December 2023

BIOTHERAPIES NEWS: Highlights and Top Stories

BIOTHERAPIES AT AABB: Community and Association News

Cell Notes: A Monthly Column by Christina M. Celluzzi, PhD, MS, CABP(H), Director, Biotherapies at AABB

Reasons to Celebrate the Year in Cellular Therapies

As we approach 2024, it's worth reflecting on the remarkable progress made in the past year in cellular therapies (CT). From cutting-edge research to groundbreaking clinical trials to practical solutions, a few notable developments helped define the year:?

• New approaches showcasing the versatility for CT. Research targeted a broader range of diseases from neurological disorders to autoimmune conditions. A few examples include chimeric antigen receptor (CAR) T cells that increased the effectiveness of T cell–based immunotherapy treatments, engineered CAR T against lupus and autoimmune disease , and allogeneic neural stem cells in people with progressive multiple sclerosis .?
• Recommendations from The Cell and Gene Therapy (CGT) Consortium. Established in 2021, the CGT, a group of stakeholders from more than 50 organizations, including CGT manufacturers, treatment centers, collection sites and ecosystem partners (e.g., couriers, technology providers), assembled to define and recommend common processes, terminologies and data requirements. Recognizing that the number of investigational and commercial CGT will scale over the next several years, as will the number of manufacturer-specific processes and solutions, increasing the burden on academic medical centers, community hospitals, clinics, collection facilities and labs, the CGT Consortium delineated a framework . This framework defines common principles, terminology and user experiences for enrolling patients, ordering therapies, and collecting starting material in a standardized way to provide background information on opportunities to streamline communications between manufacturing and health care organizations from the health care professional’s end-user’s perspective.?
• Personalized cellular therapies and their continued momentum offer the potential for tailored therapeutic solutions for various diseases. Made possible by new technologies that enable safety and performance of scale, advances in understanding individual patient profiles allowed for more precise and effective treatment strategies. ?
• CRISPR technology and targeted gene editing in CT. There were exciting breakthroughs in refining and expanding the applications of CRISPR, with promise for treating a spectrum of genetic disorders. One example is sickle cell disease (SCD), for which several studies used CRISPR gene editing technology to address the underlying genetic mutation causing SCD. A notable approach involved modifying patients’ own hematopoietic stem cells using CRISPR to correct the genetic mutation responsible for SCD, which could potentially lead to the production of healthy red blood cells. ?
• Stem cell research . – Examining stem cells from different sources remains at the forefront, with ongoing efforts to advance our understanding of stem cells and their potential applications in medicine.?

Though not a comprehensive list of all advances, these achievements underscore the value of biotherapeutic approaches, building on extensive progress and providing platforms for advances yet to come.?

I extend a heartfelt congratulations to the dedicated professionals and researchers in the field of cell therapies for their relentless pursuit of scientific excellence in 2023. Your contributions have been instrumental in shaping the future of health care.?

As we look forward to 2024, let this be an invitation to aspiring scientists and health care professionals to explore the exciting possibilities within the realm of cell therapies. May the achievements of the past year inspire new minds to join this dynamic field, bringing fresh perspectives and innovative solutions to further propel the transformative journey of cell therapies. Here's to a future filled with discovery, collaboration and the collective pursuit of advancing medical frontiers for the benefit of all.?

AABB to Attend Phacilitate’s Advanced Therapies Week in Miami ?

AABB looks forward to connecting with you at Advanced Therapies Week in Miami, taking place Jan. 16-19. Organized by Phacilitate , Advanced Therapies Week is dedicated to helping biotech companies progress on their commercialization journey and pushing the field closer to delivering more life-changing treatments to patients.?

At the event this year, AABB will host an expert-led roundtable discussion: The Need for Well-Qualified Personnel. The discussion, co-hosted by Jeff Wren , AABB’s vice president of biotherapies, and WANXING CUI , MD, PhD, CABP. ?

Cui leads the Cell Therapy Manufacturing Facility at MedStar Georgetown University Hospital , blending a diverse background in academia, industry and health care in the realm of cell therapy. As director of an FDA -registered facility accredited by AABB, Cui plays a crucial role in the pancreatic islets and hematopoietic progenitor cells transplantation programs at his institution.?

Within AABB, Cui is actively involved in the Cellular Therapies Section Coordinating Committee , contributing to the advancement of cell therapy standards. AABB is honored to have him attend Phacilitate’s Advanced Therapies Week 2024 with ABBB this year.?

Whether at one of our upcoming conference attendances or virtually at any time at your convenience, book a meeting with AABB via the contact form on our biotherapies virtual booth page .?

In Conversation With: Debra’s BenAvram, CEO at AABB: Working Together to Ensure Safety, Quality and Innovation for the Future of Biotherapies?

In anticipation of this year’s Advanced Therapies Week, Phacilitate sat down with AABB ’s CEO Debra BenAvram for a written interview. In this blog post, she shares some of her 2023 highlights when it comes to AABB’s mission to advance biotherapies around the world.?

Among other projects, she highlights AABB’s Certified Advanced Biotherapies (CABP) Certification Program and how it has grown in adoption since its inception in 2022. She shares her personal advice for professionals considering pursuing the certification in 2024 and encourages biotherapies organizations to reach out and learn more about how they can benefit from partnering with AABB.?

"As the certification benefits our entire field, it will be a continued goal for AABB throughout 2024 to get more people to sit for the exam. That’s why we’ve also been working on various ways to make the exam more approachable for those who qualify.?

In late 2023, we launched a new online exam pre-approval questionnaire for the CABP. By completing a few short questions applicants can hear back from AABB to find out if they are pre-approved for the exam. You can find the questionnaire and all the information about the CABP on our website.

I would say to anyone interested in the CABP to go for it – it’s the perfect professional goal for 2024!"- said BenAvram.

Read the full blog post on the Phacilitate website.?

Mark Your Calendars: Improving HPC Inventory Management eCast Jan. 17?

On Wednesday, Jan. 17, ?2-3 p.m. ET, AABB will host the following eCast: Improving HPC Inventory Management - Considerations Regarding Consent and Discard of Cryopreserved HPC Products (24EL-402).?

Moderated by Christina Celluzzi, PhD, MS, CABP(H) , PhD, MS, CABP(H), director, biotherapies, at AABB , with speaker Melissa Marlowe , CHS, MBA, vice president of clinical services administration at Vitalant , this intermediate-level program is intended for directors, laboratory staff, managers/supervisors and medical directors. It will address challenges that labs face related to the long-term storage of cryopreserved products and inventory management. The program will discuss examples of consent documents ?and how expectations for length of storage have changed over the past 10-15 years. It will also use case studies to demonstrate challenging situations that the lab/storage program may face and options to consider while evaluating a product for discard.?

After participating in this educational activity, participants should be able to:?

• Identify key elements of a strong consent document.?
• List common challenges faced by Cellular Therapy storage programs when evaluating products for discard.?
• Describe how changing clinical and industry expectations impact storage terms and approval for discard.?

This activity may be eligible for continuing education credit/contact hour and general participation credit. Please refer to the program page for further details.?

Registration includes access to both the live and on-demand version of this eCast and is priced as follows:?

• Single viewer registration for non AABB members $69?
• Single viewer registration for AABB members $59?

For facilities and organizations interested in participating in this eCast, please complete the group viewing registration form on the AABB website.?

Thank You to AABB’s Corporate Partners for Another Great Year?

AABB ’s established and highly successful Corporate Partner Program is a flagship offering within AABB’s corporate services portfolio. The program connects organizations with decision makers within the blood and biotherapies field – at more than 1,300 AABB-accredited institutions worldwide – while showcasing their commitment to advancing quality patient care.?

This year, AABB also announced changes to the program to make it more available to companies operating within the biotherapies field.?

“AABB’s Corporate Partner Program has been successful in providing companies with direct access to AABB resources and industry expertise,” said Jeff Wren , vice president, biotherapies. “AABB is ensuring that any organization working to advance the biotherapies field can now access these unique and invaluable benefits.”?

"With the support of our corporate partners, we've launched pioneering initiatives in workforce development and strategic investment. Together, we're poised to address crucial issues, fostering innovation, and positioning your companies at the forefront. Let's seize this opportunity to shape the future through impactful programs as we head towards the 2024 strategic planning discussion" said David Green , MSA, president and CEO of Vitalant , past president of AABB and Corporate Partner Program ambassador.?

"AABB's corporate partners remain integral to our organization, playing a pivotal role in advancing the blood and biotherapies field's health. In 2023, we've strived to deliver exceptional value through our program, fostering numerous collaborative opportunities to ensure our communities thrive. We eagerly anticipate your continued partnership in 2024, as together, we shape the future of our workforce and make a lasting impact," said AABB’s CEO Debra BenAvram .?

Thank you to all AABB corporate partners and premium corporate partners.?

For more information on the program or other ways to partner with AABB, contact Margie Boraz , AABB’s business development director, at [email protected] .?

AABB BIOTHERAPIES NEWSFEED: More Updates from the Field

Read more from AABB's Newsfeed , an essential resource offering the latest news, analysis, commentary and updates on critical topics for the blood and biotherapies community.

AABB MEMBERS ZONE: Biotherapies Resources and Events

AABB Update on Cellular Therapies Subsection Activities?

As a benefit, AABB members can enroll in subsections that meet monthly to enrich their professional experience and to keep informed about happenings in the field of cellular therapy. Some recent highlights of a few of the subsections include:?

• Asia-Pacific Group (APG) members discussed a recent publication from Dr Sadhana Mangwana , MD, MBA, “Experience of Granulocyte Collections and Transfusions in Resource-Constrained Settings .” Granulocyte transfusions are an important therapeutic modality in neutropenic patients with resistant infections, until spontaneous recovery of neutrophil count occurs in patients. The paper highlights the vital role of granulocytes in improving outcomes and saving patients in resource-constraint countries, where there is increasing emergence of multidrug-resistant bacterial infections. The use of artificial intelligence (AI) as a tool to increase productivity was another hot topic of discussion with APG members.?
• Cord Blood Subsection members discussed ways to introduce biotherapies-cell therapies as a career option to those unfamiliar with the field. Other discussion focused on transportation and shipping issues to ensure that products reach their destinations safely. AABB participated as an inspiring partner in World Cord Blood Day, created by Save the Cord Foundation - Cord Blood Education from a 501c3 non-profit and held on Nov. 15, to celebrate this life-saving gift. Thank you to all who participated in the celebration and please look out for information for the event next year.?
• CT-CET Emerging Topics (CT-CET) focused on “NK cell manipulation for enhanced immunotherapy” presented by Rizwan Romee , MD, associate professor of medicine at Harvard Medical School , Dana-Farber Cancer Institute .?
• AABB has a subsection for its Spanish-speaking members. The Spanish Language Subsection (SLS) meets the third Wednesday of each month at 5 p.m. ET and is looking for more member participation. Past presentations can be accessed on the AABB subsection website .?

Learn more information about subsections on the AABB website .?

Remember to subscribe to CellSource, share and comment.

Not an AABB Member?

Join AABB to gain access to essential benefits, including professional development opportunities, critical tools and resources, and opportunities to collaborate with leaders in the field. Be a part of a diverse community of health care professionals working to make transfusion medicine and biotherapies safe, available and effective worldwide. Find out more on the AABB website.