• Collaboration provides GondolaBio access to n-Lorem’s AI-informed, world-class ASO capabilities to discover novel ASO medicines for patients in need
• GondolaBio is a clinical-stage biotechnology company founded in September 2024 with $300 million in backing from investors, including Patient Square Capital, Viking Global Investors, and Frazier Life Sciences
• The two organizations will jointly discover antisense oligonucleotide (ASO) therapeutics for two prespecified biological targets for genetic diseases with significant unmet need
• Upon selection, any resulting therapies will be clinically and commercially developed by GondolaBio’s subsidiary companies housing the corresponding drug development programs
• These two subsidiaries represent the seventh and eight formed by GondolaBio in the first three months of operating, and the company is actively seeking additional programs in genetically defined diseases
• The payments to n-Lorem under the collaboration agreement will help to fund n-Lorem’s other activities in pursuit of the nonprofit’s mission to provide ASO medicines to patients with nano-rare conditions for free, for life

GondolaBio and n-Lorem Foundation Partner to Advance Novel ASO Therapies for Genetic Diseases

n-Lorem Contact:
Amy Williford, Ph.D.
Sr. Director of Communications
amy.williford@nlorem.org

GondolaBio Contact:
James Li, Ph.D.
President
jl@gondolabio.com

n-Lorem, a nonprofit foundation, and GondolaBio, a clinical-stage biotechnology company founded in 2024 and dedicated to developing novel medicines for patients living with genetic diseases, today announced that the two organizations have entered into multiple collaborations to discover novel antisense oligonucleotide (ASO) medicines for patients who are not currently served by available treatment options. n-Lorem will leverage its robust, automated, high-capacity ASO discovery platform and significant expertise in ASO technology to discover, screen and select ASO medicines to two prespecified biological targets in genetic diseases with significant unmet need. Once selected, GondolaBio will move forward in development of these new medicines.

n-Lorem’s mission is to discover, develop and provide experimental ASO medicines to nano-rare patients, for free, for life. Consistent with that mission, n-Lorem is collaborating with GondolaBio. Under the terms of the agreements, n-Lorem will receive upfront payments and research and development expenses from GondolaBio and could receive additional payments for each ASO that is discovered and developed by the collaboration.

“We have been impressed with the vision and pioneering spirit of the GondolaBio team and are looking forward to working with them on these important programs,” said Stanley Crooke, M.D., Ph.D., Chairman, Founder and CEO of n-Lorem. “At n-Lorem, we have industrialized the treatment of nano-rare patients and created an organization capable of scaling up to meet a growing demand while maintaining high-quality at each step. The efficiencies of our lab ensure that we can conduct the work in this collaboration in parallel with the ongoing efforts of our mission to help nano-rare patients. The funding we receive from these transactions will enable us to broaden our investment in nano-rare patients and create opportunities for sustainable revenues through potential milestone payments and equity as these programs advance.”

GondolaBio strives to serve patients living with genetic diseases by developing novel and best-in-class therapeutics which target each condition at the source of the disease. Founded in 2024, GondolaBio was backed with $300 million from investors, including Patient Square Capital, Viking Global Investors, and Frazier Life Sciences. GondolaBio started with three subsidiary companies spun out of BridgeBio Pharma in September, and now with the n-Lorem collaboration, has eight total subsidiaries each housing one drug development program. GondolaBio is actively seeking the best industry and academic partners to create additional subsidiaries and make the next generation of life-changing medicines for genetic disease patients in need.

“We are excited to partner with Stan Crooke and the n-Lorem team to bring in our seventh and eight subsidiaries to develop revolutionary medicines for patients,” said Morgan Paull, Chief Operating Officer of GondolaBio. “This partnership will combine the category-defining expertise in antisense technology which Stan and n-Lorem have developed, with GondolaBio’s ability to rapidly and efficiently progress novel therapeutics in genetic diseases. It is especially meaningful to us that in addition to the programs we develop together, our collaboration will help to fund n-Lorem’s nonprofit mission to treat patients with nano-rare conditions – this is the best kind of win-win.”

n-Lorem’s ASO technology is unparalleled in its efficiency and scale to discover ASO medicines for patients in need. n-Lorem has assembled an experienced team of scientists with expertise across all areas of RNA-targeted drug discovery and employs a nimble process that incorporates AI-informed discovery engine based on more than 35 years of experience in ASO technology and high-throughput screening enabling the screening of hundreds of ASO candidates at once. This highly efficient, cost-effective discovery platform supports n-Lorem‘s mission to discover novel experimental ASO medicines for nano-rare patients.

About GondolaBio

GondolaBio is a clinical-stage biopharmaceutical company focused on developing next-generation therapeutics for patients living with genetic diseases. Created as a spinout of BridgeBio Pharma in 2024, GondolaBio aims to leverage cutting-edge biological research to create breakthrough medicines addressing high unmet needs across multiple therapeutic areas, modalities, and stages ranging from early discovery to clinical development. Its team of experienced drug discoverers, developers and innovators are committed to translating advances in genetic science and medicine to help patients as quickly as possible.

About n-Lorem

n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat nano-rare patients diagnosed with diseases that are the result of a single genetic defect unique to only one or very few individuals. Nano-rare patients describe a very small group of patients (1-30 worldwide) who, because of their small numbers, have few if any treatment options. n-Lorem Foundation was created to provide hope to these nano-rare patients by developing individualized ASO medicines, which are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. To date, n-Lorem received over 300 applications for treatment with more than 140 nano-rare patients approved. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. Follow us on Twitter, Facebook, LinkedIn and YouTube.

To learn more about n-Lorem’s mission at www.nlorem.org, and please consider giving to n-Lorem to bring hope, possibility and treatment options to these patients and families in need.

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