Novel Therapeutic Modalities and Gene Therapies

Breakfast and Registration

Opening Remarks from the Co-Chairs

Christine Bellon
Chief Legal Officer
Beam Therapeutics, Inc

Sharick Naqi
Director & Senior Counsel
Global IP Lead – Gene Therapies
Novartis Gene Therapies

Welcome to the BioPharma Revolution: 60 Minutes on the Intersection of Science, Business, Policy, and the Law

Julie Wu
Supervisory Patent Examiner, Immunotherapy and Recombinant Antibodies
USPTO

Catherine A. Brandon
Partner
Arnold & Porter Kaye Scholer LLP

Rebecca K. Wood
Partner
Sidley Austin LLP
(Former Chief Counsel, FDA)

In this opening session, panelists will provide an overview of the driving force behind the BioPharma revolution and explain the opportunities it offers by detailing the design and delivery of new medicines and therapies, including gene therapy, cell therapy, CAR-T, and bispecific and RNA treatments.

• Understanding how mRNA, CRISPR, and CAR-T are game changers from scientific, commercial, legal, and policy perspectives
• Analyzing the impact of the Inflation Reduction Act (IRA) on BioPharma innovation, patent protection and market entry
• How will the IRA impact the ability to secure investments, partnerships, and acquisitions?
• How will the Biden EO support industry, R&D, and workforce development?
• How are these therapies changing the way we look at treatment and prevention?
• Where do they fit into the existing landscape of small and large molecules

Keynote

Julia Tierney
Chief of Staff
U.S. Food and Drug Administration (FDA)

Industry is navigating several debilitating roadblocks, including a human talent shortage that falling behind the rate of technological advancement.

Understanding Pre-Commercialization Concerns Relative to the Products of the New BioPharma Revolution

Cambria Alpha-Cobb
IP Counsel
Akouos

Aaron Pereira
Senior Director of Patents
Ferring Pharmaceuticals

Rana Sawaya
Sr. Director, Cell & Gene Therapies Patent Lead
BioNTech SE

Moderator:

Ryan Hagglund
Partner
Loeb & Loeb LLP

Despite the efficacious promise of these new modalities and their potential for commercial success, the next generation of therapies continue to face challenges that can limit their widespread consideration and commercialization.  This session will consider pre-commercialization concerns for design and delivery of new medicines and therapies, including gene therapy, cell therapy, CAR-T, and bispecific and RNA treatments.  Points of discussion will include:

The Current Pre-Commercialization Landscape:

• Examining the types of products that pharmaceutical, biotechnology and biopharmaceutical companies are seeking to develop now based on mRNA, CRISPR, and CAR-T
• Identifying impediments – through patent or regulatory restraints – which prevent these companies from pursuing the development of the desired product
• Identifying FDA hurdles that may not clear even if all patent and other IP hurdles are met
• Developing techniques for analyzing the value the product adds to the company’s portfolio, and methods for proving value

Regulatory Considerations:

• Understanding how the introduction of these new therapies will change the commercial landscape
• Examining the role of the Center for Medicare and Medicaid Services (CMS) in the approval process
• The connection between CMS approval and commercial viability via government payor systems and rebates
• Assessing the competition and analyzing potential therapeutic interchangeability considerations -if any

Morning Networking Break

INTELLECTUAL PROPERTY PRACTICE POINTS

Managing and Monetizing Patent Portfolios: Identifying Business Opportunities and Revenue Generation Through IP

Eldora L. Ellison
Director
Sterne, Kessler, Goldstein & Fox PLLC

John Haugen
Associate General Counsel Office of General Counsel / Innovation and New Ventures Office (INVO)
Northwestern University

Moderator:

Jonathan S. Caplan
Partner
Kramer Levin Naftalis & Frankel LLP

• Anticipating how new BioPharma Revolution will re-write the rules of patent portfolio management and monetization
• Understanding the importance of always keeping innovation in focus
• Finding a common thread among life cycle management, brand optimization, and new product development
• Exploring how collaborations, M&A, and other deals impact patent portfolio management and revenue streams
• Accounting for litigation risk
• Evaluating the impact of §101 and on the new research and new patents

Networking Luncheon

mRNA and CRISPR Patent Wars: What Every Life Sciences Business Executive and Counsel Need to Know

Anne Gussow
Supervisory Patent Examiner, Quality Assurance Specialist
USPTO

Kelly Morgan
VP, Head of IP and Legal
Arbor Biotechnologies, Inc.

Dan Liu
Attorney
Loeb & Loeb LLP

David Diamond
VP & Head of IP
Capstan Therapeutics

Moderator:

Irena Royzman
Partner
Kramer Levin Naftalis & Frankel LLP

Patent fights rarely demand the attention of the industry the way one battle has, and one battle may. 

After numerous skirmishes in the district courts, the latest round of the CRISPR battle took place before the USPTO’s PTAB which ruled each party completed on the gene editing tech doesn’t overlap and can be separately patented.  Additionally, the two leading COVID-19 vaccines on the market, introduced by Moderna Inc. and BioNTech SE are currently the subject of major patent litigations relating to how those therapies are delivered.

The implications of these cases are not only for the lawyers in the room – as this panel will provide an overview of these battles and detail the critical business implications which they present.  Topics of discussion will include:

• Detailing the status of these patent wars
• Understanding how they will impact collaboration and tech transfers
• Determining if these on-going cases will interfere with future R&D and commercialization
• Considering different business models and the emerging landscape of gene editing

The Freedom to Operate: Assessing Competitors, Mapping Opportunities, and Mastering the IP Due Diligence Process for New Modalities

Todd Spalding
SVP, Deputy General Counsel
Alexion, AstraZeneca Rare Disease

Chuck Sholtz
Vice President, IP
Nutcracker Therapeutics

Brian W. Nolan
Partner
Mayer Brown LLP

Moderator:

William B. Raich
Partner
Finnegan, Henderson, Farabow, Garrett & Dunner, LLP

More companies are investing heavily in the IP rights attempting to cover the revolutionary technologies driving the biopharma revolution, while simultaneously embarking upon equally aggressive licensing programs. Thus, anyone intending to use these new technologies and therapies must be wary of investing without thorough and robust IP due diligence.  As these clashes over IP begin to play out, clever participants are paying close attention and taking stock of the outcomes, as they will inform best practices for the freedom-to-operate analysis the ability to commercialize inventions. Points of discussion will include:

• Defining the scope of the patent search strategies
• Identifying and mitigating patent infringement risks prior to commercialization
• Considering how best to position and properly protect your IP
• Ensuring the freedom-to-operate via IP inventory and FTO analysis
• Conducting client competitor patent landscape analyses and preparing the FTO opinion
• Identifying common missteps to avoid with an FTO analysis specific to new modalities
• Minimizing future risk of litigation and avoiding unnecessary expenses

The Pathway from Idea to Regulatory Approval: Considerations for Clinical Trial Applications and Regulatory Clearances

Jason Bablak
Vice President, Regulatory Science, U.S.
Orchard Therapeutics

Deborah Cho
Attorney
Hogan Lovells
(Former Associate Chief Counsel, FDA)

Charles G. Raver
Attorney
Hyman, Phelps & McNamara PC

Deborah Hursh
Principal
Hursh Cell Therapy Consulting, LLC
(Former Senior Investigator, CMC Reviewer, CBER of FDA)

FDA has issued draft guidances addressing the development of the new modalities driving the biopharma revolution. These guidances offer recommendations on the information that should be included in an Investigational New Drug (IND) application. During this regulatory session, points of discussion will include:

• Analyzing the key considerations put forth in the three available guidances on gene editing, CAR T products, and regenerative medicine therapies
• Detailing FDA’s recommendations for preclinical and clinical testing, chemistry, manufacturing, and controls (CMC)
• Outlining what FDA recommends be included in Investigational New Drug applications (IND)
• Ensuring proper identity, potency/strength, quality and purity
• Reviewing the already approved biopharma revolution therapies, ongoing clinical trials, and their impact on clinical practice

Conference Adjourns to Day Two

Breakfast and Registration

Opening Remarks from the Co-Chairs

Christine Bellon
Chief Legal Officer
Beam Therapeutics, Inc

Sharick Naqi
Director & Senior Counsel
Global IP Lead – Gene Therapies
Novartis Gene Therapies

Business Considerations and New Approaches to Financing and Capital Formation for Transformative Products

Michael B. Harlin
Partner
Neal, Gerber & Eisenberg LLP

Thomas S. Brennan
Partner
ArentFox Schiff LLP

Patrick Perry
Principal
Artesia Advisors LLC

Over the past five years, the biopharma industry has seen billions of dollars invested in therapeutic-based biopharma companies. VC investors appeared to be focusing on next-gen therapies, precision medicine, machine learning, and new delivery methods.  However, even more recently, investors are leaving the space and longtime venture firms are funding less.

As investors and companies alike attempt to navigate a new world of falling valuations, this session will offer winning strategies for new approaches to financing and capital formation.

Points of discussion will include:

• Best practices for overcoming traditional financial barriers and achieving development excellence
• How to raise capital and recoup your investment
• Detailing examples of non-traditional financing and capital for the biopharma revolution
• Examining the role of private equity and SPACs in the advancement of the biopharmaceutical revolution
• Business considerations during deal making

Searching for Sustainability: The Top ESG Challenges and Opportunities for the BioPharma Revolution

Squire Servance
Founder & Managing Partner
Syridex Bio

Laurie A. Burlingame
Partner
Goodwin Procter LLP

Sustainable financing has grown in popularity in recent years, as more institutional investors and funds incorporate Environmental, Social and Governance (ESG) investing tactics. Thus, those wishing to participate in the biopharma revolution must prepare to answer for their companies’ ESG impact to capture investment capital and prepare for potential new regulatory requirements.

This session will detail ESG challenges for biopharma companies and offer proven strategies for effective communication with ESG-focused investors. Points of discussion will include:

• What are the greatest ESG risks biopharma companies are facing?
• Why is it important for biopharma companies to understand their ESG risk exposure?
• How do you provide clarity and transparency to stakeholders on sustainability attributes?

Morning Networking Break

CASE STUDY

Real World Examples on Leveraging the Power of AI, ML, Automation and Data for the Development of Better Medicines

Karlheinz Skowronek
Supervisory Patent Examiner, Bioinformatics
USPTO

Ken Nesmith
CBO, Oncology
Lunit

Moderator:

David Kim
Counsel
Gemini Law

Artificial intelligence (AI), machine learning (ML), automation, and data analytics have the potential to improve many areas of medicine, with drug development and discovery as a principal focus. The use of these technologies can make drug development faster, cheaper, and more efficient. 

Given their transformative potential, industry players need to prepare for a not-so-distant-future in which these technologies will be routinely used. This case study will:

• Teach you how these modalities are creating significant value
• Help you determine where and how these new tools can be utilized to accelerate the pace of drug development, raise profits, and get medicines into the market faster
• Learn the different AI/ML technologies available in biopharma R&D
• What are the benefits and drawbacks of each

Keynote

Kendalle Burlin O’Connell, Esq.
President and CEO
MassBio

Networking Luncheon

CASE STUDY

From Idea to Commercialized Product: Collaborations and Partnering in BioPharma

Lauren Pignataro Rakitin
Partner
Covington & Burling LLP

Jared Auclair
Vice Provost Research Economic Development
Director of Bioinnovation
Northeastern University

While smaller biotechnology companies are typically undertaking early stage research and perhaps even early clinical development in-house, the industry continues to see partnerships between big pharma and biotech in later stage development and commercialization. These collaborations are key to long-term success in the industry, as big pharma is able to provide capital and later-stage expertise and market access, while biotechnology companies are the crux of innovation. This session explores common legal and business issues at- stake in negotiating these collaborations or partnerships, such as:

• How to maintain control of and value in the biotechnology company’s platform or other technology while providing exclusivity to the partner
• How manufacturing gene and other cell therapies differs from traditional therapies and how this plays out in collaborations
• How does ownership of resulting intellectual property work if both parties are contributing to R&D

ETHICS

Ethical Considerations for Precision Medicines: Emerging Ethical, Legal and Social Concerns Related to Trends in Precision Medicine

Kevin E. Noonan
Partner
McDonnell Boehnen Hulbert & Berghoff LLP

Mercedes K. Meyer
Attorney
Banner Witcoff

Precision medicine offers many potential advancements in patient care. However, these advanced therapies raise several ethical, legal and social concerns.

• Understanding the significance of the choice of law for precision medicine research
• Appreciating the complexities associated with informed consent
• Use of data
• Disclosure of results
• Advancing the health of underserved populations through genomics and precision medicine
• Addressing major health disparities based on race, ethnicity, sex, disability, socioeconomic status, etc.
• The ethics of access

Conference Concludes