Susan Haigney

Gene Therapy, Rare Diseases, and Transfer RNA

Rare diseases, defined by FDA as “a disease or condition that affects less than 200,000 people in the United States” (1), may have the unique challenge of not being easily applied to multiple disease conditions; therefore, pharmaceutical companies may find it challenging to invest the time and resources into developing these important medicines.

Transfer RNA (tRNA)-based therapies are one option for treating rare diseases. At this year’s J.P. Morgan Healthcare Conference, being held in San Francsico from January 12–15, Tevard Biosciences will be highlighting preclinical data on the company’s suppressor tRNA platform, which has been shown to restore full-length, native full-length, native dystrophin, and titin in models of Duchenne muscular dystrophy and dilated cardiomyopathy caused by titin truncations (2).

spoke with Elisabeth Gardiner, PhD, chief scientific officer of Tevard Biosciences, about what makes tRNA unique and how it may benefit rare diseases and other conditions.

“Existing gene therapy approaches can be especially limited when the goal is to fix large structural proteins. So, the size of the gene matters. The size of the protein matters, the number of unique mutations make it really difficult to do at scale,” Dr. Gardiner explains. “So Tevard’s tRNA therapeutic approach offers…a scalable platform that's capable of correcting disease-causing premature termination codons. These are also called nonsense mutations. Essentially, what happens in normal protein production is you reach the end of the protein, you have this thing called the ribosomal assembly, and it drives along, it reaches what's called a stop codon, and then the protein is released and can work effectively.

Dr. Gardiner further explains that, in the case of the company’s suppressor tRNA technology, team members can go in and fix these nonsense mutations at any point throughout the protein and create a full-length version of that protein. Previously, this action may have only been possible to due to the large size of the genetic material, in which a researcher would only be able to make part of the protein and repair that part, she continues. However, with tRNA suppressor technology, the researcher can go in and fix just those specific codons—those nonsense mutation codons—and create a full length protein that's completely functional, that can interact with other proteins, and that can provide contractility. This can result in a fully functional protein that can promote muscular repair, Dr. Gardiner notes.

https://www.fda.gov/patients/rare-diseases-fda

Tevard. Tevard Biosciences Presents Data Showing Compelling Full-Length Protein Rescue with tRNA-based Treatment for Duchenne Muscular Dystrophy and Dilated Cardiomyopathy at the 2025 FEBS Special Meeting. Press Release. Sept. 29, 2025.

Elisabeth Gardiner, PhD, CSO, Tevard Biosciences

Dr. Elisabeth Gardiner is chief scientific officer at Tevard Biosciences, joining the leadership team in 2025 to spearhead the company’s pioneering tRNA-based gene therapies for rare diseases with high unmet need. With more than 25 years in the biopharmaceutical industry, she has directed and managed drug discovery and development efforts yielding 11 investigational new drug filings, four Phase I/II trial candidates, and one Phase III candidate. Prior to Tevard, Dr. Gardiner held senior R&D leadership roles at Tactile Therapeutics, Alterome Therapeutics, Aravive, and Kinnate Biopharma, where she directed multidisciplinary teams and advanced programs from discovery to clinical-stage development across neurology, oncology, and rare disease. Dr. Gardiner gained experience in tRNA biology while working at aTyr Pharma and at Scripps Research Institute in the lab of Paul Schimmel. Dr. Gardiner’s commitment to the ethical development of effective and accessible medicines is her key focus in life. In addition to her professional work, Dr. Gardiner acts as a patient advocate in the rare disease and oncology space. She earned her PhD from the University of Wisconsin-Madison and holds a B.S. and an M.S. from Texas A&M University.

Videos

Elisabeth Gardiner, CSO of Tevard Biosciences, discusses how transfer RNA is impacting the development of gene therapies for diseases with unmet needs.

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Medable announced on Jan 6, 2026 that it will be presenting its trial master file (TMF) AI agent, which automates labor-intensive processes involved in trial document management, at the 

 in San Francisco, which is being held from January 12–15. According to the company, the TMF Agency reduces manual effort in document management, and therefore, improves quality and consistency.

Based on Medable’s Agent Studio, an agentic AI platform for clinical development, the TMF Agency “orchestrates TMF workflows end to end” by autonomously intaking documents, classifying files, and extracting metadata (1). These data are prepared for human review before a one-click submission into commonly used eTMF systems (e.g., Veeva Vault, Wingspan, and OpenText). The AI agent is system-agnostic and can be integrated into existing workflows. Human-in-the-loop checkpoints allow for validation, quality control, and audit traceability.

"We are applying AI to improve quality and remove bottlenecks in clinical development to enable highly skilled progressions to perform more strategic work to move on the needle on clinical development" said Dr. Michelle Longmire, Medable CEO and Co-Founder, in a press release (1). "Medable agents make trial systems work smarter by removing the friction between document management and regulatory submission. We will continue building agents that address the most difficult clinical trial challenges while also enabling customers to build their own with Studio."

How varied is AI’s use in pharma development?

The use of AI and machine learning is rapidly growing in the pharmaceutical industry. Even staff at regulatory agencies are utilizing this new technology. In December 2025, 

 it was providing its staff with agentic AI capabilities to create complex AI workflows, harness AI models, and assist staff with multi-step tasks (2). The agency has been committed to embedding AI in its workflow to create more efficient operations.

“FDA's talented reviewers have been creative and proactive in deploying AI capabilities— agentic AI will give them a powerful tool to streamline their work and help them ensure the safety and efficacy of regulated products,” Chief AI Officer Jeremy Walsh stated in a press release (2).

, a generative AI tool for staff use, which was built within a high-security GovCloud environment for staff to securely access internal agency documents (3). FDA is using the large language model tool to accelerate clinical protocol reviews, scientific evaluations, and identify inspection targets.

“Following a very successful pilot program with FDA’s scientific reviewers, I set an aggressive timeline to scale AI agency-wide by June 30,” Makary said in a press release at the time (3). “Today’s rollout of Elsa is ahead of schedule and under budget, thanks to the collaboration of our in-house experts across the centers.”

Medable. Medable Launches New AI Agent for Automating Clinical Trial Document Management; Presenting at JPM Health. Press Release. Jan. 6, 2026.

FDA. FDA Expands Artificial Intelligence Capabilities with Agentic AI Deployment. Press Release. Dec. 1, 2025. 

https://www.fda.gov/news-events/press-announcements/fda-expands-artificial-intelligence-capabilities-agentic-ai-deployment

FDA. FDA Launches Agency-Wide AI Tool to Optimize Performance for the American People. Press Release. June 2, 2025. 

https://www.fda.gov/news-events/press-announcements/fda-launches-agency-wide-ai-tool-optimize-performance-american-people

Articles

The company’s new AI agent automates labor-intensive processes involved in trial document management.

Medable Presents AI Agent for Automating Trial Master File Processes at JP Morgan Healthcare Conference

Leukemia Awareness: Photomicrograph of bone marrow aspirate showing myeloblasts of acute myeloid leukemia (AML), a cancer of white blood cells. | Image Credit: ©David A Litman -stock.adobe.com

Amgen announced on Jan. 6, 2026 that it has acquired Dark Blue Therapeutics, a biotechnology company located in the United Kingdom (1). The transaction, which is valued at up to $840 million, adds an investigational small molecule to Amgen’s portfolio that targets and degrades two proteins (MLLT1/3) that drive specific types of acute myeloid leukemia (AML). According to the company, anti-cancer activity and mechanistic differentiation have been shown by preclinical data in leukemia models, suggesting single-agent and combination use may be used to overcome treatment resistance and increase remission durability (1).

"Acute myeloid leukemia remains one of the most difficult cancers to treat, and we see an urgent need for new mechanisms capable of changing the trajectory of this disease," said Jay Bradner, M.D., executive vice president of Research and Development at Amgen, in a press release (1). "This acquisition complements and extends our research in targeted protein degradation and leukemia therapeutics, advancing our strategy to invest early in rising medicines for novel therapeutic targets. The adjacency of this program to our considered expertise in cancer biology will propel MLLT1/3-targeting medicines to clinical investigation for patients facing the challenging diagnosis of AML."

What business actions has Amgen made recently?

On Dec. 19, 2025, Amgen announced it was committed to reduce the cost of drugs while continuing to innovate in response to the Trump Administration’s Most Favored Nation pricing requests (2). The company stated it was expanding its direct-to-patient program, AmgenNow, to include Aimovig (erenumab-aooe) and Amjevita (adalimumab-atto) at discounted monthly prices. Amgen launced the program in October 2025 with Repatha® (evolocumab), which it made available at 60% below the current US list price (2).

According to the company, its investments in US manufacturing, up to $2.5 billion in 2025, allowed it to gain relief from industry-specific tariffs for three years. The company is investing 

What other advancements have been made in the treatment of AML?

 showed the efficacy of a chimeric antigen receptor T cell (CAR-T) therapy developed by 

, GYA-01, against AML and T-cell acute lymphoblastic leukemia (T-ALL) (3). According to Gyala Therapeutics, the published study is the first that presents preclinical data with CAR-T cells targeting the CD84 protein, highlighting that protein as a broad hematological target that may expand CAR-T therapy opportunities beyond B-cell malignancies.

According to Gyala, many patients with AML achieve remission with chemotherapy at first; however, almost half of patients relapse following first-line treatment (2). With T-ALL, more than 90% of pediatric patients survive with chemotherapy, but long-term survival in adults is much lower, at approximately 40% (3).

“The preclinical evidence obtained confirms that GYA-01 is a highly promising CAR-T therapy, supports CD84 as a pan-hematological target, and establishes a solid foundation for advancing to clinical trials in patients with AML and T-ALL,” Nela Klein-Gonzalez, MD, PhD, medical director of Gyala Therapeutics and author of the 

 article, said in the press release (3). “In these indications, the main challenge for CAR-T therapies is the lack of specific tumor targets.”

Amgen. Amgen Acquires Dark Blue Therapeutics, Bolstering Oncology Pipeline. Press Release. Jan. 6, 2026. 

https://www.amgen.com/newsroom/press-releases/2026/01/amgen-acquires-dark-blue-therapeutics-bolstering-oncology-pipeline

Amgen. Amgen Takes Action with the U.S. Government to Lower the Cost of Medicines for American Patients. Press Release. Dec. 19, 2025. 

https://www.amgen.com/newsroom/press-releases/2025/12/amgen-takes-action-with-the-u-s--government-to-lower-the-cost-of-medicines-for-american-patients

Gyala Therapeutics Unveils First-in-Class CAR-T Therapy Targeting CD84 Showing Strong Preclinical Efficacy in Hard-to-Treat Leukemias.

Amgen has acquired Dark Blue Therapeutics for up to $840 million, enhancing its oncology portfolio with a small molecule targeting proteins MLLT1/3 in acute myeloid leukemia (AML). This acquisition aligns with Amgen's strategy to invest in novel therapeutic targets and expand its expertise in cancer biology. Additionally, Amgen is committed to reducing drug costs, expanding its AmgenNow program, and investing in US manufacturing. Meanwhile, Gyala Therapeutics has shown promising preclinical results for a CAR-T therapy targeting CD84, potentially expanding treatment options for AML and T-ALL.

The move brings Amgen first-in-class, small molecule-targeted protein degraders for oncology. 

Amgen Acquires UK Biotech Dark Blue Therapeutics

Industry Outlook 2026: Trends Transitioning from 2025 into 2026 

AI, the Internet of Things, digitalization, and other technologies became standard practice in 2025 for many pharmaceutical companies. Laine Mello, director of marketing at Ecolab Bioprocessing, sees the implementation of these tools as a significant trend in 2025 that will most likely continue into 2026. “The competitive pressure really became undeniable,” Mello explains. “And if you couldn't monitor processes in real time or predict issues before they happened, you were falling behind.”

The development of complex biologics and other novel treatments, which spurred investments and innovations in 2025, will continue to impact the industry. “These complex molecules are really reshaping how the industry thinks about manufacturing flexibility and purification,” Mello says. “The old playbook doesn't work anymore when you're dealing with products like this, sophisticated such as biologics and cell and gene therapies.”

Mello explains that these trends tie into things like sustainability and cost of goods reduction. “Cost of goods reduction is really the defining priority in 26. Pricing pressures are intensifying, particularly for complex biologics and advanced therapies. You know, manufacturers really need to squeeze more production or more productivity out of every single process and every single step that they have so continuous and intensified bioprocessing will help move from being, you know, innovated to becoming an actually standard practice with more adoption of things like modular facilities and multi column chromatography, helping deliver that flexibility without even sacrificing efficiency,” Mello stresses. “The other thing we're seeing into 2026 is this driver that will be solving the technical challenges around complex molecules, so pipelines are filled with high concentration biologics and next gen antibodies that really push the limits of current manufacturing processes … So, stability, aggregation, purification, bottlenecks.”

Laine Mello, director of marketing at Ecolab Bioprocessing, reflects on how the use of digital technologies expanded in the pharma industry in 2025 and how new complex molecules will continue to drive innovation in 2026.

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On Jan. 6, 2026, Chromatin Bioscience, Mediphage Bioceuticals, and Entos Pharmaceuticals jointly announced that the collaboration between the companies on a genetic medicines project will be supported by UKRI Innovate UK and the 

National Research Council of Canada Industrial Research Assistance Program

 (NRC IRAP) collaborative innovation program (1).

According to the companies, UKRI Innovate UK and NRC IRAP will help the companies bring three synergistic technologies together and will foster international collaboration between the United Kingdom and Canada by providing advisory services and funding for the partnership (1). The project will combine the Chromatin Bio chromatinLENS platform for synthetic promoters, the Mediphage ministring DNA (msDNA) platform technology, and the Entos Fusogenix PLV delivery platform to develop cell-type selective, durable DNA-based gene expression cargo with safe and effective, intracellular fusion-based delivery that could be applied to treat a variety of genetic and rare diseases.

Chromatin Biosciences’ platform, chromatinLENS, enables precise, cell-type-selective, and durable gene expression, according to the company (1). Mediphage’s linear, covalently closed, high-fidelity, and backbone-free msDNA, is produced using a proprietary and scalable 

-based manufacturing process and can be used in non-viral gene therapy, 

 gene editing. Entos’ Fusogenix PLV drug delivery system is formulated with FAST proteins to enable the delivery of nucleic acid to target cells through direct fusion. In April 2025, Entos entered into a research collaboration with Norway-based Circio Holding that included the joint development and 

delivery testing of Circio’s circular RNA expression vectors with the Fusogenix PLV (2).

"This collaboration brings together three complementary technologies to address one of the biggest challenges in genetic medicine: delivering safe, durable, and cell-type-selective gene expression," said Michael Roberts, founder and CEO of Chromatin Bioscience, in a press release (1). "By combining our chromatinLENS platform for synthetic promoters with Mediphage's msDNA technology and Entos' Fusogenix PLV delivery system, we're taking a major step toward creating more precise and effective treatments for patients."

"Gene therapy needs novel modalities that are safe, redosable, precise, and effective," said Dr. Nafiseh Nafissi, CTO of Mediphage, in the release (1). "By combining advanced AI-driven sequence design from Chromatin Bio with our immune-compatible and durable msDNA platform, and next-generation targeted delivery systems from Entos Pharmaceuticals, we are establishing a strong foundation for the next generation of safe, precise, and scalable genetic medicines. We are grateful to receive this support from Innovate UK and NRC IRAP. This support represents an opportunity to advance Mediphage's msDNA technology. We remain committed to advancing innovative genetic medicine approaches through collaboration and collective effort that can deliver life-changing genetic medicine treatments for patients in need."

"We deeply appreciate this support from Innovate UK and NRC IRAP to advance the use of our Fusogenix PLV platform in projects aimed to improve treatments for rare genetic disorders," said John Lewis, CEO of Entos Pharmaceuticals, in the release. "There is a crucial need to address these disorders, and the potential for novel genetic medicine approaches that combine cutting-edge technologies like chromatinLENS, msDNA, and Fusogenix PLV to create life-changing treatments for patients and their families is huge."

PR Newswire. Chromatin Bioscience, Mediphage Bioceuticals, and Entos Pharmaceuticals, collaborate on Genetic Medicines Project with support from UKRI Innovate UK and Canadian R&D Funding. Press Release. Jan. 6, 2026.

Chromatin Bioscience, Mediphage Bioceuticals, and Entos Pharmaceuticals have announced a collaboration on a genetic medicines project, supported by UKRI Innovate UK and NRC IRAP. This partnership aims to integrate three advanced technologies: Chromatin Bio's chromatinLENS platform for synthetic promoters, Mediphage's msDNA technology, and Entos' Fusogenix PLV delivery system. The goal is to develop cell-type selective, durable DNA-based gene expression therapies for genetic and rare diseases. The collaboration emphasizes the importance of safe, precise, and scalable genetic medicine solutions, with international support fostering innovation.

Chromatin Bioscience, Mediphage Bioceuticals, and Entos Pharmaceuticals will receive advisory services and funding from UKRI Innovate UK and Canada’s NRC IRAP to develop next-generation genetic medicines.

Genetic Medicines Project Gets Funding from UKRI Innovate UK and Canadian NRC IRAP

Making Smart Decisions and Investments in Trained Staff Key to 2026 Success

Tariffs imposed by the United States were a complicated issue that impacted the pharmaceutical industry in 2025, and Raj Puri, chief commercial officer at Argonaut Manufacturing, anticipates that they will continue to be an issue in 2026. Choosing to invest large amounts of money on a new manufacturing site, and/or upgrading an existing facility, becomes more complicated in an unstable geopolitical market. And this complexity can impact time to market.

“I can tell you from personal experience, even having a couple of big projects going on in the background while we're trying to do an expansion has a major impact on timelines,” Puri explains. He points out current major investments announced by the industry in 2025 as an example. “If these investments are implemented, and there's, I think, still a question of whether or not they actually get realized. If they are implemented though, the cost and timelines to build these facilities and acquire the equipment needed to actually make products is going to be extended significantly, and the cost will go up significantly, as well is my expectation. The supply and demand curve is going to be completely disrupted.”

New investment in facilities also brings a need for people with the right experience and knowledge to work with the companies that are expanding their footprint. Finding and retaining the right people to create a long-term, sustainable advantage in the pharma manufacturing market is essential for a contract development and manufacturing organization (CDMO) like Argonaut, Puri stresses.

“Anybody that has sufficient money and time can build a beautiful facility, put in great equipment. What they can't replicate, though, is the staff that run the equipment and the processes that we use to execute our manufacturing. As a midsize CDMO,” Puri says, “we can't always compete with the bigger players in terms of compensation, but what we can offer to our staff is a very dynamic work environment where they have the opportunity to work in different functional areas and progress their careers. For the staff, it's very engaging, because they get to see a lot of different areas and wear many different hats, but as an organization, we also take great benefit, because now we have people that are working together in different functional activities who actually truly understand what it is like to be in the other person's shoes. As an example, somebody that's in operations that has been in quality assurance understands why quality assurance demands certain things. And ideally, somebody that's in quality assurance has been in operations, and they understand some of the challenges on the floor from a manufacturing perspective.”

Raj Puri, chief commercial officer at Argonaut Manufacturing, talks about how navigating the pressure to respond to tariffs with investments and keeping a robust and qualified staff are key moves in the new year.