Susan Haigney

Personalized Medicine vs. Precision Medicine

, the concept of personalized medicine shifted from a general idea that all people should have personalized care based on their circumstances to how biology can be used to design a drug that works for a specific disease at a specific time.

“Precision medicine really is understanding the data, the biology that drives disease, the initial focus on this was really around in oncology, and if we look at the advancements in oncology over the past 15 years, they've been profound with the use of genetics and genomics to identify biological factors in patients tumors that have driven the development of targeted therapies and immunotherapies that have really transformed the treatment of patients with cancer,” Dr. Carter explains. “Now even deeper information on genetics has allowed us to develop gene therapies and other treatments that are transformative for rare diseases, neurodegenerative diseases, and autoimmune disease.”

Patient access to precision medicine is one of the biggest challenges to their success, according to Dr. Carter. “There's a tremendous amount of progress, but the drugs are, while they're fantastic drugs, they're incredibly expensive, they're quite esoteric in terms of how to use them and for which patients, and we need to really understand,” Dr. Carter says. “And this was where we get really into the precision medicine aspect. We need to both understand the biology of the patient’s disease and how to get patients access to the right drugs at the right time in a financially sustainable way for the system.”

Dr. Carter will be discussing how precision-guided platforms can help pharmaceutical companies manage the costs of developing precision medicines, such as cell and gene therapies, and therefore improve patient access, at the 

 in San Francisco, which is being held from January 12–15.

Dr. Jennifer Levin Carter, founder and CEO, Medzown

Jennifer Levin Carter, MD, MPH, MBA is Founder & CEO of Medzown. Dr. Carter is a globally recognized leader in precision medicine, with a proven track record of building transformative companies at the intersection of data, technology, and patient care. She was the Founder and President of N-of-One, Inc., where she served as CEO from 2008–2012 and later as President and Chief Medical Officer until its acquisition by Qiagen (Market Cap $8B) in 2019. At N-of-One, Dr. Carter led the development of award-winning solutions that continue to deliver novel treatment strategies to hundreds of thousands of cancer patients worldwide.

In 2018, Dr. Carter founded a novel clinical trials company focused on reimagining trial design and access. She served as CEO until its pre-launch acquisition by Integral Health (now Valo Health) in March 2019. These experiences cemented her reputation as an innovator in precision medicine, clinical trial delivery, and patient-centered care models.

Following these ventures, Dr. Carter became a Managing Partner and Venture Partner at Sandbox Industries and Blue Venture Fund, a unique collaboration between BCBS companies, BCBSA, and Sandbox. It was in this role—working closely with BCBS executives—that she gained a deep understanding of the challenges associated with the rising costs of cell and gene therapy. This insight inspired her to launch Medzown, where she continues to address those challenges by building scalable, technology-enabled solutions to improve patient access to advanced therapies.

Dr. Carter is also a seasoned healthcare executive, investor, and board member with expertise spanning healthcare IT and services, digital health and machine learning, genomics, drug development, and the creation of next-generation platforms to improve therapeutic access. She currently serves on the Board of Directors of Synteny.ai, TempraMed, Inc., and Target Cancer Foundation, and is a strategic advisor to numerous venture-backed healthcare and life sciences companies.

Previously, she has held board roles at Oncocyte (NASDAQ: OCX), XSphera Biosciences (Co-Founder), CareMax (NASDAQ: CMAX), DFP Healthcare Acquisitions Corp (NASDAQ: DFPH), HouseWorks, eCaring, and others. She also serves on the Director’s Advisory Board at Smilow New Haven Cancer Center at Yale University, the Board of the Blavatnik Fund at Yale University, and the MedExec Women’s Advisory Board.

Dr. Carter earned a BS in Molecular Biophysics and Biochemistry (Phi Beta Kappa, Summa Cum Laude with distinction) from Yale University, an MD from Harvard Medical School, an MPH from Harvard School of Public Health, and an MBA from the Sloan School of Management at MIT. She is a member of the American Association of Corporate Directors (Advanced Professional Director Certification), National Association of Corporate Directors, Women Corporate Directors, Women Business Leaders of the U.S. Health Care Industry Foundation, and Women in Bio.

Videos

Dr. Jennifer Levin Carter, founder and CEO of Medzown, discusses what makes precision medicine different from personalized medicine and what how challenges faced in this evolving field can be overcome. 

The Benefits of an Immune-Directed ADC Strategy 

Traditional autoimmune and inflammatory treatments have historically relied on what experts describe as a "carpet bombing strategy," which provides efficacy but causes significant collateral damage to healthy tissue. According to 

Arthur Tzianabos, CEO, Lifordi Immunotherapeutics

, while steroids are a $10 billion market, patients often cannot tolerate them long-term because they "hit not only the disease, but also a lot of other normal, healthy tissue," leading to numerous side effects. This has prompted an industry-wide shift toward "precise targeting of the immune system" through the use of antibody-drug conjugates (ADCs).

VISTA, according to Tzianabos, is a cell surface protein found primarily in the lymphoid and myeloid systems. VISTA is a particularly effective target because it "very rapidly internalizes inside the cell and drops off whatever it’s bound to" before recycling back to the surface. By using VISTA as a gateway, Lifordi’s lead program delivers a "very powerful steroid" directly to the immune cells. This targeted delivery is designed to "avoid the systemic toxicities" of widespread steroid use while maintaining high potency.

The company is currently in a Phase I trial for a rheumatoid arthritis treatment and has begun receiving data. Their progress is supported by 

, including a $41 million raise with participation from Sanofi Ventures, which followed a $70 million Series A (1). This growth occurs amid a broader biotech upswing in late 2025 and early 2026, a period marked by rising valuations and increased M&A activity.

, which is being held in San Francisco from January 12–15, to discuss its ADC strategy and meet with leaders in the industry (2). Tzianabos notes that the environment in the industry has shifted toward a "period of optimism" that was absent in previous years, positioning the company well as they share progress with investors and pharmaceutical partners.

Lifordi Immunotherapeutics. Lifordi Immunotherapeutics Secures Strategic Investment from Sanofi Ventures and Additional Capital from Existing Investors. Press Release. Nov. 18, 2025. 

https://lifordi.reportablenews.com/pr/lifordi-immunotherapeutics-secures-strategic-investment-from-sanofi-ventures-and-additional-capital-from-existing-investors

JP Morgan. JPM 2026. Accessed Jan 9, 2025. 

Arthur Tzianabos, PhD, president and chief executive officer, Lifordi Immunotherapeutics

Arthur O. Tzianabos, PhD, is president and CEO of Lifordi Immunotherapeutics and a Venture Partner at 5AM Ventures. He has more than 30 years of experience in the biotechnology industry and academia leading teams in the discovery, development, clinical translation and commercialization of new treatments for immunology and rare diseases.

Dr. Tzianabos served as President and CEO of Homology Medicines and later became Chair of the Board prior to its merger with Q32 Bio. Dr. Tzianabos is currently a member of the Board of Directors at Q32 Bio and Chair of the Board of Directors at Stoke Therapeutics. Previously, he was Chair of the Board for Akouos Therapeutics (acquired by Eli Lilly in 2022). He also serves on the Development Board for the University of New Hampshire’s College of Life Sciences and Agriculture. Earlier in his career, Dr. Tzianabos had an active role in the development, approval, and commercial launch of multiple therapies for patients with rare genetic disorders at Shire Pharmaceuticals. He also worked closely with the Business Development and Corporate Venture functions to invest in and acquire new companies and technologies to build Shire’s pipeline.

Dr. Tzianabos is trained as an immunologist and was an Associate Professor of Medicine at Harvard Medical School where he maintained laboratories at the Channing Laboratory, Brigham and Women’s Hospital and the Department of Microbiology and Molecular Genetics at Harvard Medical School. He has published more than 80 scientific papers, reviews, book chapters and patents. Dr. Tzianabos holds a PhD in Microbiology from the University of New Hampshire and a B.S. in Biology from Boston College.

Arthur Tzianabos, CEO, Lifordi Immunotherapeutics, spoke with PharmTech about the company’s immune-directed antibody-drug conjugate (ADC) strategy, the benefits of VISTA-targeted ADCs in drug delivery, and Lifordi’s plans at the JP Morgan Healthcare Conference. 

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.

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

Ai generate concept with icons. AI Powered Search Engines artificial intelligence technology drive digital marketing, workflow, dicision and seo automation. Vector illustration web banner. | Image Credit: ©Deemerwha -stock.adobe.com

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.