GondolaBio and Children’s Tumor Foundation Announce Partnership to Advance GondolaBio’s Neurofibromatosis Type 1 Program

Collaboration brings Children’s Tumor Foundation’s deep NF1 expertise and patient and family perspectives into development of GondolaBio’s neurofibromatosis type 1 (NF1) program

PALO ALTO, Calif. & NEW YORK--(BUSINESS WIRE)--GondolaBio, a clinical-stage biopharmaceutical company developing novel medicines for patients living with genetic diseases, and the Children’s Tumor Foundation (CTF), NF’s global drug discovery engine focused on neurofibromatosis and schwannomatosis (collectively known as NF), today announced a partnership to advance GondolaBio’s preclinical neurofibromatosis type 1 (NF1) program.

CTF will provide guidance as GondolaBio’s NF1 program advances through preclinical and clinical development, drawing on its deep knowledge of NF1, scientific and clinical expertise, and relationships across the NF community. The collaboration will also ensure that the experiences, needs, and priorities of people impacted by NF1 and their families inform the program’s development. In recognition of the value of CTF’s expertise and contributions, CTF will receive an equity interest in the GondolaBio NF1 program.

“We are pleased to partner with GondolaBio as it pursues a novel approach to addressing the needs of people living with NF1,” said Annette Bakker, Ph.D., Chief Executive Officer of CTF. “This partnership recognizes something important: the expertise built inside a patient-centric research foundation can play a critical role in drug development. We appreciate GondolaBio recognizing that value and building a partnership around it. Getting better treatments to patients faster requires building the infrastructure, bringing the right partners in early, and valuing what each contributes. We believe this kind of model can help change how rare-disease drug development gets done.”

For nearly five decades, CTF has acted as the catalyzer advancing NF research and treatment development, building deep expertise and bringing together patients, clinicians, scientists, and industry around the shared goal of moving promising treatments forward. Through this partnership, GondolaBio will draw on that expertise and the engine CTF has built, leveraging CTF’s understanding and real-world knowledge of NF1 as GondolaBio’s NF1 program progresses. The collaboration reflects a shared commitment to advancing therapeutic innovation and bringing more treatment options to people living with NF1 and their families.

“NF1 is a complex genetic condition that can have a profound and highly variable impact on individuals throughout their lives, underscoring the need for continued therapeutic innovation,” said Charlie Ge, Ph.D., Executive Director, Business Development & Operations and lead of GondolaBio’s NF1 program. “CTF has been a driving force in advancing NF research and bringing together patients, families, researchers, and industry around the shared goal of improving outcomes for this community. We value the opportunity to work closely with CTF as we advance our NF1 program and pursue a new approach with the potential to address important unmet needs for people living with this condition.”

“At GondolaBio, community engagement is central to who we are and how we approach the development of new therapies,” said Cybele Gouverneur, Head of Patient Advocacy at GondolaBio. “People living with NF1 and their families bring invaluable perspectives on the realities of the condition, including the challenges they face and the needs that shape their daily lives. CTF has built deep trust within the NF1 community, and we value the opportunity to work together to ensure these perspectives inform our work.”

About Neurofibromatosis Type 1

Neurofibromatosis type 1 (NF1) is a lifelong genetic condition caused by changes in the NF1 gene that can lead to tumors developing on nerves throughout the body, with signs and symptoms often emerging during childhood. NF1 is the most common form of NF, occurring in approximately 1 in every 2,500 births worldwide. NF1 affects individuals differently and is associated with a range of manifestations, including neurofibromas, bone abnormalities, and learning challenges. Although NF1-associated tumors are often benign, they can be debilitating and disfiguring, causing significant pain, functional impairment and profound effects on an individual’s health and quality of life. Some tumors can also become malignant. There is no cure for NF1, and despite recent treatment advances, there remains a need for additional therapeutic options.

About Children’s Tumor Foundation

The Children’s Tumor Foundation (CTF) is the global patient-centric engine driving NF research and treatment development. Everything we do is to bring treatments to patients as we work toward a cure. For nearly five decades, CTF has built the expertise, resources, infrastructure, and networks needed to accelerate progress across the NF research and development ecosystem. By bringing together patients, clinicians, scientists, and industry, CTF identifies opportunities, overcomes barriers to development, and moves promising therapies toward patients. Our patient-first, collaborative approach is driven by our mission to end NF. The engine has already delivered approved drugs and launched clinical trials, and CTF continues to fuel both the drug and financial pipelines that will put more treatments into development and through clinical trials.

About GondolaBio

GondolaBio is a clinical-stage biopharmaceutical company focused on developing novel medicines for patients living with genetic diseases. A sister company of BridgeBio Pharma, 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 into medicines that can help patients as quickly as possible.