We are thrilled to share some groundbreaking developments in the field of precision medicine with you. Two promising new drug candidates, Leqembi and Donanemab, have shown great potential in slowing the progression of Alzheimer’s disease, marking a significant step forward in tackling this challenging condition.

But this is just the beginning, and we must continue advancing the drug pipeline to develop the next class of drugs centered around the biology of aging to ultimately stop Alzheimer’s and related diseases in their tracks. The goal should be to address the various underlying pathologies of the disease through a precision medicine approach. Precision medicine not only benefits the patient but also the overall health systems. In addition, a precision approach to medicine is in the long run expected to help control the overall cost of health care.

A recently published PMC report showed that newly approved precision medicines in 2022 include 12 new entities and 5 newly approved gene and cell-based therapies bringing the total to 37 new molecular entities approved by the FDA’s CDER. Furthermore, 12 in vitro diagnostic testing applications that underpin precision medicine strategies were also approved or cleared for several new or expanded indications which help inform targeted treatment decisions to improve drug safety and efficacy. Some report highlights include:

• The 12 new entities represent ~34% of all newly approved therapeutic molecular entities, accounting now for at least a quarter of new drug approvals for each of the last eight years.

• The five new gene or cell-based therapies include treatments for beta thalassemia, hemophilia B, and cerebral adrenoleukodystrophy, resulting in a total of 13 FDA-approved gene or cell-based therapies to-date.

• Of the 17 new precision medicines 53% (N=9) are for cancer, 35% (N=6) for rare diseases, and 12% (N=2) for other disease treatments.

• 12 new in vitro diagnostic testing applications include three new blood-based biomarker tests to guide personalized oncology treatment decisions and several newly approved indications that will advance tumor-agnostic testing paradigms.

These new developments illustrate the importance of ensuring that our health systems are aligned with the latest science and technology so that we can deliver unprecedented benefits to both patients and health systems.

PMWC January 24-26, 2024 Silicon Valley, which will take place at the Santa Clara Convention Center, has designed the Emerging Therapeutics Track to cover critical aspects of new precision medicine developments. The focus will be on:

  • Gene & Cell Therapies for Rare Disease chaired by Yael Weiss (Mahzi Therapeutics) and Peter Marks (FDA)
  • Oncology Focused Gene Modified Cell Therapies chaired by Sharon Benzeno (Adaptive Biotechnologies),
  • Pharmacogenomics (PGx) chaired by Kelly Caudle (St. Jude Children’s Hospital)

These tracks will deep-dive into…

• why we must focus on the understanding of target/pathway biology to stop a disease in its track,

• why we must address underlying pathology drivers of a disease through a precision medicine approach, and

• why we need precision biomarkers to gain a deeper understanding of pharmacological response and possible drug-drug interactions, to decide on a treatment strategy, and to monitor drug response.

We are as excited as you to witness how recent progress in precision medicine and diagnostics continues to shape our approach to disease prevention and intervention and invite you all to join the next Precision Medicine World Conference, PMWC January 24-26, 2024, that brings stakeholders from across the industry together to engage in this important dialogue and influence the trajectory of modern health care.

Take advantage of the expiring early registration discount and secure your spot today.


Tal Behar

President & Co-founder, PMWC

PMWC 2024 Silicon Valley – January 24-26, 2024

The Precision Medicine World Conference (PMWC)


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