The newly launched Therapeutic and Medical Device Accelerator (the “Accelerator”), at Boston Children’s Hospital’s Technology and Innovation Development Office (TIDO), has announced inaugural awardees for its internal funding program, further expanding the hospital’s translational research services and ensuring that Boston Children’s research discoveries have the greatest impact on patient health.
“The Accelerator provides a one-stop shop for Boston Children’s researchers to access support in their innovation journey,” says Irene Abrams, VP for Technology Development & New Ventures and head of TIDO.
Under the leadership of Senior Director Nadine Beauger, PhD, MBA, the Accelerator serves as a bridge between discoveries from Boston Children’s renowned laboratories and clinics and the later-stage, de-risked investment opportunities sought by commercial partners and investors. With targeted funding, expert mentorship, and key technical and commercial collaborations through Strategic Alliance partnerships, the Accelerator is uniquely positioned to advance hospital innovations towards clinical impact by advancing them towards the next stages of development in collaboration with industry.
“After the successful launch of the Accelerator in April 2024, we are excited to enter this next key phase of our endeavor,“ says Beauger. “Our inaugural selected projects highlight Boston Children’s commitment to advancing groundbreaking innovations that cater to the needs of pediatric patients through leverage of expertise and resources in both academia and industry.”
Awardees and their Projects
Darius Ebrahimi-Fakhari MD, PhD (Neurology)
Comprehensive Preclinical Development of Small Molecule Modulators of Protein Trafficking to Restore AP-4 Deficiency
Hereditary spastic paraplegia is the most common cause of progressive spasticity and there are no therapies that halt disease progression. To address this unmet need, we facilitate the pre-clinical development of small molecule drugs identified through high-throughput screening. The lead compound identified crosses the blood-brain-barrier, making it an attractive candidate. This project will target key milestones for an investigational new drug application: 1) Drug target identification; 2) Pharmacokinetic testing for optimal dosing; 3) Testing in Ap4b1 knockout mice to determine reversal of key disease features.
Mark Puder, MD, PhD (Surgery)
A new intravenous lipid emulsion for the prevention and treatment of nutritional deficiencies in preterm infants
Preterm infants often require long-term intravenous nutrition including lipid emulsions (a source of fat). Currently available lipid emulsions are pro-inflammatory, liver-toxic, and contain insufficient levels of fatty acids (DHA and/or ARA) that support normal brain and eye development. We developed new, pharmaceutical-grade lipid emulsions that prevented liver injury and increased DHA/ARA levels in mice. We aim to conduct testing in large animals in order to bring this therapy to preterm humans.
Michael Kurtz, MD, MPH (Urology)
An improved vesicoamniotic shunt for fetal lower urinary tract obstruction
Mid-pregnancy, some babies are found to have blockage of the bladder; this problem is so serious that some babies will not survive. Fetal intervention (placing a tiny tube into the baby’s bladder while in the womb) more than doubles the chance of survival, but current devices tend to dislodge or clog. We have developed a device which resists both of these problems. Our goal is to ensure safe interaction with fetal tissue and allow straightforward, accurate placement by the fetal surgeon
Hao Wu, PhD & Judy Lieberman, MD, PhD (Program in Cellular and Molecular Medicine)
Activating gasdermin pores to induce pyroptosis and stimulate anti-tumor immunity.
The goal of this application is to expand the realm of immune therapy strategies and specifically advance gasdermin D agonists to preclinical development by performing ADME, toxicity, and PK. In multiple mouse tumor models, pulsed and low-level pyroptosis induction by those agonists suppressed tumor growth without harming non-cancer cells. Vaccination with gasdermin D agonist-treated cancer cells protected mice from secondary tumor challenge, indicating immunogenic cell death was induced.