WHEN:
Thursday, March 13th, 2025
5:30 PM - 8:45 PM EST

WHERE:
Alnylam Pharmaceuticals
675 West Kendall St
Cambridge, MA 02142

PROGRAM DETAILS:
Delivering Speed to Market……The Challenges Behind Program Execution

The Pharma and Biotech industries are continuing efforts across the Quality, Regulatory, and Process Development disciplines to enable product speed to patients and the market. Presentations and a panel discussion will explore the challenges and approaches employed to address some of these potential obstacles and how they may be addressed.

By sharing insights and strategies, this discussion aims to equip industry professionals across all the disciplines engaged in these  R&D and Licensure programs with the knowledge to better direct program plans and execution for product development and rapid delivery to patients in need.

AGENDA:

5:30 PM - Registration and Networking
6:45 PM - Welcome & Introductions
7:00 PM - Presentation 1*
7:30 PM - Presentation 2**
8:00 PM - Panel Discussion and Q&A
8:20 PM - Networking and program conclusion

PRESENTATION 1*: 
The Only Constant in Life is Change: Utilizing a Science-driven and Risk-based Approach to Speed Process Changes for ATMPs; Darius Pillsbury, Senior Consultant, ValSource
The only constant in life is change! This adage certainly does apply to the development and manufacture of the promising, new modalities like cell and gene therapies. How an organization manages and embraces change is paramount to the ultimate success of the commercialization of these novel products. In this presentation we will explore how to navigate various types of changes to cell and gene therapy manufacturing processes using key enablers like knowledge management and risk management in order to minimize delays. The recent draft guidance by the FDA “Manufacturing Changes and Comparability for Human Cellular and Gene Therapy Products” will be reviewed. Process changes at all stages of the product lifecycle from the manufacture and testing of initial clinical trial materials to the commercialization and long-term management of the manufacturing process based on optimization of the Process Control Strategy (referred to by the FDA as the “Cookbook”) will be presented. This presentation is targeted to subject matter experts in CMC organizations (e.g., process and analytical development, quality systems, and production), who all have shared responsibilities to rapidly advance new and exciting therapies for patients desperately in need.

A case study will be provided with an example of a significant process change made to an autologous gene-modified cellular therapy. The case study will encompass the initial identification of the need for a change, the supporting activities to implement the change, and the design and execution of an analytical product comparability study, based on best industry practices including the use of risk assessment and compliance with the expectations of global regulatory agencies. The challenges will be highlighted with solutions provided to overcome any barriers and challenges to implementation, as well as the follow up activities also important post-change. 

PRESENTATION 2**:
Accelerating Process Scale-up through enhanced Contamination Control Strategy (CCS) and Vendor Management; Thomas C. Page, Ph.D., Pioneer GMP Consulting
Navigating the path to the market for pharmaceutical products requires a systematic approach to addressing business, technical, and regulatory risks. This presentation highlights the importance of gap assessments and resulting risk-based control strategies to streamline facility design, optimize validation timelines and assure quality metrics.

Key focus areas include leveraging closed processes to isolate operations from the environment using single-use systems to enhance contamination control strategies. We will discuss a different approach to generating user requirement specifications for standard packaged equipment, the effective use of vendor-provided documentation and the emerging role of vendor design risk assessments. Additionally, the session will explore innovative approaches to installation and operational Qualification, examining the potential for pre-shipment vendor-execution and out of place qualification to compress readiness timelines.   Finally, several common gaps in how users manage critical component suppliers with potential approaches to mitigating common issues that emerge between user and vendors (sometimes contentiously so).

This presentation offers actionable strategies for pharmaceutical manufacturing professionals to mitigate risks, enhance product quality, and accelerate project timelines, ensuring success in today’s demanding regulatory landscape.

SPEAKERS:

REGISTRATION FEES:
**PDA Members, please reach out to office@ispeboston.org for your registration code**

Registration by EOD Wednesday, March 5, 2025
Members (Early Bird): $50
Non-Members (Early Bird): $95
Emerging Leaders Members (Early Bird): $20
Student Members: FREE

Registration Starting Thursday, March 6, 2025
Members: $60
Non-Members: $115
Emerging Leaders Members: $30
Student Members: $10 

**PLEASE NOTE: CANCELLATIONS RECEIVED ON/AFTER MARCH 5 ARE SUBJECT TO BILLING**