Aurora Therapeutics Launches to Realize Potential of Personalized Gene Editing for Millions of Patients with Rare Diseases

- Leveraging modular gene editors and new regulatory pathways, Aurora will develop and commercialize therapies that can be rapidly tailored to many rare variants -

- Founded by CRISPR pioneers Jennifer Doudna and Fyodor Urnov to scale up impact of gene editing therapies -

- Company incubated by Menlo Ventures with an experienced management team led by CEO Edward M. Kaye -

BOSTON--(BUSINESS WIRE)--Aurora Therapeutics today announced its official launch to transform personalized gene editing from a one-patient breakthrough into a scalable model capable of bringing therapies to millions of patients with rare diseases. The company launches with a $16 million seed financing from Menlo Ventures to create the first platform for treating rare genetic mutations that have historically been impossible to address at scale.

Aurora was founded by Jennifer Doudna, Ph.D., CRISPR co-inventor and Nobel Laureate, and Fyodor Urnov, Ph.D., a leader in translating genome-editing science into human therapeutics. Their vision builds on more than a decade of progress in CRISPR technology, including clinical validation and advances in rapid sequencing and mutation identification, opening the door to personalized therapies that correct rare, disease-causing mutations unique to each patient.

Aurora is now realizing this next frontier of genetic medicine through a repeatable, systematic approach. Following advances in guide design and AI-generated editors combined with innovations in regulatory and CMC strategies, the company aims to shift personalized gene editing from isolated breakthroughs to a platform capable of benefiting patients with rare mutations at the population scale.

“Aurora’s launch signals a turning point for personalized gene editing. We now have the science, tools and regulatory tailwinds needed to move from isolated success stories to a sustainable way of developing many therapies in parallel,” said Edward M. Kaye, M.D., Chief Executive Officer of Aurora Therapeutics. “Our team’s deep experience in rare disease drug development positions us to advance this vision with rigor and urgency.”

“Since its discovery, CRISPR has offered the promise of treating the root causes of genetic disease, but we lacked a scalable way to bring those therapies to patients with rare mutations,” said Aurora co-founder Jennifer Doudna, Ph.D. “By innovating in both clinical development and approval pathways, Aurora is showing the true promise of gene editing for patients who were previously out of reach.”

A key component of Aurora’s model is its use of emerging regulatory frameworks that support grouping multiple mutations within a disease into unified development paths. This umbrella approach is designed to make personalized therapies economically and operationally viable, addressing a longstanding gap in rare-disease drug development. Aurora is uniquely positioned to execute this model by pairing deep gene-editing expertise and hands-on clinical experience with purpose-built clinical, manufacturing, and quality systems designed for rapid, parallel development of mutation-specific therapies.

“Aurora is opening a new frontier in genetic medicine,” said Johnny Hu, Ph.D., Principal at Menlo Ventures. “We believe that the pairing of recent advances in gene editing and AI with a scalable development and regulatory strategy will dramatically expand the number of people who can benefit from gene editing. Menlo is excited to support the team in increasing access to these life-changing therapies.”

Aurora’s initial program focuses on phenylketonuria (PKU), a metabolic disorder caused by a wide range of mutations in the PAH gene that lead to toxic elevations of phenylalanine in the blood. Without early treatment and careful monitoring, PKU can lead to impaired brain development in children and adolescents. Even when early and strict disease control enables near-normal IQ, elevated phenylalanine levels continue to negatively affect cognitive functions — such as executive function, memory, and social skills — throughout life. For this reason, PKU requires lifelong management.

“Every week, PKU specialist physicians like me care for individuals with PKU who are unable to consistently maintain safe phenylalanine levels,” said Chet Whitley, Ph.D., M.D., Medical Director of the PKU Clinic and Professor of Pediatric Genetics & Metabolism at the University of Minnesota. “Gene editing has the potential to offer a definitive and durable treatment for PKU and other genetic diseases, and Aurora’s model provides a promising path to extend these therapies to far more patients — not just a limited subset.”

PKU’s high unmet need and well-characterized disease biology align closely with the U.S. Food and Drug Administration’s recently announced plausible mechanism pathway. Supported by published preclinical proof-of-concept data and encouraging regulatory feedback, Aurora is designing therapeutics intended to address multiple PKU-causing mutations from the outset, with plans to expand this approach to additional mutations over time.

Leadership Team and Board of Directors

Aurora has assembled a leadership team and Board of Directors comprising veteran biotechnology experts with deep experience in rare diseases and genetic medicine.

Leadership Team

• Edward M. Kaye, M.D., Chief Executive Officer
• Thomas Wechsler, Ph.D., Chief Scientific Officer
• David Litvak, MBA, Head of CMC and Technical Operations
• Morgan Maeder, Ph.D., Head of Research

Board of Directors

• Johnny Hu, Ph.D., Principal at Menlo Ventures (Chair)
• Greg Yap, Partner at Menlo Ventures
• Edward M. Kaye, M.D., Chief Executive Officer
• Fyodor Urnov, Ph.D., Professor of Molecular Therapeutics at University of California, Berkeley; Scientific Director of the Innovative Genomics Institute

Founders

• Jennifer Doudna, Ph.D., Li Ka Shing Chancellor’s Chair and a Professor in the Departments of Chemistry and of Molecular and Cell Biology at the University of California, Berkeley; Howard Hughes Medical Institute investigator; President and Chair of the Board of the Innovative Genomics Institute
• Fyodor Urnov, Ph.D., Professor of Molecular Therapeutics at University of California, Berkeley; Scientific Director of the Innovative Genomics Institute

About Aurora Therapeutics

Aurora Therapeutics is reinventing how personalized gene-editing medicines are developed and approved for patients with rare diseases. The company combines state-of-the-art CRISPR engineering with umbrella regulatory pathways and modular CMC frameworks that allow many mutation-specific therapies to move forward in parallel. Aurora’s approach addresses the fundamental economic and operational barriers that have historically prevented treatments for rare mutations, enabling CRISPR therapies to reach patients at population scale. For more information, visit http://auroratherapeutics.com.

About Menlo Ventures

Menlo Ventures is a leading early-stage venture capital firm investing in digital health and novel life science companies. Our portfolio includes more than 85 public companies and over 170 exits through mergers and acquisitions. Currently managing more than $7 billion in assets, we invest at the intersection of bio and tech, across consumer, enterprise, and healthcare, with a differentiated approach in core thesis areas including therapeutic platforms, transformative technologies, digital health, and healthcare SaaS. Our portfolio companies include Benchling, Chai Discovery, Delfi, Eleos Health, Function Health, Gilead, H1, PillPack, Recursion, and Xaira. We strive to have a positive impact on everything we do, and we believe innovations at the intersection of tech and bio will drive major improvements in the quality of our health and the cost of healthcare. When we’re in, we’re ALL IN.

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