Collaboration Corner
More Than Just a Meeting
Gene Therapy for Rare Disorders is the flagship for-industry gene therapy meeting, bringing together 200+ senior gene therapy decision makers, likeminded in the challenges they face and drivers they have for attending this summit.
Whether attending as a speaker, delegate, or partner, the series has a long-standing track record of facilitating new connections and collaborations beyond just one week in Boston. Structured networking sessions, facilitated meetings, and informal conversations outside the scientific sessions can all further your team’s work and individual standing in the gene therapy space by:
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- Accelerating personal network growth
- Enabling future job opportunities
- Meeting prospective clients
- Establishing relationships with a service provider
- Laying the groundwork for future inter-company deals
This page is dedicated to showcasing case studies from previous Gene Therapy for Rare Disorders attendees, highlighting how the meeting has teed up these connections and collaborations. Take a look at the free-to-access pieces for an idea of the convening power a week in March can hold for your gene therapy work in 2025 and beyond.
Jim Wang
While in his capacity as Chief Regulatory Officer at Adverum Biotechnologies, Jim Wang was a speaker at Gene Therapy for Rare Disorders 2023. His contributions focused on differentiating key strategic differences between collaborating with the FDA and EMA, part of which included participating in a panel discussion with representatives from the FDA and PMDA.
Vibha Jawa
As Executive Director, Head of the Biotherapeutics Bioanalysis in Nonclinical Disposition and Bioanalysis at Bristol Myers Squibb, Vibha joined us at Gene Therapy for Rare Disorders 2022, speaking to her work developing immunogenicity assays for gene therapy products.
Hera Lichtenbeld
Hera Lichtenbeld, Assistant Director at University of Florida, Tech Licensing, joined us as a delegate for both the 2022 and 2023 Gene Therapy for Rare Disorders meetings
Beacon H1 2023 Landscape Report
This review provides insight into gene therapy’s incredible advancements and potential, utilizing data from Beacon Gene Therapy. It covers a comprehensive analysis of the drug and trial landscape, comparing data from December 2022 to June 2023, and includes all regulatory updates made in the first half of 2023. Gain valuable insights for your drug development strategies by downloading the landscape review.
An Exclusive Interview with Dr. Katherine High M.D.
Feb 2023
Ahead of the 2023 meeting, we sat down with none other than Dr Katherine High, MD, a pioneer of the AAV gene therapy field to discuss her views of the field and hopes for the future.
Access the interview transcript to see Dr High benchmark the key challenges facing gene therapy drug developers and researchers, outline her hopes and expectations for the field, and tell us why she was excited to present at the 2023 Gene Therapy for Rare Disorders Summit.
A Deep-Dive into Tenaya Therapeutics’ Plan for 2023
Feb 2023
We sat down with Faraz Ali to discuss Tenaya’s development plans for 2023, his hopes and expectations for the future of the gene therapy field, and why he values the “convening power” of the Gene Therapy for Rare Disorders Summit Series.
AAV Durability: The Road to a One-Shot Cure
Inconsistent long-term clinical data has highlighted the need to better understand the durability of AAV gene therapy.
Hanson Wade Intelligence's Market Research division have classified 121 AAV gene therapy trials into 4 distinct groups based on the target tissue type – liver, muscle, CNS and ocular tissues. This allowed us to summarise available data on clinical durability as well as the key findings we observed across tissues, trials, doses and serotypes.
We hope that this report will provide gene therapy developers with valuable insights on the durability of the current generation of AAV gene therapies.
Gene Therapy Trial Landscape in 2020
Gene Therapy for Rare Disorders Europe Past Presentations
GMO Requirements for Gene Therapies in Europe Latest Developments
Annie Hubert, Senior Director, European Public Policy
The Importance of Strategic Partnerships in Gene Therapy
Dr. Shankar Musunuri, Chairman, CEO & Co Founder, Ocugen, Inc.
