Cell and Gene Therapy

Since the earliest developments in cell and gene therapy and regenerative medicine, Holland & Knight has represented clients – product developers, academic research institutions and healthcare providers – to help ensure compliance with U.S. Food and Drug Administration (FDA) requirements and other regulations. Our lawyers and policy advisors have a strategic understanding of the entire federal legislative and regulatory process, from Congress and the White House to the departments and agencies that develop and implement the rules.

Holland & Knight's Cell and Gene Therapy Team is chaired by Michael Werner, a leading attorney focused on the regulation and reimbursement of cell therapy, gene therapy, tissue engineering and regenerative medicine products. Mr. Werner co-founded the Alliance for Regenerative Medicine, an international advocacy organization that amplifies the voices of more than 500 industry players before key government officials. He drafted the statutory language for the FDA's expedited pathway for regenerative medicine advanced therapy (RMAT). He also has extensive experience in human subject protection issues, such as institutional review board (IRB) review and informed consent, as well as conflicts of interest and other bioethics issues arising from research and the use of new technologies.

Full Range of Client Services

Among other matters, clients rely on our cell and gene therapy lawyers and policy professionals to help guide them toward successful biologics license applications (BLA), including investigational new drug (INDs) applications, RMAT, breakthrough therapy, and accelerated approval designation, priority review, compliance with FDA advertising and promotion regulations, and other challenges. We also monitor developments involving the drafting and implementation of requirements from the Prescription Drug User Fee Act (PDUFA).

Additionally, our team:

• advises companies on a range of pre- and post-market FDA regulatory strategies, including product classification, application filings, Good Manufacturing Practice (GMP) requirements and other manufacturing issues, and compliance
• provides counsel on MasterFiles, platform technology designations, Advanced Manufacturing technologies and other non-traditional routes to market
• performs regulatory and reimbursement due diligence for investors in biotechnology and medical device companies
• assists cell and gene therapy transactions by assessing intellectual property (IP) value – including patent strengths – and drafting complex IP licensing agreements
• advocates for clients before Congress
• represents companies in meetings with FDA officials
• helps companies navigate the regulatory pathway to market for novel technologies
• advises companies on FDA marketing rules and labeling claims
• counsels clients during inspections and post-inspection matters, responses to Form 483s, FDA untitled and warning letters, and other enforcement actions
• applies for appropriate product codes to facilitate tracking and future payment of FDA-regulated products
• organizes and manages coalitions of healthcare stakeholders to help secure policy objectives through representation before Congress, FDA, the Centers for Medicare & Medicaid Services (CMS) and other federal agencies
• provides analysis and strategies for reimbursement under Medicare (Parts A and B) and Medicaid

Representative Matters

Our Cell and Gene Therapy Team's experience includes:

• co-founding and currently serving as senior policy counsel to the Alliance for Regenerative Medicine, which focuses on regulatory, legislative and reimbursement advocacy for gene therapy, cell therapy, advanced therapy and regenerative medicine products for more than 500 emerging and established biotechnology companies, academic and medical research institutions, and patient organizations
• drafting a new RMAT designation in a federal statute that allows companies and researchers to access FDA-expedited approval programs, reducing the typical waiting period of 10 to 12 years for FDA approval
• advising a cell therapy company regarding its FDA regulatory strategy to obtain FDA approval to launch Phase III trial
• securing $8 million in federal funding to support regenerative medicine standards development efforts for a nonprofit; work included identifying federal funding sources and working with key members of Congress and their staffs
• securing FDA Fast Track designation for a regenerative medicine/biotech company developing a treatment for a degenerative disease, helping to ensure that the company's treatment will have a smoother and faster path to market, which will save the company tens of millions of dollars in development costs and get the product to patients faster
• advising a company closing a tissue storage business regarding disposition of products
• assessing IP asset value of cell and gene therapy companies being bought or sold, including strength of patents against potential competitors
• advising major cancer and other academic medical centers and pharmaceutical companies on complicated, multilayered license agreements for cellular tissue and other materials in various stages of scientific development and patent prosecution
• representing and defending clients in FDA-related IP matters handled by IP litigators and prosecutors, including patent attorneys and agents with advanced science and engineering degrees