We are honored to announce Dr. James M. Wilson, a pioneer in gene therapy, as our distinguished Pioneer Honoree at PMWC February 5-7, 2025 Silicon Valley, where we will spotlight the transformative effect of cell and gene therapies in addressing complex medical challenges.

In an exclusive interview with PMWC, Dr. Wilson shared insights into his groundbreaking work, which “has significantly impacted the treatment of rare diseases, offering hope for millions,” as he emphasized. His innovations have overcome critical barriers in gene therapy, particularly addressing the body’s immune response to viral vectors, thus paving the way for safer and more effective treatments.

Looking Ahead: Gene Therapy Beyond Rare Diseases
Dr. Wilson’s vision for gene therapy is optimistic. He envisions viral vector-based therapies for treating more common diseases. However, Dr. Wilson emphasized the crucial need to “align viral vector attributes with therapeutic needs.” He believes ongoing research holds the key to unlocking this platform’s potential beyond rare diseases.

Beyond the Hype: Safety First in Gene Therapy
See Dr. Wilson’s full insights and perspectives in the interview listed below my signature.

Equally pivotal is Dr. Carolyn Bertozzi of Stanford University, recipient of the PMWC Luminary Award. Her pioneering advancements in bioorthogonal chemistry have bridged the gap between basic science and clinical application, shaping the landscape of precision medicine.

The Cell and Gene Therapies track at PMWC 2025, chaired by industry leaders Yael Weiss of Mahzi Therapeutics and Morten Sogaard of Astellas, will delve into critical topics such as next-generation delivery systems, harnessing epigenetics in rare disease treatment, and navigating regulatory landscapes.

Don’t miss your opportunity to network with visionaries like Dr. Wilson and Dr. Bertozzi, as well as other pioneers shaping the future of gene therapy. Secure your place today to participate in this dynamic exchange of ideas at PMWC 2025, Feb 5-7.

See The Gene & Cell Therapies in Rare Disease’s Track Program below:

      PMWC Award Ceremony:
            ● Pioneer Honoree: James M. Wilson, University of Pennsylvania
            ● Luminary Honoree: Carolyn Bertozzi, Stanford

      Keynotes:
            ● Bioorthogonal Chemistry, from Basic Science to Clinical Translation
                  o Carolyn Bertozzi, Stanford
            ● The Promise of Genetic Medicines Unless You’re Too Poor or Too Rare
                  o James M. Wilson, University of Pennsylvania

      Session Schedule:
            Reimbursement for Genetic Medicines in Rare Diseases
                  ● Chair: James M. Wilson, University of Pennsylvania
                  ● Speakers:
                        o Erin Satterwhite, Optum Specialty Pharmacy

            Next-Generation Delivery Systems for Gene Therapies
                  ● Chair: Morten Sogaard, Astellas
                  ● Speakers:
                        o Mathieu Nonnenmacher, Voyager Therapeutics
                        o Kevin Friedman, Kelonia Therapeutics
                        o Haig Aghajanian, Capstan Therapeutics
                        o James M. Wilson, University of Pennsylvania

            Beyond the Genome: Harnessing Epigenetics in Rare Disease Treatment
                  ● Chair: Charles Gersbach, Duke
                  ● Speakers:
                        o Prashant Mali, UCSD
                        o Nadav Ahituv, UCSF
                        o Blythe Sather, Tune Therapeutics
                        o Fyodor D Urnov, Berkeley University

            Navigating the Regulatory Landscape for Gene and Cell Therapies
                  ● Chair: Yael Weiss, Mahzi Therapeutics
                  ● Speakers:
                        o Courtney Silverthorn, Bespoke Gene Therapy Consortium (BGTC)
                        o Peter Marks, FDA

            How We Use AI and ML to Develop Gene Therapies of the Future
                  ● Speakers: TBA

We look forward to seeing you at PMWC Feb. 5-7, 2025. Don’t miss this opportunity to engage with industry leaders and gain valuable insights into the future of precision medicine.

Register Now & SAVE HERE

Sincerely,

Tal Behar

President & Co-founder, PMWC
PMWC 2025 Silicon Valley – February 5-7, 2025

The Precision Medicine World Conference (PMWC)


PMWC’s Interview with Dr. Wilson continued:

Looking Ahead: Gene Therapy Beyond Rare Diseases
Dr. Wilson’s vision for gene therapy is optimistic. He envisions viral vector-based therapies for treating more common diseases. However, Dr. Wilson emphasized the crucial need to “align viral vector attributes with therapeutic needs.” He believes ongoing research holds the key to unlocking this platform’s potential beyond rare diseases.

Beyond the Hype: Safety First in Gene Therapy
Safety remains paramount. Dr. Wilson highlighted the importance of understanding potential risks and improving safety profiles. He stated, “Targeted delivery and the development of gene regulation mechanisms will be crucial for expanding this therapy to a wider range of conditions.” – like a gene switch – for more precise control. This innovation holds immense promise for expanding the reach of gene therapy while prioritizing safety.

A World Beyond AAV: Exploring New Delivery Technologies
The conversation delved into the future of delivery technologies beyond Adeno-Associated Virus (AAV). While Dr. Wilson acknowledges AAV as the current leader for gene replacement therapies due to its safety profile, he sees promise in non-viral delivery systems for gene editing applications. As Dr. Wilson explained, “These systems don’t require long-term expression of the therapeutic gene.” Additionally, advancements in engineering AAV capsids offer exciting possibilities for improved targeting and reduced dosage, making AAV an even more powerful tool.

Gene Editing and Gene Therapy: A Powerful Duo
Dr. Wilson shed light on how recent advances in gene editing will work alongside gene replacement therapies. He highlighted the distinct needs of each approach. “Gene editing holds significant potential for treating liver diseases due to the efficient delivery of non-viral systems,” Dr. Wilson explained. However, for diseases affecting other organs, AAV-based gene therapy remains the preferred method.
Furthermore, Dr. Wilson suggests that advancements in gene editing could eliminate the need for persistent viral vector expression. This shift would focus on integrating the therapeutic gene directly into the patient’s chromosomes.

The Potential of Other Viral-Based Approaches
Dr. Wilson acknowledged the renewed interest in retroviruses and the development of virus-like particles for gene delivery. While he acknowledged the success of herpes viruses in specific applications like skin diseases, he expressed some skepticism about their broader applicability. He also discussed past challenges encountered with lentiviruses and retroviruses, such as toxicity and manufacturing difficulties, suggesting these issues might resurface with these new technologies.

Join us in Celebrating Dr. Wilson’s Achievements at the PMWC conference!
Register Early & SAVE HERE

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