The next wave of medical innovation is taking shape in local labs. From new gene therapy delivery systems to radiopharmaceuticals designed to more precisely attack cancer, recent advancements are changing how diseases are treated. In this section, we highlight innovators pushing the boundaries of medicine and bringing new hope to patients.

Apertura Gene Therapy

Apertura is redefining gene therapy with engineered Adeno-associated virus (AAV) capsids designed to overcome delivery challenges to treatment. Apertura has created capsids that bind to specific human receptors, enabling targeted tissue delivery and improved clinical translation for severe, unmet medical needs.

Current brain-targeted gene therapies often require invasive surgical injections. Apertura’s lead asset, TfR1 CapX, offers a less-invasive, safer alternative — unlocking the potential for systemic delivery of therapies to the CNS. TfR1 CapX is an intravenously dosed AAV capsid that binds to human transferrin receptor 1 (TfR1) to cross the blood–brain barrier (BBB) and achieve broad distribution across the brain and spinal cord. TfR1 is highly expressed throughout the BBB with stable expression across the lifespan, making it an ideal gateway for central nervous system (CNS) delivery.

Apertura is advancing TfR1 CapX in a gene therapy program for tuberous sclerosis complex (TSC). The company is partnering with a range of organizations — from academic labs to disease non-profit groups to private and public biotechnology companies — to address a variety of CNS disorders.

Founded in 2021 with technology from The Broad Institute, Harvard Medical School, and financial and operational support from Deerfield Management Company, Apertura is driven by the mission to make gene therapy accessible to patients with the greatest needs.

For more information, visit www.aperturagtx.com and follow us on LinkedIn.

ARTBIO is redefining how radiation is used in precision cancer care.

ARTBIO is a clinical-stage radiopharmaceutical company pioneering a new class of targeted cancer therapies. These therapies, called alpha radioligand therapies (ARTs), deliver radiation directly to tumors while limiting damage to healthy tissue. By pairing a tumor-targeting molecule with a powerful cancer-killing payload (isotope Pb-212), they are creating therapies that combine precision with power.

They are advancing a pipeline of new medicines, starting with their lead program, AB001, now in clinical trials for advanced prostate cancer. This program represents a critical step toward making ARTs a practical and accessible option for patients with hard-to-treat cancers.

The payload Pb-212 has a short half-life that requires production, therapy manufacturing, and patient injections to occur in less than one day. ARTBIO’s AlphaDirect™ technology overcomes this challenge. As the first-of-its-kind method, AlphaDirect™ enables a distributed manufacturing model that brings production closer to patients and clinics, ensuring a more reliable and timely supply.

ARTBIO is built on nearly a century of pioneering science in radiation therapy. The foundation traces back to groundbreaking work at the University of Oslo and Norway’s Radium Hospital. Today, ARTBIO carries that legacy forward with the goal of transforming cancer care and shaping the future of precision radiopharmaceuticals worldwide. By combining the power of molecular precision oncology with the cancer-killing power of radiation, ARTBIO creates a new class of targeted medicines with the potential for unparalleled efficacy and safety.

For more information, visit artbio.com