Forum on IP, Funding & Tech Strategies for Novel Therapeutic Modalities & Gene Therapies - DS

March 21–22 • Omni Parker House Boston • Boston, MA

The National Forum on IP, Funding, and Tech Strategies for Novel

Therapeutic Modalities and Gene Therapies

Leveraging Technology, Safeguarding IP, and Securing Funding to Accelerate Development in mRNA, CRISPR, and CAR T

Key Speakers from Government:

Julia Tierney

Chief of Staff

U.S. Food and Drug Administration (FDA)

Anne Gussow

Supervisory Patent Examiner, Quality

Assurance Specialist

USPTO

Karlheinz Skowronek

Supervisory Patent Examiner, Bioinformatics

USPTO

Julie Wu

Supervisory Patent Examiner, Immunotherapy and Recombinant Antibodies

USPTO

Critical Sessions Will Cover:

Angle-Right The mRNA and CRISPR Patent Wars

Angle-Right Mastering the IP Due Diligence Process for Transformative Products

Angle-Right Securing Financing and Capital Formation

Angle-Right Leveraging the Power of AI, ML, Automation and Data

Industry Acumen from:

Akouos

Alexion, AstraZeneca Rare Disease

Arbor Biotechnologies, Inc.

Supporting and Cocktail Sponsor

EARN CLE/ETHICS CREDITS

2023 Co-Chairs:

Christine Bellon

Chief Legal Officer

Beam Therapeutics, Inc.

Sharick Naqi

Director & Senior Counsel

Global IP Lead – Gene Therapies

Novartis Gene Therapies

Pre-Conference Workshops:

A | Guide to mRNA, CRISPR and CAR

T-Therapies

B | AI and ML 101

Page 4 for details.

Angle-Right Connection between Commercial Viability for New Modalities and Gene Therapies via Government Payor Systems and Rebates

Angle-Right Mitigating ESG Risk-Exposure

Angle-Right Pivoting from Saving Patients to Saving the Planet

Angle-Right Ethical Considerations for Precision Medicines

Beam Therapeutics, Inc.

BioNTech SE

Ferring Pharmaceuticals

Supporting Sponsor

Lunit

Novartis Gene Therapies

Nutcracker Therapeutics

Premium Exhibitor

mRNA, CRISPR, CAR T – all acronyms for emerging and novel therapeutics that are changing modern medicine as we know it. However, the complexities concerning ownership, funding, and tech strategies for these next generation therapeutic modalities and gene therapies are the greatest barriers to commercialization.

ACI has created The National Forum on IP, Funding and Tech Strategies for Novel Therapeutic Modalities and Gene Therapies to provide you with solutions to some of the greatest challenges impeding market access for these products.

We are pleased to announce that representatives from the USPTO and FDA, as well as, distinguished in-house and outside counsel, will present at this groundbreaking event to discuss IP and regulatory initiatives that are essential to the proliferation of these therapeutics.

We anticipate this event selling out, so register today to take advantage of our best rates.

Be part of the discussions that will shape and transform the future of therapeutics.

Esteemed Faculty

2023 CO-CHAIRS

Christine Bellon

Chief Legal Officer

Beam Therapeutics, Inc.

Sharick Naqi

Director & Senior Counsel

Global IP Lead – Gene Therapies Novartis Gene Therapies

REPRESENTATION FROM KEY GOVERNMENT AGENCIES

Julia Tierney

Chief of Staff

U.S. Food and Drug Administration (FDA)

Anne Gussow

Supervisory Patent Examiner, Quality Assurance Specialist

USPTO

Karlheinz Skowronek

Supervisory Patent Examiner, Bioinformatics

USPTO

Julie Wu

Supervisory Patent Examiner, Immunotherapy and Recombinant Antibodies

USPTO

ASSOCIATIONS

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

ACADEMIA

Jared Auclair

Vice Provost Research Economic Development Director of Bioinnovation Northeastern University

John Haugen

Associate General Counsel, Office of General Counsel / Innovation and New Ventures Office (INVO)

Northwestern University

IN-HOUSE COUNSEL

Cambria Alpha-Cobb IP Counsel

Akouos

Jason Bablak

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

David Diamond VP & Head of IP Capstan Therapeutics

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

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

Ken Nesmith CBO, Oncology Lunit

Aaron Pereira Senior Director of Patents Ferring Pharmaceuticals

Patrick Perry Principal Artesia Advisors LLC

Chuck Sholtz Vice President, IP Nutcracker Therapeutics

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

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

Squire Servance Founder & Managing Partner Syridex Bio

OUTSIDE COUNSEL

Catherine A. Brandon Partner Arnold & Porter Kaye Scholer LLP

Thomas S. Brennan Partner

AentFox Schiff LLP

Laurie A. Burlingame Partner Goodwin Procter LLP

Jonathan S. Caplan Partner

Kramer Levin Naftalis & Frankel LLP

Deborah Cho

Attorney, Hogan Lovells

(Former Associate Chief Counsel, FDA)

Eldora L. Ellison Director

Sterne, Kessler, Goldstein & Fox PLLC

Ryan Hagglund Partner

Loeb & Loeb LLP

Michael B. Harlin Partner

Neal, Gerber & Eisenberg LLP

David Kim Counsel

Gemini Law

Dan Liu Attorney Loeb & Loeb LLP

Mercedes K. Meyer Attorney Banner Witcoff

Brian W. Nolan Partner Mayer Brown LLP

Kevin E. Noonan Partner

McDonnell Boehnen Hulbert & Berghoff LLP

William B. Raich Partner

Finnegan, Henderson, Farabow, Garrett & Dunner, LLP

Lauren Pignataro Rakitin Partner

Covington & Burling LLP

Charles G. Raver Attorney

Hyman, Phelps & McNamara PC

Irena Royzman Partner

Kramer Levin Naftalis & Frankel LLP

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

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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.

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DAY ONE • Tuesday, March 21st

7:45 Breakfast and Registration

8:45

Opening Remarks from the Co-Chairs

microphone-alt Christine Bellon, Chief Legal Officer, Beam Therapeutics, Inc.

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

9:00

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

microphone-alt 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

10:00

Keynote Address

microphone-alt Julia Tierney, Chief of Staff, U.S. Food and Drug Administration (FDA)

10:30

Understanding Pre-Commercialization Concerns Relative to Novel Therapeutic Modalities and Gene Therapies

microphone-alt Cambria Alpha-Cobb, IP Counsel, Akouos

Aaron Pereira, Senior Director of Patents, Ferring Pharmaceuticals

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

MODERATED BY:

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

11:30 Morning Networking Break

11:45 INTELLECTUAL PROPERTY PRACTICE POINTS

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

microphone-alt

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

MODERATED BY:

Jonathan S. Caplan, Partner, Kramer Levin Naftalis & Frankel LLP

• Anticipating how novel therapeutic modalities and gene therapies 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

» How companies that use bioinformatics can best navigate the courts and USPTO

12:45 Networking Luncheon

2:00

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

microphone-alt

David Diamond, VP & Head of IP, Capstan Therapeutics

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

MODERATED BY:

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 Novel Therapeutic Modalities and Gene Therapies

microphone-alt Todd Spalding, SVP, Deputy General Counsel, Alexion, AstraZeneca Rare Disease

Chuck Sholtz, Vice President, IP, Nutcracker Therapeutics

Brian W. Nolan, Partner, Mayer Brown LLP

MODERATED BY:

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

More companies are investing heavily in the IP rights attempting to cover these revolutionary technologies, 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

4:00

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

microphone-alt Charles G. Raver, Attorney, Hyman, Phelps & McNamara PC

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

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

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

FDA has issued draft guidances addressing the development of the new modalities like cellular and gene therapies. 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 therapies, ongoing clinical trials, and their impact on clinical practice

7:45 Breakfast and Registration

8:30

Opening Remarks from the Co-Chairs

microphone-alt Christine Bellon, Chief Legal Officer, Beam Therapeutics, Inc.

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

8:45

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

microphone-alt Thomas S. Brennan, Partner, AentFox Schiff LLP

Michael B. Harlin, Partner, Neal, Gerber & Eisenberg 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 these therapies

• Examining the role of private equity and SPACs

» Business considerations during deal making

9:45

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

microphone-alt 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 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?

11:00 Morning

11:15 CASE STUDY

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

microphone-alt Karlheinz Skowronek, Supervisory Patent Examiner, Bioinformatics, USPTO

Ken Nesmith, CBO, Oncology, Lunit

MODERATED BY:

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

12:15

Keynote Address

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

12:45 Networking Luncheon

2:00 CASE STUDY

From Idea to Commercialized Product: Collaborations and Partnering in BioPharma

microphone-alt 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

3:00 ETHICS

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

microphone-alt 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

4:00 Conference Concludes

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Venue Information

HOTEL:

Omni Parker House Boston

ADDRESS: 60 School Street; Boston, MA 02108

RESERVATIONS: 1-800-THE-OMNI

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 “ACI’s Novel Therapeutic Modalities”.

Please note that the guest room block cut-off date is February, 21, 2023. After that date OR when the room block fills, guestroom availability and rate can no longer be guaranteed.

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