Cell & Gene Therapy Expert Consulting Team

By partnering with us, clients can be confident that they have access to the knowledge and expertise they need to successfully bring their therapies to market. Contact us today to learn more about how we can help you achieve success in cell and gene therapy development.

Early Stage Development

At ProPharma, we understand the emotional significance of taking your cell and gene therapy (CAGT) product from molecule to the bedside. Our unwavering commitment to your vision drives us to offer comprehensive early-stage development services that touch lives.

IND/CTA Preparation

Turn your groundbreaking therapy into a reality with ProPharma's seasoned regulatory affairs specialists and consultants. We are your guiding light through the maze of regulatory complexities. Our experts craft meticulous and compelling Investigational New Drug (IND) applications or Clinical Trial Applications (CTAs) that capture the essence of your therapeutic potential. With our unwavering advocacy, we simplify the process and increase the likelihood of success. Let us ignite the spark of possibility and bring your vision to life.

ProPharma assists with tasks such as conducting animal/toxicology studies, developing dosing strategies, and performing long-term follow-up analyses (LTFU). Our experts also offer strategic advice and help with manufacturing and control of materials, as well as analytics and feasibility of specifications. Additionally, we help shape the design of both clinical and nonclinical studies, incorporating novel adaptive approaches.

Quality, Compliance & Manufacturing

Every step of your journey is sacred. We understand the profound responsibility of ensuring that your therapy is not only innovative but also safe and reliable. Our expert team will work hand in hand with you to establish an unwavering commitment to quality, compliance, and manufacturing excellence. Together, we will weave an unbreakable shield of protection around your therapy, ensuring that it reaches patients with the utmost integrity.

We are experts in Good Manufacturing Practices (GMP), CQV (Commissioning, Qualification, and Validation), and the development of Quality Management Systems (QMS) for inspection readiness. Our team will help you select the right contract manufacturing organizations (CMOs) and perform risk assessments of critical steps. We can help alleviate the challenges associated with analytical development, process development, validation, and mapping, as well as technology transfer.

Safety & Risk Assessment

Embrace the future with confidence, knowing that your therapy has undergone meticulous safety scrutiny. At ProPharma, we leave no stone unturned in identifying and mitigating risks. We understand the immeasurable importance of patient safety and the trust placed in your therapy. Through rigorous risk assessments and strategic planning, we will help you navigate the turbulent waters, safeguarding the dreams of those awaiting life-changing treatments.

We conduct risk-benefit evaluations and provide support in developing Risk Management Plans (RMP) and Risk Evaluation and Mitigation Strategies (REMS). Our team knows how to create key clinical documents like the Investigator's Brochure (IB) and protocols. We can help address environmental risk and containment for GMO licensing and formulate safety data strategies for the Biologics License Application (BLA) or Marketing Authorization Application (MAA). And in preparation for market, our global labeling development experts will ensure you meet industry standards.

Development Planning

Every journey requires a map to guide you through uncharted territories. Together, we will chart a course, meticulously planning every milestone and overcoming hurdles along the way. Our seasoned team of experts will become your compass, providing strategic guidance and support to navigate the scientific, regulatory, and logistical challenges. Let us be your guiding star, lighting the path toward transformative success.

Our cell and gene therapy consultants and experts provide guidance in formulating an overall regulatory strategy to ensure that Sponsors align their development plans with the regulatory landscape. This includes considering orphan and pediatric indications, as well as exclusivity options to maximize market exclusivity and commercial potential. Our expertise in process optimization, validation, and scalability supports Sponsors in developing robust and scalable manufacturing processes. We help define the Target Product Profile (TPP) to ensure that development efforts remain focused on meeting patient and market needs. Additionally, we’ll recommend reimbursement strategies that help Sponsors navigate the complex reimbursement landscape, enhancing the commercial potential of their future products.

Clinical Development

As your therapy transitions from the lab to real-world impact, we understand the profound emotional weight of this stage. At ProPharma, we stand shoulder to shoulder with you, providing unwavering support and empowering you to make a lasting difference.

Clinical Trial Execution and Support

Every trial holds the potential to change the course of history. With our deep understanding of the unique nature of each trial, we tailor our comprehensive clinical trial services to meet your specific needs and objectives. Our experienced team guides you through the intricacies of clinical research, ensuring the safe and efficient delivery of your innovative therapies to patients. From protocol design to patient recruitment, data management to analysis, we leave no stone unturned in upholding the highest standards of scientific rigor. Together, we will create a chapter of hope, courage, and resilience, making a lasting impact.

ProPharma’s cell and gene therapy consultants provide comprehensive clinical trial support that addresses the unique challenges and complexities of these groundbreaking therapies. Our full-service clinical trials cover every aspect of the trial process, including protocol design, patient recruitment, and data management. With adherence to the highest scientific standards, our experienced team offers specialized expertise in patient eligibility, treatment administration, and follow-up assessments.

Additionally, we offer functional service provider solutions, allowing you to leverage our expertise in specific areas such as medical writing, biostatistics, and clinical monitoring. We embrace decentralized clinical trials, bringing the trial directly to the patient's home or local healthcare setting through remote capabilities and innovative technologies. Our embedded solutions further enhance support and integration by seamlessly becoming an extension of your team, optimizing trial design, execution, and project management.

Partner with ProPharma to navigate the complexities of cell and gene therapy clinical trials and unlock the full potential of your groundbreaking therapies. Together, we will make a meaningful difference in the lives of patients and contribute to the advancement of medical science.

CMC & Manufacturing for CAGT

Witness the culmination of your efforts as your therapy takes shape within the walls of our trusted manufacturing partners. We leave no room for compromise when it comes to Chemistry, Manufacturing, and Controls (CMC). With meticulous precision, we will optimize your manufacturing processes, ensuring that each dose carries the essence of your vision. Together, we will breathe life into your therapy, igniting the flames of hope.

At ProPharma, we offer comprehensive CMC & Manufacturing services for cell and gene therapy products. Our expertise spans across various key areas, ensuring the highest standards of quality, compliance, and efficiency throughout the manufacturing process. We work closely with you to develop a global CMC strategy, encompassing all necessary regulatory requirements and considerations. With a focus on Quality by Design (QbD) and Quality Risk Management, we meticulously analyze and optimize your manufacturing processes, mitigating risks and maximizing product quality. Our team includes Qualified Persons (QP) and Responsible Persons (RP) for EU release and distribution, providing the necessary expertise to navigate the regulatory landscape and ensure compliance. Additionally, we conduct supply chain audits to ensure the integrity and reliability of your therapy's supply chain. With our end-to-end CMC and manufacturing capabilities, we empower you to bring your cell and gene therapy products to patients with confidence and integrity.

Regulatory Strategy and Submissions

Clear the path for your therapy to reach those in need. Our experienced regulatory affairs team will stand by your side, crafting a strategic roadmap for regulatory success. We will immerse ourselves in the intricate dance of regulations, leveraging our expertise to navigate the ever-evolving landscape. Let us shoulder the burden of regulatory complexities, allowing you to focus on what truly matters: transforming lives.

ProPharma's Regulatory services encompass a comprehensive range of expertise to support the successful development and approval of cell and gene therapy products. Our regulatory experts specialize in developing robust regulatory strategies for both the US and EU markets, ensuring compliance with regional requirements. We also assist in planning evidence strategies for reimbursement, helping you navigate the complex landscape of healthcare reimbursement systems.

Our close collaboration with regulatory agencies includes providing advice and conducting pre-submission meetings to address any potential concerns or queries. We have extensive experience in the development and implementation of Risk Management Plans (RMP) and Risk Evaluation and Mitigation Strategies (REMS), as well as the preparation of Product Safety Management Files (PSMF). Our skilled medical writers contribute to the development of high-quality regulatory documents, including the authoring and publishing of clinical and scientific manuscripts.

Finally, we offer expert support in the submission and management of Biologics License Applications (BLA) for the US market and Marketing Authorization Applications (MAA) for the EU market. Partner with us to navigate the complex regulatory landscape, ensuring compliance and maximizing the potential for successful approval of your cell and gene therapy products.

Commercialization

The moment has come to bring your therapy to the world, and we understand the profound emotions that accompany this pivotal stage. At ProPharma, we will be your guiding light, helping you navigate the path to successful commercialization:

Product Launch

A symphony of excitement and anticipation fills the air as your therapy prepares to make its grand entrance into the world. Our commercialization experts will orchestrate a flawless launch, meticulously planning every detail to maximize impact and reach. We understand the transformative power of first impressions, and together, we will captivate the world with your therapy's potential.

ProPharma offers comprehensive support for product launches, ensuring a successful introduction of your cell and gene therapy products to the market. Our services encompass various crucial aspects, including process validation and continued process verification to ensure the reliability and consistency of manufacturing processes. We also provide guidance on Good Distribution Practice Quality Management Systems (GDP QMS) to ensure the integrity and safety of your products throughout the supply chain. Additionally, we assist in the implementation of compassionate use programs and post-authorization safety studies (PASS) to address the needs of patients and collect real-world evidence. Our consulting expertise extends to product lifecycle management, encompassing strategies to optimize product performance and adapt to evolving market dynamics. We also provide guidance on reimbursement strategies, helping you navigate the complex landscape of healthcare reimbursement systems to maximize access to your therapy. Our regulatory intelligence services keep you up to date with the latest regulatory requirements and trends. Lastly, we offer review services for promotional materials, ensuring compliance with regulatory guidelines in both the EU and US markets. With our comprehensive support, you can confidently navigate the product launch phase, ensuring a successful market entry for your cell and gene therapy products.

Global Patient Safety

The safety of patients is our unwavering priority. As your therapy spreads its wings across borders, we will ensure a harmonized global patient safety strategy. Our vigilant pharmacovigilance experts will establish robust systems to monitor and address any safety concerns swiftly and comprehensively. Trust us to safeguard the well-being of patients around the globe, allowing your therapy to shine as a beacon of hope.

ProPharma prioritizes global patient safety, offering comprehensive services to ensure the well-being of patients receiving cell and gene therapy products. Our expertise includes the establishment of Global Vigilance and Pharmacovigilance Quality Management Systems (GVP QMS) to ensure robust safety monitoring and reporting processes. We provide Medical Information (MI) inquiries support, offering 24/7/365 MI contact center services in over 30 native languages to address queries from healthcare professionals and patients. Our dedicated team handles the intake, processing, and aggregate reporting of adverse events and serious adverse events (AE/SAE) to regulatory authorities. We also have qualified personnel, including the EU Qualified Person for Pharmacovigilance (QPPV) and Local Persons for Pharmacovigilance (LPPV), who oversee the compliance of pharmacovigilance activities. Additionally, we utilize EudraVigilance, the European database for the collection and analysis of suspected adverse reactions, to enhance safety surveillance and reporting. With our comprehensive global patient safety services, you can be confident in the integrity and vigilance of your cell and gene therapy products, ensuring the utmost safety for patients worldwide.

Program Types

ProPharma provides comprehensive support for a wide range of cell and gene therapy programs, enabling the development and advancement of transformative treatments.

Our consulting and expertise extends to various program types, including:

• CAR-T cells (autologous and allogenic)
• TILs (Tumor-infiltrating lymphocytes)
• AAVs, dual AAVs
• Gene-editing
• Vector-based gene transfer
• Stem cells, MSC and iPSCs
• Therapeutic vaccines
• Tissue engineering products
• Combination products
• γδ T cells

Therapeutic Areas

Our team of cell and gene therapy consultants and experts possesses extensive experience across a diverse range of therapeutic areas, allowing us to provide specialized support tailored to your specific indications. We have a deep understanding of the unique requirements and complexities associated with different therapeutic areas. Whether your focus lies in oncology, hemato-oncology, or tissue-agnostic indications, we have the knowledge and expertise to support your program. Our therapeutic expertise includes:

• Oncology, hemato-oncology, tissue-agnostic indications
• Hematology: hemophilia B, hemophilia A, thalassemia, sickle cell disease, porphyria
• Inherited retinal disorders (IRDs)
• Autoimmune diseases
• Other rare diseases: paroxysmal nocturnal hemoglobinuria (PNH), phenylketonuria (PKU), GM2 gangliosidosis, Kra

Decentralizing the Manufacturing of Cell and Gene Therapy Products

Decentralizing the manufacturing of cell and gene therapy products is a transformative approach that brings production processes closer to the point of care or the patient population. Shifting away from a centralized manufacturing model aims to overcome challenges associated with personalized and patient-specific therapies. By localizing manufacturing, logistical hurdles, transportation issues, and time constraints can be minimized, ensuring patients receive timely access to life-changing treatments.

This decentralized approach offers numerous benefits, including enhanced accessibility, streamlined supply chains, and increased flexibility. Patients can receive therapies more efficiently, as manufacturing can be tailored to individual needs and administered promptly. However, decentralization also presents challenges such as maintaining consistent quality control, regulatory compliance, and establishing robust infrastructure at multiple locations.

At ProPharma, we embrace the significance of decentralizing cell and gene therapy manufacturing. With our expertise and industry insights, we help clients navigate the complexities of this emerging trend. Through strategic guidance, regulatory support, and operational excellence, we optimize manufacturing processes and unlock the full potential of decentralized models.

Together, we shape a future where innovative treatments are readily accessible, overcoming the limitations of centralized manufacturing and bringing hope to patients in need.