Unveiling Tomorrow's Wellness: Exploring the Top 5 Potential Therapies in 2024

In the ever-evolving landscape of healthcare, the pursuit of groundbreaking therapies is at the forefront of scientific innovation. As we step into the promising realms of 2024, the horizon is adorned with potential therapies that hold the key to transforming the way we approach and treat various health conditions. Join us on a journey through the cutting-edge advancements that have the potential to shape the future of medicine. From revolutionary therapies to transformative technologies, this article unveils the top 5 potential therapies that stand as beacons of hope, paving the way for a healthier and more resilient world.

1. Johnson & Johnson, Inc.’s nipocalimab was granted U.S. FDA Breakthrough Therapy Designation in February 2024 for the treatment of individuals at high risk for severe hemolytic disease of the fetus and newborn (HDFN). HDFN is a serious and rare condition that occurs when the blood types of a pregnant individual and the fetus are incompatible, potentially causing life-threatening anemia in the fetus or infant. Nipocalimab is currently the only therapy reported to be in clinical development for the treatment of alloimmunized pregnant individuals at risk of severe HDFN.

2. CG Oncology’s Cretostimogene grenadenorepvec is currently being evaluated in multiple studies including Phase 2, and two Phase 3 studies in the U.S. and Canada in BCG unresponsive, Intermediate-Risk and MIBC. Cretostimogene grenadenorepvec is an investigational, intravesically delivered oncolytic immunotherapy being evaluated in BOND-003 (NCT04452591), a Phase 3 clinical trial for the treatment of BCG-unresponsive Non-Muscle Invasive Bladder Cancer. In addition, Cretostimogene grenadenorepvec, in combination with pembrolizumab in the same indication, is being evaluated in the Phase 2 CORE-001 (NCT04387461) clinical trial.

3. Alkeus Pharmaceuticals, Inc.’s ongoing TEASE-3 clinical trial in early-stage Stargardt disease, showed positive interim data showing gildeuretinol halted Stargardt disease progression for up to six years. TEASE-3 is the first clinical trial in early-stage Stargardt disease, a leading cause of inherited blindness in children and young adults. Gildeuretinol acetate (ALK-001) is a novel molecule created as a specialized form of deuterated vitamin A designed to reduce the dimerization of vitamin A without disrupting vision. The first three teenage patients enrolled in TEASE-3 and treated with oral gildeuretinol acetate remained asymptomatic and free of disease progression for their treatment.

4. Zevra Therapeutics, Inc. in January 2024 announced that the U.S. Food and Drug Administration (FDA) has acknowledged receipt of the resubmission of the New Drug Application (NDA) for arimoclomol as an orally delivered, first-in-class treatment for Niemann-Pick disease type C. Arimoclomol is indicated for the treatment of NPC and has been granted Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, and Rare Pediatric Disease designation by the FDA, and Orphan Medicinal Product designation for the treatment of NPC by the European Medicines Agency (EMA).

5. Verve Therapeutics Inc. in November 2023 announced the first human proof-of-concept data for in vivo base editing from the ongoing heart-1 phase 1b clinical trial of VERVE-101. Treatment with VERVE-101 led to dose-dependent reductions of disease-causing low-density lipoprotein cholesterol (LDL-C) in people living with heterozygous familial hypercholesterolemia (HeFH), a life-threatening inherited disease characterized by lifelong elevations in blood LDL-C and accelerated atherosclerotic cardiovascular disease (ASCVD). VERVE-101 is an investigational, in vivo base editing medicine designed to be a single-course treatment that inactivates the PCSK9 gene in the liver to durably lower blood LDL-C.