March 19–20, 2025 | Seaport Hotel, Boston, MA
3rd Annual Forum on
Advanced Therapeutics
Regulatory, IP and Funding Strategies for Cell and Gene Therapies, RNA Therapeutics, and Regenerative Medicines
2025 Conference Co-Chairs FDA Keynote USPTO Interview
Derek Johnson Managing Director and General Counsel Portal Innovations
Lulu Wang Head of Intellectual Property Orna Therapeutics
Julie Tierney Deputy Center Director Center for Biologics Evaluation and Research FDA
Andrea Grossman Legal Advisor Office of Patent Legal Administration USPTO
AdaptImmune
Alnylam
Beam Therapeutics
IN-HOUSE INSIGHTS FROM:
Caribou Biosciences
The Broad Institute of MIT
Flagship Pioneering
COCKTAIL SPONSOR:
Novo Nordisk
Nutcracker Therapeutics
Orchard Therapeutics
ACI’s Advanced Therapeutics Forum: Uniting Innovation, Law and Strategy in RNA, Cell and Gene Therapy,
and Regenerative Medicines
American Conference Institute is pleased to present the 3rd Annual Forum on Advanced Therapeutics, where the brightest minds in RNA, cell and gene therapies, and regenerative medicine converge to shape the future of this rapidly evolving field.
Positioned at the critical juncture of innovation, commercialization, and compliance — this premier event is dedicated to unraveling the complex interplay of evolving market trends, dynamic regulatory frameworks, and critical intellectual property strategies.
Join us in Boston on March 19–20 in this collaborative endeavor to drive forward the most groundbreaking developments in advanced therapeutics, marrying scientific discovery with strategic business and legal acumen for global impact.
Attend to engage with leading industry thought leaders and legal pioneers who will illuminate the path from groundbreaking research to global market success. Here, we explore in-depth the nuances of funding methodologies, AI’s transformative role in drug discovery, and the intricacies of navigating the legal, regulatory and IP landscapes
REGISTER NOW!
MEET ACI’s ADVANCED THERAPEUTICS ADVISORY BOARD:
Cristin Berkey VP, IP and Legal Affairs Abata Therapeutics
Elbert Chiang Vice President, Intellectual Property Beam Therapeutics
Forrester Liddle Head of Global Research and Early Development Legal Head of IP and Legal for US R&D Novo Nordisk
Adrian Looney Chief Intellectual Property Counsel Vertex Pharmaceuticals
Sharick Naqi Director & Senior Counsel
Global IP Lead – Gene Therapies Novartis Gene Therapies
David Silverstein Vice President, Intellectual Property Rocket Pharmaceuticals, Inc.
Lulu Wang Head of Intellectual Property Orna Therapeutics
Olga Zimmerman Director, Intellectual Property Caribou Biosciences, Inc.
ESTEEMED CO-CHAIRS
Derek Johnson Managing Director and General Counsel Portal Innovations
Lulu Wang Head of Intellectual Property Orna Therapeutics
DISTINGUISHED FACULTY
Jared Auclair
Vice Provost Research Economic Development Director of Bioinnovation Northeastern University
Elbert Chiang
Vice President, Intellectual Property Beam Therapeutics
Jennifer Chheda Partner
Jones Day
Erin Foley Counsel Foley Hoag LLP
Allison Glasunow Counsel
Perkins Coie
Andrea Grossman Legal Advisor, Office of Patent Legal Administration USPTO
Ryan Hagglund Partner Loeb & Loeb LLP
Christina M. Hartman Senior Director, Government Affairs Orchard Therapeutics
John Haugen
Senior University Counsel Stanford University, Office of the General Counsel
Jae Kim Partner DLA Piper
Forrester Liddle
Head of Global Research and Early Development Legal Head of IP and Legal for US R&D Novo Nordisk
Dan Liu Attorney Loeb & Loeb LLP
Chia-Feng Lu Shareholder
Greenberg Traurig
Bonnie Weiss McLeod Of Counsel Cooley LLP
Deirdre Parsons VP Public Policy and Government Relations Alnylam
Rebecca (Reba) Rabenstein Partner Latham & Watkins LLP
Jocelyn Ram IP Counsel The Broad Institute of MIT
Chuck Sholtz Vice President, Intellectual Property Nutcracker Therapeutics
Laurence Shumway Vice President, Intellectual Property Counsel Flagship Pioneering
Meghann Teague Head of Intellectual Property AdaptImmune
Julie Tierney
Deputy Center Director Center for Biologics Evaluation and Research FDA
Mary Till Of Counsel Finnegan, Henderson, Farabow, Garrett & Dunner, LLP
Rebecca Wood Partner
Sidley Austin LLP (Former Chief Counsel, FDA)
Olga Zimmerman Director, Intellectual Property Caribou Biosciences, Inc.
Day 1
Wednesday, March 19, 2025
8:45 Co-Chairs' Opening Remarks
Derek Johnson Managing Director and General Counsel Portal Innovations
Lulu Wang Head of Intellectual Property Orna Therapeutics
9:00 Charting the Course for Tomorrow’s Advanced Therapeutics: Exploring How Legislation, Policy and Market Dynamics Will Shape Next-Gen Medicines in 2025 and Beyond
Our opening panel will delve into how the latest legislative initiatives and policy changes are influencing strategic decision-making in R&D, investments and market entry for cuttingedge therapies. Additionally, explore how the results of the election may impact the future regulatory landscape for novel and advanced therapeutics and how companies can prepare to ensure both compliance and competitiveness in a rapidly changing environment.
Topics of discussion will include:
Deirdre Parsons VP Public Policy and Government Relations Alnylam
Christina M. Hartman Senior Director, Government Affairs Orchard Therapeutics
• Examining updates in key policies surrounding drug approval pathways, pricing negotiation frameworks, intellectual property, patient access, and post-market surveillance, and their implications for advanced therapies as we enter 2025
• Highlighting recent trends in biopharma, major drug approvals, and global market dynamics to support data-driven decisions and capture growth in emerging markets
• Analyzing the potential impact of election outcomes on healthcare priorities, FDA authority, and innovation funding to help companies adapt proactively
• Understanding how regulatory foresight can guide company strategies for advancing innovative therapies
10:00 FDA Keynote
Julie Tierney Deputy Center Director Center for Biologics Evaluation and Research FDA
10:30 Morning Coffee Break
11:00 Industry Perspectives on Overcoming FDA Hurdles: Insights from Companies and Their Counsel on Tackling Key Regulatory Challenges in Advanced Therapeutics
Following on the comments of Julie Tierney, hear from regulatory counsel who routinely work in the trenches with the FDA on what they perceive to be the biggest FDA regulatory hurdles impacting advanced therapeutics. Explore real-world strategies for managing regulatory uncertainties, accelerating development timelines, and preparing for shifts that could redefine the path to market for advanced treatments.
Rebecca Wood Partner
Sidley Austin LLP (Former Chief Counsel, FDA)
Jae Kim Partner
DLA Piper
11:45 Preparing to Launch: Designing Your Pre-Commercialization Blueprint for Advanced Therapies and Companion Diagnostics
For advanced therapies and companion diagnostics, successful commercialization requires a strategic roadmap that addresses both regulatory requirements and market readiness from early stages. This panel will provide actionable insights into creating a streamlined pre-commercialization plan that aligns regulatory compliance with commercial objectives. Topics of discussion include:
Chia-Feng Lu Shareholder
Greenberg Traurig
• Identifying key considerations for designing a robust regulatory approval plan that aligns with FDA standards for advanced therapies
• Designing effective clinical trials to accelerate timelines
• Coordinating development timelines and regulatory paths for therapies requiring companion diagnostics
• Identifying and mitigating potential regulatory, clinical, and market risks early in the development process
• Optimizing market access:
» Techniques for aligning clinical trial and economic evidence to support payer engagement and ensure smooth market entry
12:45 Networking Luncheon
1:45 Redefining Due Diligence for Future-Forward Treatments: Tailoring Evaluation Strategies for Novel Drug Modalities
As companies pivot toward developing and acquiring advanced therapeutic modalities such as cell and gene therapies, RNA therapeutics, and regenerative medicine, traditional due diligence practices are often insufficient to address the unique complexities of these cuttingedge treatments. This panel will delve into how biopharma companies can adapt their due diligence frameworks specifically for advanced therapies. Key discussion points include:
• Establishing due diligence processes to ensure a comprehensive evaluation of scientific, regulatory, IP, and commercial factors unique to advanced therapies
• Assessing the scientific foundations and clinical feasibility of your novel therapeutic modality
• Tackling challenges in production, manufacturing, supply chain, and scalability that can impact the success of advanced therapies
• Evaluating market demand, competitive positioning, and payer perspectives specific to novel therapeutic categories
• Freedom to Operate and beyond:
» Structuring due diligence to account for unique financial risks and intellectual property considerations critical to advanced therapies
Forrester Liddle
Head of Global Research and Early Development Legal; Head of IP and Legal for US R&D Novo Nordisk
Ryan Hagglund Partner
Loeb & Loeb LLP
Allison Glasunow Counsel
Perkins Coie
2:45
CUSTOMIZING YOUR IP STRATEGIES FOR ADVANCED THERAPEUTICS
Optimizing Your Patent Prosecution Playbook for Cell and Gene-Based Therapies: Key Tactics for Maximizing Monetization and Value
Innovators in the cutting-edge fields of cell and gene therapies, CAR-T, and CRISPR-based treatments need robust, forward-looking patent strategies to secure strong, defensible IP portfolios that support their market position and attract investment. This panel will bring together legal and industry experts to discuss effective prosecution strategies tailored to the unique challenges of advanced therapeutics. Topics of discussion include:
• Exploring the types of claims that best protect advanced therapies, including claims on methods, compositions, vectors, and delivery technologies
• Anticipating patent eligibility challenges in emerging therapeutic areas
Elbert Chiang Vice President, Intellectual Property Beam Therapeutics
Mary Till Of Counsel Finnegan, Henderson, Farabow, Garrett & Dunner LLP
• Understanding recent trends in patent eligibility rulings and how to adapt prosecution strategies to address evolving standards for cell and gene-based therapies
• Best practices for differentiating advanced therapeutics from prior art, addressing written description and obviousness challenges, and securing favorable examination outcomes across global jurisdictions
• Harmonizing your patent application strategy with your commercial strategy when deciding what jurisdictions to file in
3:30 Afternoon Break
3:45 Tactical Tools for Patent Lifecycle Management: Adopting Robust Strategies to Protect Your Advanced Therapy IP and Maximize Market Exclusivity
For companies entering the advanced therapeutics market, effectively managing the lifecycle of your patents is crucial. This panel will delve into patent lifecycle management strategies specifically tailored to advanced therapies, including best practices for maximizing the commercial potential and patient impact of these innovations. Topics of discussion include:
• Understanding the scope of allowable exclusivity in advanced therapeutics
• Understanding how to effectively extend patent lifecycles within regulatory frameworks, including strategies for patent term extensions (PTEs), supplementary protection certificates (SPCs), and market exclusivities
• Strengthening patent protection through strategic filing of secondary patents on formulations, processes, and delivery mechanisms
• Strategies for harmonizing patent timelines with regulatory approvals and market launches, maximizing the protection and impact of each therapeutic innovation
Erin Foley Counsel Foley Hoag LLP
Meghann Teague Head of Intellectual Property AdaptImmune
Bonnie Weiss McLeod Of Counsel Cooley LLP
Accreditation will be sought in those jurisdictions requested by the registrants which have continuing education requirements. This course is identified as nontransitional for the purposes of CLE accreditation.
ACI certifies this activity has been approved for CLE credit by the New York State Continuing Legal Education Board.
ACI certifies this activity has been approved for CLE credit by the State Bar of California.
ACI has a dedicated team which processes requests for state approval. Please note that event accreditation varies by state and ACI will make every effort to process your request. For more information on ACI’s CLE process, visit: www.AmericanConference.com/Accreditation/CLE EARN CLE CREDITS
4:30
Lessons from the Latest mRNA and LNP Patent Litigation: Understanding How Ongoing and Potential Patent Wars Could Shape the Trajectory of Novel Therapeutics
As companies vie for control over foundational IP in the advanced therapeutics space, the outcome of recent mRNA and LNP patent wars could significantly impact the development, accessibility, and cost of future therapeutics. This panel will unpack key lessons from the latest litigation and provide insights on the potential ripple effects of these disputes for stakeholders in advanced therapeutics. Topics of discussion include:
• Assessing the impact of recent pivotal cases, including contested patent claims and judicial rulings, with a focus on what these cases reveal about IP vulnerabilities and priorities in mRNA and LNP technologies
Chuck Sholtz
Vice President, Intellectual Property Nutcracker Therapeutics
Dan Liu
Attorney Loeb & Loeb LLP
• Exploring how evolving case law is impacting patentability criteria and freedom-to-operate analyses for advanced modalities
• Understanding the broader implications of these disputes on therapeutic development pipelines, collaborations, and accessibility, and how the outcome of these cases might shape patenting approaches across the biotech sector
5:15 Cocktail Reception Sponsored by
Global Sponsorship Opportunities
With conferences in the United States, Canada, Latin America and Europe, the C5 Group of Companies: American Conference Institute, Canadian Institute, and C5 Group, provides a diverse portfolio of conferences, events and roundtables devoted to providing business intelligence to senior decision makers responding to challenges around the world.
Don’t miss the opportunity to maximize participation or showcase your organization’s services and talent. For more information please contact us at: SponsorInfo@AmericanConference.com
The C5 Group, comprising American Conference Institute, the Canadian Institute and C5 in Europe, is a leading global events and business intelligence company.
For over 40 years, C5 Group has provided the opportunities that bring together business leaders, professionals and international experts from around the world to learn, meet, network and make the contacts that create the opportunities. Our conferences and related products connect the power of people with the power of information, a powerful combination for business growth and success.
Day 2
Thursday, March 20, 2025
8:45 Co-Chairs Opening Remarks
9:00 AI and the Future of Next-Gen Medicines: Harnessing AI for Drug Discovery & Development While Minimizing IP Challenges and Compliance Risks
AI is rapidly transforming the advanced therapeutics industry, offering unprecedented opportunities for innovation in areas like drug discovery, precision medicine, and clinical trials. However, integrating AI also presents challenges, from data privacy to inventorship, licensing issues, and regulatory compliance. This panel will explore both the opportunities and the complexities that AI brings to advanced therapeutics.
Key discussion points will include:
• Exploring how AI can reduce timelines and costs in drug discovery, streamline clinical trials, and enhance the development of novel therapies through predictive modeling and big data analysis
• Understanding AI’s role in personalizing treatments by analyzing patient data to predict therapeutic responses, enhance targeting, and improve outcomes for complex conditions
• Implementing best practices for managing sensitive patient data/data transfers, and navigating ethical concerns in AI-driven therapeutic applications
• Navigating IP and inventorship challenges associated with patenting and licensing of AI-driven therapeutics
• Gaining insights into current and emerging regulations around AI in the life sciences, and adopting compliance strategies for advanced therapeutic companies embracing new technologies
10:00 Morning Coffee Break
10:30 Spotlight Interview: Strategic Approaches to Accelerating Advanced Therapeutics Through Venture Financing
11:00
Securing funding for advanced therapeutic companies is increasingly challenging as these innovations often require substantial investment and long development timelines.
As traditional funding models may fall short for cutting-edge therapies like gene editing, CAR-T, and mRNA platforms, it is essential to explore new financial strategies that meet the unique demands of this sector. This panel will examine the key funding challenges for advanced therapeutics and present:
• Insights into venture philanthropy, milestone-based financing, and royalty-based investments as alternatives to traditional venture capital
• Considerations when weighing the pros and cons of government grants, public-private partnerships, and collaborations with large pharmaceutical companies to secure diverse funding streams
• Insights into securing grants and subsidies that offer financial support without equity dilution
Jocelyn Ram IP Counsel
The Broad Institute of MIT
Derek Johnson Managing Director and General Counsel Portal Innovations
12:00 Critical Considerations for Advanced Therapeutic Partnerships: Overcoming IP, Compliance, and Commercialization Challenges in Alliances and Licensing
For companies in the advanced therapeutics space, collaboration with major pharmaceutical companies and licensing agreements with universities can accelerate innovation and market entry. However, these partnerships come with their own set of challenges, from IP ownership and data rights to regulatory hurdles and commercialization terms. This panel will offer guidance on navigating these complex relationships to maximize benefits and mitigate risks.
Key discussion points will include:
• Negotiating IP rights and ownership to align with both short-term R&D goals and longterm commercialization strategies
• Protecting proprietary data, managing shared research results, and establishing clear boundaries around data use and access
• Understanding regulatory obligations, compliance requirements, and risk-sharing strategies in partnerships that span global markets
John Haugen
Senior University Counsel
Stanford University, Office of the General Counsel
Laurence Shumway
Vice President, Intellectual Property Counsel Flagship Pioneering
Jared Auclair
Vice Provost Research Economic Development, Director of Bioinnovation Northeastern University
• Designing financial arrangements, including royalties, milestone payments, and profit-sharing, to ensure alignment and value creation for both parties
Andrea Grossman Legal Advisor, Office of Patent Legal Administration USPTO
Therapeutic Biosimilars:
Strategies for Market Access and IP Protection
As biosimilars for advanced therapeutics approach market readiness, stakeholders across the industry must brace for the unique opportunities and challenges that lie ahead. This panel will explore what companies can do to prepare for and capitalize on the arrival of advanced therapeutic biosimilars, with a focus on navigating IP, market access and compliance challenges. Topics of discussion include:
• Examining the evolving regulatory landscape for advanced therapeutic biosimilars and understanding the guidelines, timelines, and data requirements for approval in key global markets
LuLu Wang Head of Intellectual Property Orna Therapeutics
Jennifer Chheda Partner Jones Day
• Providing insights into pricing, reimbursement policies, and how to address payer hesitations to facilitate broader adoption of biosimilars for complex therapeutics
• Exploring the anticipated market dynamics, including strategies for branding, differentiation, and managing IP challenges to gain a competitive edge
• Strengthening IP portfolios to safeguard proprietary advances: strategies for building robust IP protection around proprietary technologies, manufacturing processes, and delivery systems to maintain a competitive advantage as biosimilars enter the field
3:15 Balancing Disclosure and Protection: Strategies for Safeguarding Trade Secrets in Early-Stage Advanced Therapeutics
For early-stage advanced therapeutic companies, securing investment often requires a delicate balance between sharing enough information to attract potential partners or investors and safeguarding proprietary knowledge critical to their competitive edge. This panel will discuss how advanced therapeutics companies can strategically navigate disclosure and protection to attract investment while maintaining robust trade secret protection. Topics of discussion include:
• Establishing clear boundaries on information sharing: determining what information to disclose to investors and partners versus what to keep as proprietary, with a focus on minimizing exposure without diminishing investment appeal
Olga Zimmerman Director, Intellectual Property Caribou Biosciences, Inc.
Rebecca (Reba) Rabenstein Partner
Latham & Watkins LLP
• Implementing best practices for NDAs and confidentiality clauses that effectively protect sensitive information while allowing for meaningful engagement with potential investors and collaborators
• Developing internal policies, processes, and employee training that reinforce the confidentiality of proprietary methods, formulas, and data from early stages onward
• Navigating the due diligence process with prospective investors to best protect valuable IP
4:00 Conference Concludes
Upcoming Events
VENUE INFORMATION
ACCOMMODATIONS
American Conference Institute is pleased to offer our delegates a limited number of hotel rooms at a negotiated rate. To take advantage of these rates, please contact the hotel directly and quote "American Conference Institute".
Please note that the guest room block cut-off date is February 25, 2025. After that date OR when the room block fills, guestroom availability and rate can no longer be guaranteed.
Register and pay to lock in your early rate and be eligible for a full refund until March 5, 2025. If you are unable to attend for any reason, you will have the following options: y A full credit note for you, or a colleague to attend another event. y A full refund.
All cancellations and changes must be submitted to CustomerService@AmericanConference.com by March 5, 2025