Advanced Therapeutics

With the second Trump administration initiating changes across healthcare policy, regulatory priorities, and federal funding programs, the advanced therapeutics sector faces both uncertainty and potential opportunity. This session will examine how shifting priorities under the current administration — including the influence of RFK Jr.’s leadership at HHS — is shaping the regulatory, IP, and commercialization landscape for cell and gene therapies, RNA-based treatments, and regenerative medicines.

Discussion points include:

• Examining the latest federal policy shifts impacting FDA approval pathways, clinical trial requirements, and enforcement approaches for advanced therapeutics
• Exploring how a reconfigured HHS under RFK Jr. is reshaping NIH and FDA funding priorities, scientific independence, and public trust in therapeutic innovation
• A look at the future of pricing, reimbursement, and market access under changing CMS policies and budgetary constraints
• Updates on new and pending IP reforms impacting advanced therapeutics, including the REMEDY Act, PREVAIL Act, and RESTORE Patent Rights Act
• Assessing the current and future landscape of government march-in rights and the latest moves by the current administration to invoke Bayh-Dol

As U.S. policy uncertainty continues to raise questions around approval timelines, pricing, and market access, global markets are becoming increasingly critical for the advancement of cell and gene therapies, RNA therapeutics, and other advanced therapies. This session will explore how companies are rebalancing their regulatory and commercialization strategies to secure approvals across the EU and UK, while also addressing the challenges of R&D offshoring and multi-jurisdictional compliance.

Discussion points include:

• Strategic approvals outside the U.S.: Evaluating pathways in the EU and UK to ensure early market entry and risk diversification
• Balancing R&D footprints: Managing the shift toward global R&D hubs, clinical trial sites, and manufacturing networks amid rising U.S. policy and cost pressures
• Navigating the complexities of differing regulatory frameworks, data requirements, and post-market obligations across jurisdictions
• Building resilience through global planning: Integrating regulatory, IP, and commercialization strategies to sustain innovation and protect long-term ROI

In today’s rapidly evolving policy environment, companies developing advanced therapeutics must be prepared to adapt to shifting regulatory priorities, funding structures, and geopolitical dynamics. From FDA oversight to global market pressures, the ability to anticipate and proactively manage policy swings is becoming a core competency for biotech and pharma leaders. This session will unpack:

• Strategies for embedding policy risk management into corporate planning to safeguard R&D pipelines, commercialization timelines, and investor confidence
• Scenario planning and agility: Building flexible business models and development strategies that can withstand sudden shifts in oversight or funding
• Aligning internal teams and stakeholders: Integrating compliance, legal, R&D, and investor relations into a unified risk management approach
• Protecting long-term value: Using IP strategy, funding diversification, and regulatory engagement to reduce exposure to policy volatility

The IRA and recently proposed Most Favored Nation (MFN) pricing model, are reshaping the reimbursement and commercialization landscape for advanced therapeutics. For companies in the rare disease space — where development costs are high and patient populations are small — these policies carry significant implications for market access, deal-making, and innovation pipelines. This session will examine the legal, regulatory, and strategic dimensions of evolving federal drug pricing initiatives and provide practical guidance for managing their impact.

Discussion points will include:

• Breaking down the IRA’s impact on rare disease therapies: Pricing negotiations, coverage challenges, and long-term reimbursement risks
• Decoding the MFN Executive Order: What it means in practice, whether it is legally binding, and potential enforcement or implementation pathways
• Addressing the tension between cost-containment measures and sustaining R&D for rare and ultra-rare conditions
• Preparing your company and pipelines for shifting federal drug pricing landscapes and cross-border ripple effects
• Exploring how the IRA and proposed MFN are influencing patent litigation strategies for advanced therapies

As the current administration signals a stronger intent to invoke march-in rights under the Bayh-Dole Act, companies in advanced therapeutics must reassess their exposure and obligations tied to federally funded research. For organizations ranging from early-stage start-ups to established biopharma players, understanding how government rights may apply—and how to safeguard product pipelines—is now a critical strategic priority. This panel will tackle topics including:

• Clarifying the scope of government march-in rights under Bayh-Dole and how they may affect cell, gene, and RNA therapy developers
• Assessing the level of risk for companies with past or ongoing federally funded research, including start-ups with early-stage support
• Strategies to mitigate exposure and protect product lines while balancing commercialization goals with compliance
• Preparing for potential government action: proactive steps legal, regulatory, and business teams should be taking now

In today’s cautious capital environment, advanced therapeutics companies face unprecedented challenges in securing sustainable funding. With investors scrutinizing risk profiles, reimbursement uncertainty, and lengthy development timelines, it is more critical than ever to implement resilient financing strategies that balance near-term survival with long-term innovation. This session will explore practical approaches to capital planning, investor engagement, and deal-making that can help companies withstand financial headwinds while keeping pipelines on track. Join our panelists as they provide insights on:

• What current trends signal for early- and late-stage financing rounds
• Leveraging partnerships, licensing deals, and alternative funding models to sustain operations
• Balancing short-term liquidity with long-term growth: Building financial resilience without stalling innovation pipelines
• Case studies and lessons learned: How advanced therapeutics companies are navigating capital scarcity in real-world scenarios