DIPG is a devastating childhood brain cancer with few treatment options and a median survival of just 9–12 months. Targepeutics’ investigational therapy, GB13, is designed to target cancer cells and may enhance the effectiveness of radiation when given beforehand. After more than $4 million in investment, completion of required preclinical studies, and a clinical partnership with Beat Childhood Cancer’s network of 50+ children’s hospitals, GB13 is now preparing to enter clinical trials. The remaining $125,000 will fund FDA-required GMP storage and distribution of the drug to clinical trial sites across the U.S.—the final infrastructure needed to begin testing in children. Your support can help close this critical gap and move GB13 one step closer to the children and families who urgently need new options.
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DIPG (Diffuse Intrinsic Pontine Glioma) is one of the most devastating forms of childhood brain cancer. It develops in the pons, a small but critically important part of the brainstem that controls essential functions such as breathing, swallowing, and movement.
Because DIPG grows throughout this delicate part of the brain rather than forming a tumor that can simply be removed, surgery is not an option. This leaves children with very few treatment choices and an extraordinarily poor prognosis.
Today, the standard treatment is radiation therapy, typically given over about six weeks. Radiation can temporarily reduce symptoms and improve a child’s quality of life, but it does not eliminate the cancer. Sadly, most children survive only 9–12 months after diagnosis.
Targepeutics is developing GB13, an investigational therapy designed to recognize and target cancer cells while sparing healthy cells. This targeted approach could offer something children with DIPG urgently need: a treatment that attacks the cancer itself rather than simply managing its effects.
Importantly, Targepeutics’ research suggests that giving GB13 before radiation may make radiation more effective against the cancer. Rather than relying on radiation alone, this approach could potentially help weaken the cancer first, allowing radiation to have a greater impact.
Targepeutics has completed the extensive work required before a new treatment can be tested in children. This includes manufacturing the drug under the required conditions, completing safety and toxicity studies, and confirming that the drug can be safely used with the medical devices needed to deliver it.
The team is now preparing its Investigational New Drug (IND) application, which it plans to submit to the FDA this fall. This application is the critical regulatory step that allows a new therapy to move from laboratory research into human clinical trials.
Importantly, Penn State University has funded the work completed to date, as well as the anticipated clinical costs of the Phase 1 and Phase 2 trials. The team has therefore already crossed many of the most difficult and expensive hurdles between discovery and the clinic.
There is now one final funding gap: GMP-compliant storage and shipping of the drug to clinical trial sites. The FDA requires that an investigational drug used in a clinical trial be stored and transported under strict, quality-controlled conditions to ensure its safety and integrity.
This means that a relatively focused investment could unlock the next major step: getting GB13 into the hands of clinicians and, ultimately, into a clinical trial for children with DIPG.
The final step is to secure a qualified partner to store the GB13 drug and safely deliver it to clinical trial sites across the United States.
Targepeutics has selected Almac Clinical Services, a specialized clinical supply company, to provide these services. The vendor has been vetted by the Targepeutics management team and selected through a competitive and responsible bidding process.
The total cost for the required storage, handling, and distribution services is $125,000, which will be paid over a six-month period beginning in December 2026.
This funding will provide the critical infrastructure needed to ensure that GB13 can be stored and transported under the FDA-required conditions and ready for use when the clinical trial begins. In other words, this is the final piece of the puzzle needed to help move GB13 from the laboratory into the clinic and, ultimately, to children with DIPG.
Targepeutics has already completed the FDA-required manufacturing, safety, toxicity, and drug-device compatibility studies needed to prepare GB13 for clinical testing.
The team has also established an important clinical partnership with Beat Childhood Cancer, a consortium of more than 50 children’s hospitals across the United States. This network provides the clinical infrastructure and expertise needed to conduct clinical studies of GB13 in children with DIPG.
This is the final funding needed to move GB13 into clinical trials. More than $4 million has already been invested to complete the manufacturing, safety, and preclinical work required to reach this point. With this final investment, Targepeutics can complete the clinical supply requirements and take the next critical step toward testing GB13 in children with DIPG.
If this funding is not secured immediately, the team will need to identify an alternative source, potentially delaying the start of clinical trials. For children with DIPG, where treatment options are extremely limited and survival is short, even a modest delay has real consequences.
The broader societal impact is therefore not simply the loss of a $125,000 investment. It is the risk of delaying a potentially important new treatment for children facing one of the most devastating forms of childhood cancer. After millions of dollars and years of work have brought GB13 to the threshold of clinical testing, closing this final $125,000 gap could help ensure that the program does not lose momentum at the very moment it is ready to reach patients.
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Chief Executive Officer & Chair – Board of Directors
Sil started his career at Deliotte & Touche in audit and corporate finance positions. Since then, he has brought an entrepreneurial spirit to a number of start-ups in the early drug development space. His experiences include executive level managerial and business development roles with companies developing cancer and inflammatory therapies and iron replacement therapies. He has successfully negotiated in and out license agreements with academic institutions, and both private and public biotech companies.
Co-founder, Business Development and Operations Advisor
Dr. Maines brings over 25 years of drug development experience to the team. He is the Vice President for Research and Business Affairs at Apogee Biotechnology Corporation. He has experience in all facets of drug development, previously leading regulatory efforts to successfully advance new drugs into and through clinical trials as well as executing out-licensing agreements with these drugs.
Dr. Maines received his Ph.D. in Pharmacology and MBA from the Pennsylvania State University.
Chief Scientific Officer
Dr Schrecengost brings 20 years of cancer research to the team with more than a decade focusing on drug discovery and development. He has been with Targepeutics since 2020 and has lead orphan drug designation approval for the lead platform and successfully applied for SBIR grant funding. Randy oversees all aspects of research at Targepeutics and facilitates and manages collaborative research projects at multiple research institutions.
Prior to joining Targepeutics, he was Senior Scientist at Apogee Biotechnology Company where he managed the prostate cancer research program. His work was instrumental in moving the lead compound forward into prostate cancer clinical trials.
Dr Schrecengost received his PhD in Microbiology from the University of Virginia and completed a post-doctoral fellowship (studying prostate cancer) at Thomas Jefferson University.
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