Alltrna CEO Michelle Werner Spearheads tRNA-Based Treatments for Rare Genetic Diseases

Michelle Werner, CEO of Alltrna and CEO-partner at Flagship Pioneering, is leading the charge in developing innovative tRNA-based treatments for rare genetic diseases. With a personal connection to the cause and extensive experience in the pharmaceutical industry, Werner is driving Alltrna's mission to create transformative therapies for patients with limited treatment options.

A Personal Mission Driving Innovation

Werner's journey in the pharmaceutical industry began in the late 1990s when she worked as a clinical data manager at Beth Israel Deaconess Medical Center. A pivotal moment came when she formed a bond with a cancer trial participant who passed away during the study. This experience ignited her passion for developing better medicines for patients in dire need.

In 2020, while serving as AstraZeneca's global hematology franchise head, Werner's mission became deeply personal when her son was diagnosed with Duchenne muscular dystrophy on his 10th birthday. Frustrated by the lack of effective treatments and limited clinical trial options, Werner's resolve to address the inadequacies in rare genetic disease treatments was strengthened.

Alltrna's Groundbreaking Approach to Genetic Diseases

Alltrna, which emerged from stealth in late 2021 with a $50 million investment from Flagship Pioneering, is pioneering a novel approach to treating rare genetic diseases. The company focuses on developing tRNA-based medications to correct stop codon errors, a common cause of multiple genetic disorders.

Unlike traditional approaches that target diseases one at a time, Alltrna's strategy aims to develop drugs that can be used for multiple conditions. This innovative method has the potential to address thousands of known rare genetic diseases, many of which currently lack approved treatments.

Since Werner took the helm in April 2022, Alltrna has made significant progress:

• Advanced from promising in vitro and cell culture data to robust preclinical proof of concept
• Demonstrated efficacy of engineered tRNAs across three different disease mouse models
• Conducted work in nonhuman primates
• Raised $109 million in a Series B funding round in 2023
• Initiated investigational new drug-enabling work for clinical trials in rare liver diseases

Challenges and Future Prospects

Despite the promising advances, the field of tRNA-based therapeutics faces significant challenges. No tRNA drugs have yet reached the market or entered clinical trials. The recent closure of HC Bioscience, another startup in the field, highlights the difficulties in targeted delivery and other factors associated with this novel approach.

Nevertheless, Werner remains optimistic about the potential of Alltrna's technology. The company is preparing for clinical trials using a basket trial concept borrowed from oncology, which will allow them to test their engineered tRNA in patients with multiple diseases stemming from the same underlying mutation.

As the rare disease space continues to evolve, Werner believes that the industry is approaching a "golden age" of drug development. With advances in understanding DNA and RNA, coupled with new technological tools, the potential for breakthrough treatments is greater than ever. However, Werner emphasizes the need for urgency in the field, recognizing that patients cannot afford to wait for these life-changing therapies.