The Vector
Volume 8, Issue 10: October 2019
Editorial Team
Melvin Rincon, MD, PhD – Editor, The Vector
Edith Pfister, PhD – Associate Editor, The Vector
Karen Bulaklak, PhD – Junior Editor, The Vector
Inside This Issue
Leadership Message
Breaking Through
Society News
Career Center
Public Policy
Industry News
Leadership Message
ASGCT Returns to Washington, D.C. for Expanded Policy Summit
After the success of ASGCT’s first-ever Value Summit last year, we’re looking forward to going back to Washington, D.C. on November 4-6 for an expanded, three-day Policy Summit. Over the past year, the gene therapy, cell therapy, and gene editing fields have continued their rapid expansion, and hundreds of therapies are currently in the pipeline. This amazing progress in the development of transformational treatments for those with devastating diseases comes with continued refinement around how we think of global regulatory, access, and ethical issues. Building on the important work done by organizations over the past decade, the ASGCT Policy Summit will bring together many of the stakeholders that have a critical voice around the responsible use of this technology.
During this year’s Policy Summit, we’ll hear from—and, importantly, listen to—a variety of perspectives across the field, including those from international regulators, bioethicists, patient advocates, scientists, clinicians, and biotechnology leaders. We all certainly won’t agree on every issue. But by expanding the scope of the program and including more diverse viewpoints, we hope to improve the dialogue, processes, and policies surrounding the development of gene therapy, gene editing, and cell therapy technologies. Our speakers come from different parts of the field, but I believe are united by a common mission: alleviating human diseases by delivering these potentially transformative treatments to patients for whom the status quo too often means death or serious disability.
The first day of the Summit will focus on global regulatory issues that affect the development of gene therapy across the world. The keynote speaker that day will be Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. On the second day, speakers will address the real-world challenges of, and possible solutions for, access to gene therapies within the major U.S. health programs: Medicaid, Medicare, and commercial plans. The third and final day is dedicated to current and future frameworks governing germline gene editing around the world. Speakers will explore a range of topics, including whether we should proceed someday with germline gene editing. They will also examine potential mechanisms for oversight of germline gene editing and ways to achieve societal consensus. Our keynote speakers on the final day will be NIH Director Francis Collins, M.D., Ph.D., and former FDA Commissioner Margaret Hamburg, M.D., co-chair of an important World Health Organization Commission on gene editing.
Given the remarkable progress in the field over the past decade, we believe these three major topics— global regulation, access, and ethics—will be central features that help shape the next decade of gene therapy, cell therapy, and gene editing treatments.
I hope to see many of you at the ASGCT Policy Summit to participate in these important discussions. We are offering daily registration so you can tailor your experience to your interests and expertise. Thank you for your commitment to the field.
Sincerely,
Tim Hunt, J.D.
Chair, ASGCT Government Relations Committee
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Breaking Through
Gene-Loaded, Nanoparticle-Coated Sutures Provide Effective Gene Delivery to Enhance Tendon Healing
Zhou YL, Yang QQ, Yan YY, Zhang L, Wang QH, Ju F, Tang JB.
DOI: https://doi.org/10.1016/j.ymthe.2019.05.024
Summary by Karen Bulaklak, Ph.D.
Tendon injuries are a common problem found in the clinic due to trauma or excessive exercise. Serving as a linkage between the muscle and bone, the tendon is a vital component in the functioning of the musculoskeletal system. Unfortunately, the tendon structure and scarcity of cells and growth factors lead to slow healing times after injury and pose significant barriers for creating therapies. Researchers at the Nanomedicine Research Laboratory in Nantong, China have devised a clever way to promote tendon healing by combining gene therapy with materials that are already used in the clinic to repair the tissue: nanoparticle-loaded sutures.
First, the group generated nanoparticles from PLGA, a biocompatible and biodegradable material, and complexed these with plasmid DNA encoding basic fibroblast growth factor (bFGF) and vascular endothelial growth factor A (VEGFA). These genes have been shown previously to enhance tenocyte proliferation, angiogenesis and collagen synthesis. This approach has an advantage over direct plasmid application, as the injected volume is limited by the compact tendon tissue. The researchers confirmed efficacious gene delivery in chicken tendon and rat tendon cells (58% and 61%, respectively) using these nanoparticles. The nanoparticle/plasmid complexes were adhered to both non-absorbable and absorbable sutures commonly used for repairing tendons. Scanning electron microscopy revealed that the complexes were evenly distributed along the surface of the sutures and remained bound even after stitching. Furthermore, the bound nanoparticles released their DNA cargo in a sustained fashion due to the slow degradation of PLGA.
The researchers next evaluated the therapeutic benefit of their gene-modified sutures in chicken and rat injury models. After treatment with absorbable and non-absorbable complex-loaded sutures, significant increases in bFGF and VEGFA protein expression were observed in tissue. Various functional assays were performed to determine the condition of the repaired tendon, including tendon gliding excursion and ultimate strength. Significant functional improvements were observed as early as 1 week post-treatment in the rat injury model, which continued to increase with time. The chicken injury model showed notable improvements as early as 2 weeks post-treatment with continued benefit at later time points as well. No major differences were observed between the performance of the absorbable and non-absorbable complex-loaded sutures.
In summary, the authors present a novel approach that overcomes tendon-specific therapeutic barriers, which can also be seamlessly implemented in the clinic. These gene-modified sutures provide hope for decreased recovery time and a better treatment option for those who are affected by tendon injuries.
Society News
Dr. Margaret Hamburg Will Speak on Germline Gene Editing at the ASGCT Policy Summit
Margaret Hamburg, M.D., an internationally recognized expert in medicine and public health, will be one of the keynote speakers on November 6, the third and final day of the ASGCT Policy Summit in Washington, D.C. The program that day will focus on Ethical, Societal, and Policy Issues in Germline Gene Editing.
Bridging the Gap Between Scientists and Patients in Gene Therapy Development
In an article originally appearing on Cell & Gene, ASGCT Executive Director David Barrett discusses the challenges of bridging the gene therapy knowledge gap between scientists and patients. Scientists need to be cautious and honest about the information they provide, and they need effective ways to broadcast this information so that it reaches the patients who need it.
ASGCT Will Exhibit at NORD Summit in Washington, D.C.
ASGCT staff will be exhibiting at the National Organization for Rare Disorders (NORD) Rare Diseases & Orphan Products Breakthrough Summit in Washington, D.C. on October 21-22. The purpose of ASGCT’s engagement in this program is to inform attendees about our reliable, accurate, patient-friendly resources on gene therapy. Additionally, staff will be promoting the Annual Meeting, our Clinical Trials Finder, and ASGCT advocacy efforts. If you missed the first two education webinars from ASGCT and NORD, watch “Gene Therapy: Yesterday, Today and Tomorrow,” "The Science Behind Gene Therapy," and mark your calendar for future webinars.
Career Center
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Public Policy
House Resolution 606 Includes ASGCT-Recommended Language
Congresswoman Barbara Lee (D-CA), along with 11 bipartisan cosponsors, included ASGCT-recommended language in House Resolution 606, a sickle cell trait resolution introduced on September 27. The resolution encourages federal action on sickle cell trait research, surveillance, and public education and awareness. ASGCT recommended the inclusion of language recognizing the importance of the development of, and access to, new treatments for sickle cell disease, as well as ensuring equitable access to new treatments among all economic, racial, and ethnic groups. While many of ASGCT’s policy impacts are behind the scenes, in this case the Society directly influenced a piece of federal legislation.
ASGCT Co-Hosts Congressional Briefing on CAR T-Cell Therapy
ASGCT cohosted a briefing on Medicaid for CAR T-cell therapy on September 17 with the American Society for Transplantation and Cellular Therapy, the American Society of Hematology, and the Lymphoma Research Foundation. The Congressional Caucus to Cure Blood Cancers, including co-chairs Representatives Gus Bilirakis (R- FL) and Doris Matsui (D-CA), were also hosts. Dr. Shannon Maude and Dr. Krishna Komanduri gave an overview of the science of CAR T-cell therapy and spoke about the following key policy issues:
- Only about half of states have publicly available Medicaid coverage policies for CAR T-cell therapy, and some states have coverage restrictions not consistent with labeled indications, such as site of care restrictions or restricting to trial eligibility criteria.
- Reimbursement levels under Medicaid vary by state, with most reimbursing through bundled payments that only reimburse enough for services, not the biologic product, leaving providers insufficiently reimbursed for the therapy.
- Some providers, such as the University of Miami, are not providing CAR T-cell therapy to Medicaid patients due to the unsustainable losses.
Requests for legislative staff included to request their state Medicaid program for its written coverage and reimbursement policies for approved gene and cell therapies, and to ensure inclusion of CAR-T therapies in proposed new payment models. ASGCT will continue to monitor and support policies that enhance patient access to CAR T-cell therapies for Medicaid beneficiaries.
Society Affirms Position on Clinical Use of Germline Gene Editing
The International Commission on the Clinical Use of Human Germline Genome Editing, convened by the Royal Society and the US National Academies of Sciences and Medicine, published a call for evidence about the clinical use of germline gene editing in August. In ASGCT comments submitted on September 27, the Society reiterated its position that any clinical use of germline gene editing is inappropriate at this time, and that such use should not be revisited until a comprehensive dialogue takes place that includes members of patient, caregiver, scientific, medical, ethical, cultural, and other civil society organizations and communities. Our comments outlined the ethical and scientific concerns that would need to be addressed before the status quo on this issue is revisited including, but not limited to, optimizing the efficiency and precision of on-target modification; defining and minimizing off-target mutations; preventing on- and off-target mutation mosaicism; understanding how novel on- and off-target mutations might interact with existing human genetic diversity when these new alterations are passed on to future generations; and addressing intergenerational medical and ethical implications of human germline genome editing. Finally, ASGCT advocated for a balance between the strong international governance required to prevent abuses of gene editing technology and the sufficiently flexible regulatory environment necessary to allow somatic cell treatments to be developed.
Industry News
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