6 biotech companies making a difference in pediatric care

Pediatric companies

As several diseases tend to affect children at an early age, it is no surprise that there are a number of biotech companies around the world looking to find new treatments specifically for children. In this article, we have listed six pediatric companies we think you should know about – some of which solely focus their attention on children’s medicines, while others have innovative pediatric programs in their pipeline among other adult indications.

Table of contents

    BrainChild Bio

    Launched by Seattle Children’s in December 2023, BrainChildBio, which describes itself as a “kids-first” biotech company, is harnessing CAR-T cell technology for tumors in the central nervous system (CNS), with an initial focus on pediatric indications. BrainChild Bio has been granted an exclusive license to novel CAR-T cell technology for CNS tumors developed at Seattle Children’s.

    The pediatric company’s initial CAR-T cell therapy program will focus on children’s brain tumors, prioritizing diffuse intrinsic pontine glioma (DIPG), which is an incurable type of childhood cancer that forms in the brainstem. Its clinical programs will be founded upon the work already started at Seattle Children’s Therapeutics, which consists of four clinical trials designed to validate the safety and confirm early efficacy of several different targets for CAR-T cell therapy in pediatric CNS tumors, with plans for preliminary results to be presented at some point in 2024. 

    The BrainChild-04 clinical study was initiated last year. It is evaluating four different targets in a single CAR-T cell therapy. Following the achievement of clinical proof-of-concept in DIPG, BrainChild Bio plans to seek pediatric registration for DIPG and then extend the therapeutic application of its novel CAR-T cell therapies to target additional difficult-to-treat pediatric – as well as adult – brain tumors, including glioblastoma and brain metastases. 

    Day One Biopharmaceuticals 

    Day One Biopharmaceuticals was founded to address the innovation gap between children and adults living with cancer. But, instead of the company focusing exclusively on developing medicine for children, it pushes for early clinical trials in both children and adolescent patients, rather than waiting for a drug to first be approved for adults before pursuing a pediatric indication.

    Last October, the U.S. Food and Drug Administration (FDA) accepted the pediatric company’s new drug application (NDA) for its lead candidate, called tovorafenib, as a monotherapy in relapsed or progressive pediatric low-grade glioma, which is the most common brain tumor diagnosed in children. Tovorafenib is a brain-penetrant, highly-selective type 2 Raf kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway; the dysregulation of this pathway has been shown to occur in many cancer types and it is one of the most commonly mutated oncogenic pathways in cancer.

    Meanwhile, the company’s pipeline also includes pimasertib, a small molecule inhibitor of mitogen‐activated protein kinases 1 and 2 within the MAPK signaling pathway. To date, pimasertib has been dosed in over 850 patients across more than 10 phase 1/2 clinical trials for various tumor types. Plus, the company has also recently entered into an exclusive, worldwide license agreement and research collaboration with Sprint Bioscience for its VRK1 program, which aims to develop treatments for pediatric and adult cancers with high unmet need.

    Oncoheroes Biosciences 

    Oncoheroes Biosciences is a Boston-based company exclusively focused on the discovery and development of better drugs for children and adolescents with cancer. The company says it actively looks for in-licensing opportunities in the pediatric oncology space, while also working to generate new proprietary assets for a number of pediatric cancer indications with high unmet medical needs.

    Oncoheroes is developing a small molecule polo-like kinase 1 (PLK-1) inhibitor, called volasertib, to treat rhabdomyosarcoma (RMS), which is a rare pediatric soft tissue cancer. PLK-1 is a known cancer target, as it is a key regulator of the cell cycle, is over-expressed in many cancers, and has been associated with poor survival. Volasertib has already been studied as a single agent in phase 1 in children with leukemia and refractory solid tumors. The company is also developing dovitinib, a pan-tyrosine kinase inhibitor targeting fibroblast growth factor receptor, vascular endothelial growth factor receptor, and other receptor tyrosine kinases. It is a drug class that is considered interesting for the treatment of pediatric bone sarcomas.

    In August last year, Oncoheroes submitted two Investigational New Drug (IND) applications to the FDA, seeking permission to initiate clinical trials for both volasertib and dovitinib. Then, in October, the company reported that both INDs had received the green light from the FDA to move forward. Not long after, Dovitinib also received orphan drug designation from the FDA. 

    Renaissance Pharma 

    A biotech company launched just last year, Renaissance Pharma’s vision is to provide breakthrough therapeutic improvements in rare pediatric diseases. As it launched, it also announced the signing of an exclusive license agreement with St. Jude Children’s Research Hospital for its lead candidate Hu14.18, a humanized antibody in development by St. Jude for the treatment of newly diagnosed high-risk neuroblastoma.

    Hu14.18 is one of the few anti-GD2 monoclonal antibodies that have been developed. The GD2 antigen is highly expressed in neuroblastoma tumors, making it a promising drug target for this type of cancer, which affects children, mostly under the age of five. The drug candidate is designed to bind to the surface of neuroblastoma tumor cells, which essentially rallies immune cells to kill the tumor cells. 

    Renaissance Pharma has already conducted a phase 2 trial which showed positive results. For the study, 64 patients were given chemotherapy drugs busulfan and melphalan over six cycles, along with Hu14.18, after which they also received radiation therapy and further immunotherapy. It was found that, after the first two cycles of the co-administered chemotherapy and immunotherapy, a 75% reduction in the tumor was observed in 42 of the patients.

    Sarepta Therapeutics 

    Sarepta Therapeutics is a company focused on gene therapy, with a mission to engineer precision genetic medicine for rare diseases. It has a pipeline of more than 40 programs in various stages of development. This broad pipeline is driven by its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. Currently, Sarepta has one gene therapy and three RNA-based therapies on the market in the U.S.

    The company’s AAV-based gene therapy for children, called ELEVIDYS, received accelerated approval from the FDA in June last year, making it the first and only gene therapy to be given the green light to treat Duchenne muscular dystrophy (DMD). The company’s asset is intended for the treatment of ambulatory pediatric patients aged four to five years old with DMD who have a confirmed mutation in the DMD gene. It works by addressing the root genetic cause of DMD, which involves mutations in the dystrophin gene that result in the lack of dystrophin protein. It does this by delivering a gene that codes for a shortened form of dystrophin to muscle cells known as ELEVIDYS micro-dystrophin. 

    Sarepta is also looking to broaden the approved indication of ELEVIDYS by removing age and ambulation restrictions and converting the approval from accelerated to traditional. On February 16, the company announced that the FDA had accepted its efficacy supplement to the Biologics License Application for ELEVIDYS so that the FDA can review this request.


    Using a three-pillar approach of restoration, treatment, and prevention, hearing loss company Sensorion is working on developing a portfolio of small molecule and gene therapy programs to address an unmet medical need in inner ear disorders. It has built a research and development (R&D) technology platform to expand its understanding of the pathophysiology and etiology of diseases related to hearing loss, which then helps the company select the best targets and mechanisms of action for drug candidates. 

    As part of its broad strategic collaboration with Institut Pasteur, the company also has two ongoing gene therapy programs aimed at correcting hereditary monogenic forms of deafness. Both are pediatric programs; OTOF-GT targets deafness caused by mutations of the gene encoding for otoferlin, while GJB2-GT targets hearing loss related to mutations in the GJB2 gene, the most common form of childhood deafness. 

    OTOF-GT was granted a rare pediatric disease designation and an orphan drug designation by the FDA in 2022. And, more recently, in January 2024, Sensorion announced that it had received approval for its clinical trial application to initiate a phase 1/2 trial of OTOF-GT in France, Italy, and Germany.

    Global pediatric drugs market size is growing year by year

    According to Precedence Research, the global pediatric drugs market size was estimated at $115.6 billion in 2022, and is projected to hit around $363.86 billion by 2032, registering a compound annual growth rate (CAGR) of 12.2% from 2023 to 2032. There are multiple driving factors behind the global pediatric market, including the escalating incidence of pediatric health issues, heightened awareness of children’s healthcare needs, and government-driven incentives to promote the research and development of pediatric drugs.

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