Molecular Therapy

Meet the Editor: Gerhard Bauer, Molecular Therapy—Methods & Clinical Development

ASGCT Staff - November 25, 2019

ASGCT member and director of the Good Manufacturing Practice (GMP) laboratory at the UC Davis Institute for Regenerative Cures Gerhard Bauer will become the editor-in-chief of ASGCT's journal, Molecular Therapy—Methods & Clinical Development on January 1, 2020.

ASGCT recently announced the appointment of Gerhard Bauer as editor-in-chief of one of the Society’s field-leading journals, Molecular Therapy—Methods & Clinical Development, effective January 1, 2020. Get to know Bauer and his plans for the journal as part of this enlightinging Q&A.

Bauer replaces Roland W. Herzog, incoming editor-in-chief of Molecular Therapy, at the helm of Molecular Therapy—Methods & Clinical Development after serving as an associate editor of the same journal for more than four years. Bauer is an assistant professor of hematology and oncology and director of the Good Manufacturing Practice (GMP) laboratory at the UC Davis Institute for Regenerative Cures.

What drew you to become the editor-in-chief of Molecular Therapy—Methods & Clinical Development?

I was approached by the leadership of ASGCT to consider applying for the position based on my track record in the field of cell and gene therapy. I have been a member of ASGCT since its inception, and I have also seen the journal Molecular Therapy grow from very humble beginnings to an outstanding, high impact factor journal that is tremendously respected, nationally and internationally. For several years now I have been serving as the associate editor of Molecular Therapy—Methods & Clinical Development and have gotten to know this journal very well. I was truly honored to hear, after a thorough interview, that the committee had voted for me to become editor-in-cheif. This position will allow me to continue working on improving Molecular Therapy—Methods & Clinical Development's content, impact factor and visibility.

Can you tell me about your career path and expertise? How does someone get to the point at which they’re leading a journal?

I have a long career in the development, manufacturing and regulatory aspects of biologics and cell and gene therapy products. This started in the early 1980s when I worked for the Austrian FDA, being in charge of the safety of blood products (extremely important during the first wave of the HIV epidemic) and eventually in charge of the regulatory evaluation of tests for the detection of HIV. When I saw many of my friends die from HIV and AIDS, I wanted to make a difference and work in HIV research to perhaps find a way to cure HIV infection. I ended up being hired at the University of Maryland, where I ran the HIV laboratory trying to find out how HIV is transmitted from mother to child; then I was hired by the Johns Hopkins University where I worked on the very early development of hematopoietic stem cell gene therapy for HIV, trying to create an HIV resistant immune system.

In the early 1990s I was then hired by Dr. Donald Kohn at Children’s Hospital Los Angeles (CHLA) to continue working on stem cell gene therapy for HIV, preparing for stem cell gene therapy clinical trials and pioneering the procedures for the clinical grade manufacturing of cell and gene therapy products going into patients. I was very lucky at that time that I was able to work with the leaders in the field of gene therapy: Kohn, as well as Dr. John Rossi and Dr. John Zaia at City of Hope, where the adult patients for our clinical gene therapy trials were treated. I then helped to design and build the GMP facility at CHLA where all cell manufacturing took place.

I was able to put GMP grade cell transduction procedures in place at that time that are still being used today, almost unaltered, for the transduction of hematopoietic stem and progenitor cells, leading to clinical efficacy.  In 2002 I was asked to move to Washington University in St. Louis to design, build and run a new GMP facility for cellular and gene therapy manufacturing. I accomplished this task successfully, but was called back to California in 2006 to build a large, six manufacturing room GMP facility at the University of California Davis and direct it. I made several improvements to academic GMP facility design and operation, developed a GMP compliant fluorescent activated cell sorter (FACS) for clinical product sorting and switchable room pressurization in GMP manufacturing suites, so aerosolizable product manufacturing, such as gene therapy vector manufacturing and cell sorting via FACS could be safely conducted.

Up to the present, many novel cell and gene therapy products have come out of my facility, and many patients have been treated with often life saving products, such as FACS sorted, engineered bone marrow grafts and CAR T cells for hematologic malignancies. The UC Davis GMP facility serves both academic and industry clients and has become a highly valued resource within the state of California and also nationwide, we currently manufacture 15 different products with the appropriate spatial and temporal segregation required for a multi-use facility.

During my career, I have written a large number of journal articles, many of them highly cited, also several text book chapters, and I developed and taught the first graduate class in the nation in cell and gene therapy GMP manufacturing practices. Since 2011, this class has educated over 100 graduate level scientists, many of them have gone on to careers in the stem cell and gene therapy field. All of these efforts at UC Davis have been made possible by funding received from the California Institute for Regenerative Medicine (CIRM); we were successful in winning grant funding to build the GMP facility, then support clinical cell and gene therapy manufacturing efforts, plus the educational efforts.

With all of this experience, I have been able to help Molecular Therapy—Methods & Clinical Development as an associate editor for a good number of years, and I believe I will be able to lead the journal well in the coming five years.

What is it about gene and cell therapies that inspire your work?

As you may have seen from my previous answer, my inspiration re. cell and gene therapies was fueled by the desire to find a cure for HIV, achieved not by taking antiviral drugs daily, but by engineering an immune system that would be resistant to HIV and could eliminate HIV infected cells for the rest of the life of the patient. Prior to the onset of highly active antiretrovirals, this was a critical endeavor, only achievable by cell and gene therapy. However, after many years of working on stem cell gene therapy for HIV, I came to the realization that I had also made other approaches of clinical stem cell gene therapy viable by applying the GMP grade transduction procedures I had developed to other therapeutic products. Additionally, in the GMP facilities I built, many different cell and gene therapy products can be manufactured to treat a wide variety of diseases. In addition, when the gene therapy downturn after the year 2000 happened, I worked diligently on GMP grade culture and transduction procedures that would satisfy the regulatory agencies and would lead to clinical efficacy. Also, efficient lenti- and retroviral vector manufacturing in an academic GMP facility was a major goal I wanted to accomplish. Looking back now, we, at the UC Davis GMP facility have achieved a lot of these milestones, and the field overall has made a huge impact by saving many lives. I am so glad I stayed with cell and gene therapy and was able to help develop it to the point where it is now.

What separates Molecular Therapy—Methods & Clinical Development from other journals covering gene and cell therapies?

This journal is extremely helpful and valuable when it comes to publishing research that enables investigators in our community to further the clinical development of cell and therapy clinical applications. There is often quite a gap to bridge between basic research and clinical applications of cell and gene therapy. Over the years that I have been looking at manuscripts submitted to the journal, I have always loved the articles dealing with translational research, getting the products ready for clinical applications, improving their utility and solving problems in the manufacturing of these products. Now, at a time where we have clinical efficacy and also commercially approved cell and gene therapy products that save lives, it is even more important to strengthen the translational research field. Molecular Therapy—Methods & Clinical Development is set up perfectly to doing exactly that and should be able to position itself in the forefront of this activity.

What challenges face the field, and how can the Molecular Therapy family of journals help address them?

Many new cell and gene therapy products will reach efficacious clinical application status in the near future, particularly in the treatment of malignancies other than the ones currently targeted by already commercialized gene therapies. I believe we need to focus on fostering this new research, the development of efficacious treatments based on cell and gene therapy, solicit and publish articles that deal with this subject matter and encourage the community to keep improving existing cell and gene therapies. A very important challenge to the field will be the broad availability of these novel cell and gene therapy products, and patients having equitable access to these treatments.

The current price of gene therapy applications that are possibly curative is way too high. I believe we need to encourage the community to come up with solutions that will allow safe and affordable gene therapeutics to be made available to a large number of patients. This can be done by scaling out manufacturing, developing closed system and decentralized product manufacturing. Will we need new GMP facilities or can we perhaps use isolator and closed system technology to manufacture these products in more remote areas? Can we bring this technology to developing countries and to underserved populations? This is something we should think about.

Is there anything else you would like to tell the ASGCT community about yourself or your vision for the journal?

After having seen the development of cell and gene therapy from the very beginning, having gotten to know the pioneers in the field personally and treasuring their friendship, and having been very fortunate also to be involved in shaping our field, particularly the area of GMP manufacturing of cell and gene therapy products, I could not think of any other and more exciting field than ours, the one I grew up in. It is such a reward to now see cell and gene therapy matured and having cured formerly incurable diseases. I would like to be part of this exciting community as long as I am able, however, at the same time, would like to pass on my knowledge to as many young scientists as possible. I believe cell and gene therapy has a very bright future, and we definitely need a good number of new scientists that will join us and develop it further, perhaps much further than I can envision now, for the benefit of our patients.

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