Since the inauguration of President Trump in January, the United States has been constantly in the international news, but which US biotech companies are making headlines? Diana Spencer highlights five innovators.

According to Cervicorn Consulting, the US biotechnology market size could reach around $1,470.82 billion by 2033¹  – though it is worth bearing in mind that the prediction was made prior to extensive cuts and tariffs implemented by the Trump administration. This article takes a closer look at some of the companies that could be driving growth in the country in the next five years.

March Bio: Advancing autologous CAR-T therapies

Houston-based March Biosciences is a spin-out from the Center for Cell and Gene Therapy at Baylor College of Medicine, Houston Methodist Hospital, Texas Children’s Hospital. Its lead asset, MB-105, is a CD5-targeted CAR-T cell therapy currently in Phase I trials in patients with refractory T-cell lymphoma and leukaemia, with promising signals of efficacy and safety to date. A Phase II trial is expected to begin in early 2025. The company says that MB-105 is specifically engineered to overcome major hurdles related to T-cell targeting by overcoming T-cell fratricide while maintaining high potency against CD5 positive tumour cells.

March Bio raised $28.4 million in an oversubscribed Series A financing in October 2024, taking its total funding to $52 million. The company also agreed a partnership with Volnay Therapeutics to develop a robust manufacturing process for its lead asset, pre-emptively tackling a common issue with these therapies of a lack of manufacturing capacity.

Then in early 2025, March Bio received US Food and Drug Administration (FDA) orphan drug designation for MB-105 for the treatment of relapsed / refractory CD5-positive T-cell lymphoma. “Beyond an important regulatory milestone, securing orphan drug designation for MB-105 from the FDA underscores the critical need for new therapeutic options for patients with T-cell lymphoma,” comments Sarah Hein, Co-Founder and Chief Executive Officer. “Currently, patients with treatment-resistant or recurrent T-cell cancers face an extremely poor prognosis. The MB-105 Phase I trial has shown promising safety and efficacy signals in relapsed / refractory T-cell lymphoma patients. This designation further validates our development strategy as we prepare to initiate our Phase II clinical trial in early 2025.”

The company says MB-105 has potential in other CD5-positive haematologic malignancies, including T-cell acute lymphoblastic leukaemia (T-ALL), chronic lymphocytic leukaemia (CLL), and mantle cell lymphoma (MCL). 

Tune Therapeutics: Editing the epigenome

Tune Therapeutics launched in 2021 with foundational intellectual property from Duke University, $40 million in seed funding, and the aim of bringing gene, cell, and regenerative therapies into a “new era of human medicine”. “Genetic medicine is at a tipping point,” the company’s previous Chief Executive Officer Matt Kane said at the company’s launch. “We now understand that the driving force of human health and disease is not our genes, but the epigenomic elements that shape and control them. Until now, scientists and bioengineers lacked the combined understanding, clinical expertise, and technology needed to make epigenomic therapies a practical reality. Now, we have all three.”

In 2023 Tune announced the durable repression of gene PCSK9 in non-human primates using genetic tuning. This gene repression was accompanied by an enduring reduction of LDL cholesterol levels that is still ongoing almost two years after a single, transient delivery of the epi-silencing construct.  

The company is expected to launch a Phase Ib clinical trial for Tune-401, its investigational epigenetic silencing therapy designed to treat chronic hepatitis B (CHB), in 2025, following clinical trial approval from the Hong Kong Department of Health and the New Zealand Medicines and Medical Devices Safety Authority (Medsafe). In January 2025, Tune also announced the completion of over $175 million in financing to support the development of Tune-401 and additional gene, cell, and regenerative therapy programmes already underway.

Derek Jantz, Chief Scientific Officer, expands: “Despite the well-established role of epigenetics in virus control, Tune-401 is the first treatment to employ direct epigenetic silencing to inactivate a virus. We feel confident that this unique mechanism of action will deliver long-awaited benefits for HBV patients and create renewed interest and momentum in the broader field of genetic medicine.”

MAIA Biotechnology: Targeting telomeres

The most clinically advanced biotech on this list, MAIA is a targeted therapy, immuno-oncology company focused on the development of potential first-in-class drugs with novel mechanisms of action against cancer. Its lead programme is THIO, a cancer telomere targeting agent in clinical development for cancers with telomerase-positive cancer cells.

In late 2024, the US Food and Drug Administration (FDA) designated THIO for the treatment of paediatric-type diffuse high-grade gliomas (PDHGG) as a drug for a rare paediatric disease. THIO also holds orphan drug designations (ODD) in hepatocellular carcinoma (HCC), small cell lung cancer (SCLC), and glioblastoma.

“THIO is a versatile anti-cancer agent that has demonstrated positive results in multiple difficult to treat cancer types, including paediatric high-grade glioma, which is among the most treatment-resistant cancers in children. THIO is shown to activate the immune system while evading tumour immunosuppression, a novel therapeutic approach for this devastating childhood disease,” explains MAIA Chairman and Chief Executive Officer Vlad Vitoc.

A previous research collaboration between MAIA and Nationwide Children’s Hospital found that THIO combined with ionising radiation resulted in significantly decreased cell proliferation and produced potent anticancer effects in highly aggressive diffuse intrinsic pontine glioma (DIPG). THIO induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. THIO-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses.

In February 2025, Maia announced positive efficacy updates for its Phase II THIO-101 trial in advanced non-small cell lung cancer (NSCLC), which significantly boosted its share price. In the trial, THIO was sequenced with Regeneron’s immune checkpoint inhibitor (CPI) cemiplimab. “Treatment with THIO now shows a 99% probability that overall survival will extend past chemotherapy’s measure by a wide margin,” comments Vitoc. “THIO’s efficacy in advanced stages of NSCLC continues to exceed our expectations, especially in third-line treatment where the cancer is typically even more resistant to therapy.”

Based on the results, MAIA believes they could achieve accelerated FDA approval of THIO, dependant on the final results of the ongoing expansion of the THIO-101 trial.

Orna Therapeutics: Navigating nanoparticles

Orna Therapeutics’ focus is on a new class of fully engineered circular RNA (oRNA) therapeutics to unlock the potential of RNA medicine. The company says its circular RNA transcripts have advantages over traditional mRNA approaches, including simplified production, improved formulation into lipid nanoparticles (LNPs), and superior protein expression. Orna’s two unique LNP delivery platforms reach parts of the body other than the liver: panCAR, which targets multiple lineages of the immune system at once, and SiTu Editing in the Marrow (STEM), which targets stem cells in the bone marrow.

Orna is hoping to reach the clinic in 2026 with its in vivo CAR platform (panCAR), which targets B cell-driven autoimmune diseases and B cell malignancies. Meanwhile, its STEM platform is in development, which combines oRNA with CRISPR genome editing and LNPs to create therapies that can potentially correct genetic diseases of the bone marrow (such as sickle-cell disease and beta-thalassemia) within the patient. The company is also working on vaccine candidates in collaboration with Merck.

At the start of 2025, Orna announced plans to partner with Vertex Pharmaceuticals on gene therapies for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The three-year strategic research collaboration will utilise Orna’s lipid nanoparticle (LNP) delivery solutions in return for payments of $65 million, and up to $635 million based upon the achievement of specified milestones. Orna is also eligible to receive up to $365 million in additional option fees and milestones per product for up to 10 additional products. 

“Vertex is a leader in delivering next-generation approaches to treating haemoglobinopathies, and we are excited to collaborate with them to develop in vivo therapies that leverage our proprietary technologies to achieve unprecedented delivery to haematopoietic stem cells (HSCs),” says Amit Munshi, Chief Executive Officer. “Today’s collaboration further validates our industry leading extra-hepatic LNP delivery chemistries and highlights the importance of delivery to enable the next wave of RNA medicines.” 

Nuvig Therapeutics: Investigating immunomodulation

California-based Nuvig Therapeutics launched in 2022 with a $47 million Series A financing. The company develops therapies that are designed to induce natural mechanisms to restore immune homeostasis, rebalance immune function following inflammation, and improve the treatment options for patients.

“Recent scientific insights into mechanisms of immune regulation have led to our identification of novel therapeutic targets and protein therapeutic candidates that do not rely on immune suppression but rather promote a natural rebalancing of normal immune function,” says Pamela Conley, Chief Executive Officer and Co-founder. “Based on initial preclinical data, we believe Nuvig’s therapeutic approach has the potential to be broadly active in a variety of autoimmune diseases and could dramatically improve the health and overall quality of life relative to currently available therapies and products in development for patients with chronic inflammatory diseases.”

At the end of 2024, Nuvig closed a $161 million Series B financing. The company’s lead candidate NVG-2089, a first-in-class, recombinant, Fc fragment immunomodulator, is engineered to bind type II Fc receptors and engage an endogenous regulatory mechanism that improves autoimmune dysregulation. It has demonstrated powerful anti-inflammatory effects in animal models that are characterised by activated B cells, T cells, and certain myeloid immune cells. 

The company says the proceeds from the Series B financing will support clinical proof-of-concept studies for NVG-2089 and progression to Phase II clinical development in chronic inflammatory demyelinating polyneuropathy (CIDP). It is also developing NVG-2089 for autoimmune indications in dermatology and plans to initiate Phase II trials in 2025. The FDA has granted fast track designation for NVG-2089’s development in bullous pemphigoid. In addition, Nuvig is working on BESTech full-length antibodies and second-generation type 2 receptor agonists, currently in the discovery stage.

From DDW Volume 26 – Issue 2, Spring 2025 Read the digital issue

Reference:

1. https://www.cervicornconsulting.com/biotechnology-market

About the author

Diana Spencer is Senior Digital Content Editor at DDW. She has Diana 20 years’ experience in both print and digital editorial roles for B2B publications dedicated to the life sciences sector, including medical and pharmaceutical industry journals/magazines.