At the cutting edge of science, cell and gene therapies offer the potential to transform modern medicine from managing to curing a wide range of diseases. As both established and emerging biopharmaceutical companies seek to develop, test, and obtain regulatory approval for these novel treatments and bring them to market, they turn to Sidley’s multidisciplinary team of lawyers to solve their unique legal and regulatory challenges.

We have significant experience partnering with cell and gene therapy clients in the context of licensing and commercial collaborations; protecting IP; designing and conducting clinical trials; securing regulatory approvals in the U.S., EU, and Asia; securing supply chain and GxP compliance; obtaining coverage, coding, and reimbursement; structuring patient support and engagement programs; and managing fraud and abuse risk, among other issues.

We bring particular value to cell and gene therapy companies and their investors in the following areas:

• Regulatory: Advanced therapeutics are subject to a rapidly evolving regulatory environment that can quickly derail product development and launches when not handled appropriately. Our lawyers leverage unparalleled experience and knowledge to help advanced therapy companies devise their pre-market strategies, avoid obstacles in the pre-submission process, navigate the pathway to approval, explore potentially available exclusivities, and communicate appropriately with prescribers, patients, and other stakeholders. When necessary, we advocate for our clients in disputes with the approval authorities. We offer our clients insight into how regulators think, as many of our lawyers have held senior positions at the FDA and other governmental agencies.
• GxP Compliance: Many advanced therapies, including CAR T-cell and gene therapies, do not fit neatly into the FDA’s or other health authorities’ existing frameworks for ensuring GxP compliance. Sidley has long been committed to providing practical, timely, and strategic GxP compliance advice to applicants and manufacturers of CAR T and other advanced therapies struggling to understand how these statutory and regulatory requirements apply to their products. We have witnessed first-hand the challenges faced by these novel therapies with the FDA and other health authorities, and use this experience to help clients anticipate challenges and to minimize the business impact when such challenges arise.
  
  We combine this experience with an understanding of FDA’s thinking about advanced therapies with deep technical knowledge. Our dedicated team of regulatory compliance professionals, including a number of former FDA investigators and industry experts, helps companies assess GxP compliance at every phase of development, from early clinical through commercialization.
• Licensing and Collaborations: Given the cutting-edge nature of cell and gene therapy technologies, traditional structures for collaborations, licenses, and manufacturing often do not adequately meet the needs of companies in this space. Many of our practitioners have sophisticated scientific training, providing the insight necessary to fundamentally understand the range of cell and gene therapy technologies and create contractual arrangements that meet both their scientific and business needs.In many cases, structures and terms need to be created in a bespoke manner to address these needs, requiring a deep, fundamental scientific understanding of the range of advanced therapy technologies. Our practitioners leverage the broad range of their experience, developed through a significant number of transactions, to create structures and craft agreements that meet the unique needs of advanced therapeutics companies.

• Coverage, Coding, Pricing, and Reimbursement: Cell and gene therapy companies face additional scrutiny as a result of what is typically a high price point, which can lead to reimbursement challenges. We help these companies manage the complex legal, policy, political, and public relations risks associated with pricing cell and gene therapy products and navigating a pathway to coverage and reimbursement. A lack of clear guidance and evolving coverage and reimbursement landscapes can present further challenges, but as recognized industry leaders in this area, our lawyers interface with all relevant regulatory and governmental agencies and work with clients and trade associations to guide legislative and regulatory decision-makers as they develop coverage, coding, and reimbursement policies for existing and novel cell and gene therapy products. We also help clients anticipate issues that may arise related to provider reimbursement and acquisition cost for these products.
• Market Access/Payer Contracting: Advanced therapeutics companies must think strategically about market access, customer contracting, and patient support strategies along with the related legal implications before, during, and after product launch. Our team works with companies throughout the product life cycle to manage possible legal risks while helping to ensure optimal coverage of and access to our clients’ products.
• Patient Access: Sidley is a thought leader on issues arising with respect to cell and gene therapy access for financially needy patients and cases of medical hardship and urgency. We have successfully obtained advisory opinions from the Office of Inspector General (OIG) in the patient access space, including those pertaining to travel and lodging programs. Additionally, our team regularly advises clients on the considerations that may arise with providing or administering apheresis, genetic and diagnostic testing, and other points of care that precede cell and gene therapy.
• Healthcare Compliance: Cell and gene therapies are different; so too are their manufacturers’ needs to interact with healthcare professionals, patients and caregivers, and payers. Our team of fraud and abuse and compliance lawyers assists these clients to evaluate risk, design creative approaches to compliant arrangements with referral sources and patient support activities, and build and assess compliance controls. To do this, we leverage our extensive experience and benchmarking data that is specific to cell and gene therapies to design controls that are tailored to each business, instead of advancing a one-size-fits-all approach.

Representative Matters:

• Provided FDA and EU regulatory counsel, including on orphan drug exclusivity issues, to a gene therapy company, as well as advised the client on a strategic transaction and the potential acquisition of a life sciences company.
• Advised a biotechnology company on market access and other commercial launch matters for multiple gene therapy products involved in advanced clinical investigations.
• Assisted a gene therapy client in responding to an FDA complete response letter (CRL) and BLA resubmission.
• Represented Intellia Therapeutics, a leading genome editing company, in negotiating license and collaboration agreements with several cell therapy companies.
• Advised several companies developing cell-based therapies and immunotherapy products on patent and related intellectual property matters, including offensive and defensive patent litigation, strategic advice on patent matters, and intellectual property due diligence for strategic partnerships, investments, acquisitions, and mergers. Examples of products being developed by these clients include allogeneic and autologous CAR T products, therapeutic tumor infiltrating lymphocyte (TIL) products, targeted monocyte products, and immune-stimulating antibodies, as well as techniques to produce these products.
• Represented a cell-based immunotherapy biotechnology company in a strategic partnership with an immuno-oncology company focused on personalized anticancer T-cell therapies against solid tumors. Sidley also counsels the client on healthcare and regulatory compliance matters.
• Advised a gene and cell therapy biotechnology company on the EU/UK regulatory legal framework applicable to the company's products in development and assists in developing a regulatory and commercial strategy to secure prompt orphan indication approval of their product and to obtain an appropriate reimbursement price and a positive recommendation by health technology authorities.
• Counseled a global biotechnology company on a variety of matters related to its gene therapy product, including pricing, managing shortage issues with the FDA, negotiating post-marketing requirements, providing FDA regulatory advice, and assisting with FDA supervisory appeal.