Fertilo’s iPSC Technology: A Game-Changer in Reproductive Medicine

Gameto, a clinical-stage biotechnology company focused on advancing women’s health through innovative therapies, has recently achieved a significant milestone. The U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for Fertilo, paving the way for the first U.S.-based Phase 3 clinical trial of an induced pluripotent stem cell (iPSC)-based therapy. This development underscores the transformative potential of iPSC technology in addressing critical challenges in reproductive medicine.

Fertilo, Gameto’s flagship ovarian support cell (OSC) technology, is designed to mature eggs outside the body using iPSC-derived cells. This approach represents a stark departure from traditional in vitro fertilization (IVF) methods, which typically require 10-14 days of high-dose hormonal stimulation to mature eggs. Fertilo, on the other hand, leverages engineered ovarian support cells to replicate the natural egg maturation process in a laboratory setting. This innovation reduces the need for hormone injections by 80% and shortens the treatment cycle to just 2-3 days, offering patients a less invasive and more comfortable experience. Additionally, it mitigates risks such as ovarian hyperstimulation syndrome and minimizes side effects associated with high-dose hormone treatments.

Dr. Dina Radenkovic, CEO and Co-Founder of Gameto, emphasized the significance of this achievement: “FDA clearance to advance Fertilo into a Phase 3 trial is a landmark moment for Gameto and for iPSC technology. Our team is dedicated to empowering women by providing a solution that enhances the IVF journey, making it less invasive and more accessible. Fertilo is not just a breakthrough for women but for anyone seeking to build a family, addressing the global challenge of declining birth rates.”

The Phase 3 trial will evaluate Fertilo’s efficacy and safety in maturing healthy eggs outside the body, with a focus on embryo development, pregnancy rates at 12 weeks, and maternal health outcomes. This follows the historic achievement of the world’s first live human birth using Fertilo technology, which occurred in December 2024 at the Santa Isabel Clinic in Lima, Peru.

Gameto plans to conduct the Phase 3 trial across 15 sites in the United States. The study will employ a double-blind, randomized controlled design to ensure robust data collection and analysis. Participants will undergo up to two days of hormonal stimulation and up to two embryo transfers. Couples who have struggled with infertility for at least one year are eligible to participate, and those who do not achieve pregnancy during the trial will be offered a voucher for a second attempt.

Martin Varsavsky, Co-Founder and Chairman of Gameto and Founder of Prelude Fertility, highlighted the broader implications of Fertilo’s technology: “Fertility clinics have long sought a solution that makes the path to parenthood shorter, safer, and more effective. Fertilo’s three-day IVF and egg freezing process is a groundbreaking answer to this need.”

Dr. Christian Kramme, Chief Scientific Officer of Gameto, further elaborated on the potential of iPSC-based therapies: “Fertilo exemplifies how cell engineering can address reproductive health challenges. By targeting ovarian health at the cellular level, we are redefining fertility care. This milestone also underscores the broader applications of iPSCs in regenerative medicine, including potential treatments for menopause and ovarian cancer.”

Gameto’s work builds on the Nobel Prize-winning discovery of induced pluripotent stem cells by Shinya Yamanaka, which revolutionized regenerative medicine. As the first company to bring iPSC technology to a Phase 3 clinical trial in fertility care, Gameto is at the forefront of transforming patient outcomes and advancing the field of cell therapy.

In addition to its U.S. trial, Gameto has secured regulatory clearance for Fertilo in Australia, Japan, Argentina, Paraguay, Mexico, and Peru. The company has also announced a strategic partnership with IVFAustralia, part of the Virtus Health group, to expand access to Fertilo for broader patient populations.

Commentary by SuppBase columnist Alice Winters

Gameto’s Fertilo represents a paradigm shift in reproductive medicine, leveraging iPSC technology to address longstanding challenges in IVF. The reduction in hormonal stimulation and treatment duration is a significant advancement, offering patients a safer and more comfortable experience. However, while the potential is immense, it is crucial to consider the broader implications of this technology.

1. Ingredient and Formulation Analysis: Fertilo’s use of iPSC-derived ovarian support cells is innovative, but the long-term effects of these engineered cells remain to be fully understood. The technology’s reliance on cellular reprogramming raises questions about genetic stability and potential off-target effects.

2. Efficacy and Safety: The Phase 3 trial will provide critical data on Fertilo’s efficacy and safety. While the initial results are promising, rigorous evaluation is necessary to ensure that the benefits outweigh any risks, particularly in diverse patient populations.

3. Market and Consumer Impact: Fertilo’s ability to reduce treatment time and hormonal exposure addresses key pain points for IVF patients. However, its accessibility and affordability will be critical factors in determining its widespread adoption. The partnership with IVFAustralia is a positive step, but broader global access remains a challenge.

4. Ethical and Environmental Considerations: The use of iPSCs raises ethical questions about cellular manipulation and consent. Additionally, the environmental impact of scaling up such advanced biotechnological processes must be carefully managed.

5. Future Directions: Gameto’s focus on ovarian health opens doors for addressing other reproductive health issues, such as menopause and ovarian cancer. The potential applications of iPSC technology in regenerative medicine are vast, but they must be pursued with caution and transparency.

In conclusion, Fertilo is a groundbreaking innovation with the potential to transform fertility care. However, its success will depend on rigorous clinical validation, ethical considerations, and equitable access. As Gameto continues to pioneer this field, it must prioritize patient safety, transparency, and inclusivity to fully realize the promise of iPSC-based therapies.