GondolaBio Partners with Children’s Tumor Foundation to Advance Preclinical NF1 Drug Development Program
GondolaBio, a clinical-stage biopharmaceutical company focused on developing innovative medicines for people living with genetic diseases, has announced a strategic partnership with the Children’s Tumor Foundation (CTF) to advance its preclinical neurofibromatosis type 1 (NF1) program. The collaboration brings together GondolaBio’s drug development capabilities and CTF’s extensive expertise in neurofibromatosis research, clinical development, patient engagement, and the broader NF community.
Under the partnership, CTF will provide guidance and support as GondolaBio advances its NF1 program through preclinical research and toward potential clinical development. The collaboration is intended to incorporate CTF’s scientific and clinical knowledge, its relationships with researchers and healthcare professionals, and its understanding of the experiences of people affected by NF1 and their families.
The partnership also establishes a framework for recognizing the value of patient-focused research organizations in advancing therapeutic development. In acknowledgment of CTF’s expertise and contributions, the foundation will receive an equity interest in GondolaBio’s NF1 program.
Through this arrangement, the organizations aim to strengthen the connection between scientific innovation and the needs of people living with NF1. Their collaboration reflects a shared commitment to advancing new treatment approaches for a complex genetic condition that can affect individuals in different ways throughout their lives.
The partnership also highlights the importance of involving patient organizations early in drug development, particularly in rare diseases where specialized scientific knowledge, access to patient communities, and a detailed understanding of disease burden can help guide research priorities.
Partnership to Support NF1 Program Development
The collaboration will enable GondolaBio to draw on CTF’s established expertise as the company progresses with its preclinical NF1 program. CTF will provide guidance informed by its understanding of the condition, its scientific and clinical relationships, and its longstanding involvement in efforts to accelerate research and treatment development for neurofibromatosis and schwannomatosis.
The organizations intend to ensure that the experiences, priorities, and needs of people affected by NF1 remain an important consideration throughout the program’s development.
Patient perspectives can provide valuable context for therapeutic research, particularly when a disease has a broad range of manifestations and affects patients differently depending on their age, symptoms, and individual circumstances. By incorporating these perspectives early, drug developers can gain a better understanding of the challenges patients and families face and the outcomes they consider most meaningful.
For GondolaBio, the partnership provides access to CTF’s established network and specialized knowledge as the company works to advance its investigational approach. For CTF, the collaboration offers an opportunity to contribute its expertise to another drug development program intended to address unmet medical needs within the NF community.
The equity arrangement is a central feature of the partnership. Rather than limiting the foundation’s involvement to general consultation, the agreement recognizes CTF’s contribution through an ownership interest in the NF1 program. The announcement did not disclose the size of the equity interest or additional financial terms.
This structure reflects an approach to rare-disease drug development in which patient-focused organizations can contribute specialized expertise and receive a direct stake in the program they help support.
By aligning the organizations around a shared therapeutic objective, the collaboration seeks to strengthen the development process while maintaining a focus on the people who may ultimately benefit from a potential treatment.
Children’s Tumor Foundation Brings Decades of NF Research Experience
The Children’s Tumor Foundation has spent nearly five decades helping advance research and treatment development for neurofibromatosis and related conditions. The organization serves as a global drug discovery engine focused on neurofibromatosis and schwannomatosis, collectively referred to as NF.
Over the years, CTF has worked to bring together patients, families, scientists, clinicians, and industry partners around the common goal of improving outcomes for people affected by these conditions.
This experience has enabled the foundation to develop a broad understanding of the scientific, clinical, and practical challenges associated with NF research. Its work across the research community provides a foundation for helping drug developers navigate the specialized knowledge and relationships needed to advance programs in this area.
CTF’s involvement in GondolaBio’s NF1 program will draw on this accumulated experience. The foundation will help inform the program as it progresses through preclinical and clinical development, with the aim of ensuring that relevant scientific insights and patient-community perspectives are considered.
Annette Bakker, Ph.D., chief executive officer of the Children’s Tumor Foundation, emphasized the importance of recognizing the role that patient-focused research organizations can play in developing new medicines.
“We are pleased to partner with GondolaBio as it pursues a novel approach to addressing the needs of people living with NF1,” Bakker said.
She explained that the partnership acknowledges the value of expertise developed within a patient-centric research foundation and its potential contribution to drug development.
According to Bakker, advancing better treatments more quickly requires the right infrastructure, early engagement with relevant partners, and recognition of the contributions each organization brings to a development program.
She also expressed the foundation’s belief that collaborative models of this kind could help change how rare-disease drug development is conducted.
The partnership demonstrates how organizations with complementary capabilities can work together to support therapeutic innovation. By combining scientific development with disease-specific expertise and community engagement, the collaboration aims to establish a more integrated approach to advancing potential treatments for NF1.
Understanding Neurofibromatosis Type 1 and Its Impact
Neurofibromatosis type 1 is a genetic condition characterized by highly variable clinical manifestations. It can affect multiple parts of the body, and its impact may change as an individual grows older.
The condition is associated with alterations in the NF1 gene, which encodes neurofibromin, a protein involved in regulating cell growth and signaling. Disruption of this regulatory function can contribute to the development of tumors and other manifestations associated with the disease.
People living with NF1 may experience a wide range of symptoms and complications. These can include café-au-lait skin spots, neurofibromas, changes affecting the nervous system, skeletal abnormalities, vision-related complications, and learning or developmental challenges. The specific manifestations and their severity vary substantially between individuals.
Some patients experience relatively mild symptoms, while others face complications that require ongoing medical monitoring, repeated interventions, or multidisciplinary care.
The variable nature of NF1 creates challenges for both patients and healthcare professionals. Disease manifestations can emerge at different stages of life, and the condition may require long-term management involving specialists from multiple medical disciplines.
The impact also extends to families and caregivers, who may need to coordinate appointments, monitor symptoms, navigate treatment decisions, and respond to changing care requirements.
These challenges underscore the need for continued research into therapies that could address important disease manifestations and improve outcomes for affected individuals.
GondolaBio’s preclinical NF1 program is being advanced in this context. The company and CTF have described the program as a novel approach intended to address unmet needs within the NF1 community. However, the announcement did not disclose the specific therapeutic target, mechanism of action, drug candidate name, or detailed preclinical findings.
Further information about the program’s scientific strategy and development milestones will be needed to assess its potential clinical applications.
GondolaBio Emphasizes the Need for Therapeutic Innovation
Charlie Ge, Ph.D., executive director of Business Development and Operations and lead of GondolaBio’s NF1 program, highlighted the complexity of NF1 and the importance of continuing to develop new therapeutic approaches.
Ge noted that the condition can have a profound and highly variable impact on individuals throughout their lives, reinforcing the need for ongoing innovation.
He also recognized CTF’s role in advancing NF research and connecting patients, families, researchers, and industry partners around the goal of improving outcomes for the community.
GondolaBio values the opportunity to work closely with the foundation as it advances its NF1 program and explores an approach that could potentially address significant unmet medical needs.
The collaboration provides a way for GondolaBio to incorporate disease-specific knowledge into its research strategy. This may help the company better understand the clinical context in which its program is being developed and the priorities of the population it ultimately aims to serve.
For a preclinical program, such input can be particularly useful as researchers continue investigating a potential therapy before it advances into human studies. Scientific development involves evaluating a candidate’s properties, understanding its biological effects, and generating evidence needed to support subsequent development decisions.
Although the partnership does not itself establish the safety or efficacy of GondolaBio’s investigational approach, it creates a framework for bringing specialized expertise and patient-community insight into the development process.
The collaboration also reflects the broader importance of connecting laboratory research with the practical realities of living with a genetic condition.
Patient Advocacy to Remain Central to Development
Patient engagement is another important component of the partnership. GondolaBio has emphasized that community involvement is central to its approach to developing new therapies.
Cybele Gouverneur, head of Patient Advocacy at GondolaBio, highlighted the value of hearing directly from people living with NF1 and their families.
She noted that these individuals bring important perspectives on the daily realities of the condition, the challenges they encounter, and the needs that influence their lives.
Gouverneur also recognized the trust CTF has developed within the NF1 community and emphasized the opportunity to work with the foundation to ensure that patient perspectives inform GondolaBio’s efforts.
The involvement of patient organizations can help drug developers understand aspects of disease burden that may not be fully captured through laboratory research or clinical measurements alone.
For example, patients and caregivers can provide insights into the practical consequences of symptoms, the demands of ongoing care, the difficulties associated with accessing specialized services, and the outcomes they value when considering new treatments.
These perspectives can help researchers and development teams better understand the broader context in which a potential therapy may eventually be used.
In rare and complex genetic diseases, patient communities can also serve as important sources of shared experience and disease-specific knowledge. Organizations such as CTF can help connect researchers with these communities while supporting communication between scientific teams and the people affected by a condition.
Through the GondolaBio partnership, patient engagement is intended to remain part of the program’s development rather than being treated as a separate activity.
The collaboration may therefore help strengthen alignment between scientific objectives and patient priorities as the NF1 program moves forward.
A Collaborative Model for Rare-Disease Drug Development
The partnership between GondolaBio and CTF illustrates a collaborative model in which a biopharmaceutical company works with a disease-focused foundation to advance a potential therapy.
Rare-disease drug development can involve substantial scientific and operational challenges. Researchers may need to address complex disease biology, limited patient populations, specialized clinical expertise, and the need to identify meaningful outcomes for conditions that vary considerably between individuals.
Patient-focused foundations can contribute by bringing together scientific communities, supporting research networks, sharing disease-specific knowledge, and facilitating engagement with affected individuals and families.
For GondolaBio, CTF’s experience offers a source of guidance as the company advances its NF1 program. For the foundation, the partnership provides a direct opportunity to contribute to the development of a potential therapeutic approach.
The equity interest awarded to CTF further distinguishes the collaboration by recognizing the foundation’s contribution through a financial stake in the program.
While the organizations have not disclosed the financial details of this arrangement, the structure signals an effort to recognize the value of expertise and community relationships alongside conventional drug development capabilities.
The announcement also highlights the importance of building partnerships early. GondolaBio’s NF1 program remains at the preclinical stage, and CTF’s involvement is intended to support development as the program progresses toward potential clinical evaluation.
Early collaboration may create opportunities to incorporate scientific, clinical, and patient perspectives before later-stage development decisions are made. However, the program’s eventual progress will depend on its scientific results, development milestones, and the evidence required to support clinical testing.
Next Steps for GondolaBio’s NF1 Program
GondolaBio and the Children’s Tumor Foundation will work together as the company advances its preclinical NF1 program through subsequent development stages.
CTF will contribute its scientific and clinical expertise, understanding of the NF1 community, and established relationships across the research ecosystem. The partnership will also seek to ensure that the experiences and priorities of people living with NF1 and their families remain part of the development process.
The organizations have not announced a clinical trial start date, a development timeline, or specific milestones for the investigational program. Additional information will be needed to understand the candidate’s therapeutic approach and the steps required before potential human testing.
Nevertheless, the collaboration represents a meaningful development in efforts to advance research for a complex genetic condition with diverse clinical manifestations and continuing treatment needs.
By combining GondolaBio’s drug development capabilities with CTF’s longstanding commitment to NF research and patient engagement, the partnership aims to support a more coordinated path toward potential therapeutic innovation.
Ultimately, the success of the program will depend on whether its scientific approach can demonstrate sufficient evidence of safety and efficacy through the required stages of development. The partnership itself does not guarantee a successful treatment, but it establishes a framework for incorporating specialized knowledge and patient perspectives as the research progresses.
For people living with NF1 and their families, continued investment in research and collaboration among biopharmaceutical companies, foundations, clinicians, and patient communities remains important to expanding the range of potential treatment options.
Through this partnership, GondolaBio and the Children’s Tumor Foundation aim to strengthen that collaborative effort and advance the development of a potential new approach for addressing unmet needs in the NF1 community.
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.