event and continually working to #AccelerateAction in pursuit of greater gender equity.