Cell therapy weekly: iPSC therapy IND for Phase III trial cleared
The US Food and Drug Administration (FDA; MD, USA) has cleared an IND (Investigational New Drug) application for an iPSC-based therapy in a Phase III trial, while a gene therapy for retinitis pigmentosa has received a positive opinion for Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency (EMA; Amsterdam, Netherlands). Plus, the California Institute for Regenerative Medicine (CA, USA) has awarded Keck School of Medicine of University of Southern California (USC; USA) researchers a US$6 million grant to develop a novel genetic therapy for glioblastoma.
The news highlights:
- iPSC therapy IND for Phase III trial cleared
- First gene therapy in Phase III for broad retinitis pigmentosa
- US$6 million grant to develop glioblastoma gene therapy
iPSC therapy IND for Phase III trial cleared
The FDA has cleared a Phase III IND application for Gameto’s (NY, USA) Fertilo, an iPSC-based therapy designed to mature reproductive eggs outside the body using engineered ovarian support cells. This approach reduces hormone injections by 80% and shortens treatment cycles to 2–3 days, offering a less invasive and more comfortable option for patients. It also lowers the risks of ovarian hyperstimulation syndrome and side effects from high-dose hormones.
Gameto will conduct the Phase III trial across 15 US sites, evaluating embryo development, pregnancy rates and safety outcomes. The trial will involve up to two days of hormone injections and up to two embryo transfers, with a voucher for a second attempt if pregnancy is not achieved.
Dina Radenkovic, CEO and Co-Founder of Gameto commented on the milestone, stating:
“We are proud to be pioneers in this field, with technology that has the potential to transform reproductive medicine. I’d like to express my gratitude to our team, whose work is focused on empowering women by offering a solution that is less invasive and improves the IVF journey. Fertilo is a solution for both men and women from all walks of life, offering millions of people new possibilities for family building at a time when declining birth rates present a growing global challenge.”
First gene therapy in Phase III for broad retinitis pigmentosa
Ocugen (PA, USA) announced that the EMA’s Committee for Advanced Therapies has given a positive opinion on classifying OCU400 as an ATMP, making it the first gene therapy in Phase III for broad retinitis pigmentosa. This classification allows Ocugen to engage more frequently with the EMA for advice as it prepares to file for Marketing Authorization in 2026. Both the FDA and EMA have confirmed that the ongoing Phase III trial can support Biologics License Application and Marketing Authorisation Application submissions, with simultaneous filings planned upon completion.
The Phase III liMeliGhT trial is enrolling 150 patients aged eight and older with early to late-stage retinitis pigmentosa.
“Receiving ATMP classification is another significant milestone toward bringing OCU400 to the market in Europe,” said Shankar Musunuri, Chairman, CEO and Co-founder of Ocugen. “This designation makes it possible to stay on track with our clinical and commercial strategy and potentially provide this novel modifier gene therapy candidate to all [retinitis pigmentosa] patients in the United States and Europe by 2027.”
US$6 million grant to develop glioblastoma gene therapy
The California Institute for Regenerative Medicine has awarded USC researchers a US$6 million grant to develop a gene therapy for glioblastoma. This gene therapy will use a novel delivery system designed to target cancer cells more precisely, reducing damage to healthy tissue. The three-year grant, led by USC in collaboration with the University of Florida’s (USA) Zolotukhin Lab, aims to advance the treatment toward clinical trials. The team will work with the USC/Children’s Hospital Los Angeles (USA) current Good Manufacturing Practice facility to ensure the therapy meets FDA safety and efficacy standards, while conducting further preclinical testing to confirm laboratory results.
“This is the best time to be in this field as a researcher and as a clinician,” said David Tran, associate professor at USC. “When I started seventeen years ago, I spent most of my time telling patients to get their affairs in order. Today, we’re talking about prolonging survival. In the next five to ten years, we’re going to see major changes in what a diagnosis of glioblastoma means for patients.”
