11INFLUENTIAL WOMEN IN ADVANCED THERAPIES

11 Influential Women in Advanced Therapies | 2

TThere’s no question about it, the advanced therapies industry is an exciting place to be and is growing ferociously. We’ve gone past the dream of commercialised products into the stark reality of manufacturing challenges, scaling up, scaling out and, crucially, getting treatments to patients that need them.

Advanced therapies are going to change the standard of healthcare and practitioners will be able to consider a diverse portfolio of treatments for their patients. There are still challenges that the industry is working to resolve, some scientific, some technical and some are economic.

However, one hurdle that is proving tough to conquer is more of a human challenge; talent and the lack thereof. Do a quick search on LinkedIn and you’ll see that some of the major advanced therapy developers are looking to enhance their workforce. Kite Pharma and bluebird bio both have 25 vacancies, Orchard Therapeutics and Adaptimmune are advertising 17 positions each and Oxford BioMedica has 24 positions available.

How will the industry bridge this talent gap as it continues to grow? Diversity is certainly central to solving this problem and also makes great business sense, in fact, we now have a wealth of research demonstrating a direct correlation between high-skilled immigration and an increase in the level of innovation and economic performance in cities and regions. For example, Douglas McWilliams’ book ‘The Flat White Economy’ describes immigration as the UK’s (and London in particular) secret weapon.

Gender, of course, plays a significant role in diversity. However, women are generally underrepresented, particularly in leadership roles, with only 4% of the S&P 500 CEOs being women. Attracting and retaining more women in the STEM workforce will maximise innovation, creativity, and competitiveness.

To inspire and attract female talent it’s important to celebrate and value the contribution of women in science. We’ve seen women being awarded the Nobel Prize in Science, Chemistry and Medicine four times in the last six years, which is progress, albeit slow progress. As Sheryl Sandberg famously said, “you cannot be what you cannot see…”. So, for all the women and girls looking towards the advanced therapies industry, and even the wider STEM industry, here are 11 of the most influential women in advanced therapies today.

I’d like to extend a huge thank you to Jana Stoudemire, Delfi Krishna and Sara Mills for their

contribution to this list, as well as being mentors for the Women in Advanced Therapies mentorship programme hosted by Phacilitate.

This is dedicated to all the amazing women in our equally amazing industry. Enjoy!

Fore word

Nicola AmblerEditor, Phacilitate:Exchange

11 Influential Women in Advanced Therapies | 3

Tara Britt, Associate Chair / Founder and President, NC Rare Disease Advisory Council / Rare Disease Innovations Institute, Inc

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Jennifer A. Doudna, Li Ka Shing Chancellor’s Professor of Biomedical Science, University of California, Berkeley

Maria Fardis, PhD, MBA, President & Chief Executive

Officer, Iovance Biotherapeutics, Inc.

Catriona Jamieson, M.D., Ph.D., Professor of Medicine, Division of Hematology-Oncology, UC San Diego Moores Cancer Center

Jeanne F. Loring, PhD, Director of the Center for Regenerative Medicine and Professor Emeritus at the Scripps Research Institute, La Jolla, California.

Martina Schussler-Lenz, Chair of the Committee for Advanced Therapies at the European Medicines Agency (EMA)

Christi Shaw, CEO, Kite, a Gilead Company

Dr Samantha Yammine, PhD, neuroscientist, science communicator and digital media producer

Ran Zheng, CTO, Orchard Therapeutics

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Contents

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Emily Whitehead, cancer patient and Emily Whitehead Foundation

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Claudia Zylberberg, CEO, Akron Biotechnology

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Tara BrittAssociate Chair, NC Rare Disease Advisory Council and Network Founder and President, Rare Disease Innovations Institute, Inc.

Find out more about RDII

Get more information about the council and the network here

Jennifer A. DoudnaLi Ka Shing Chancellor’s Professor of Biomedical Science, University of California, Berkeley

Tara previously worked in the finance, human resources and policy arena before becoming Associate Chair of the NC Rare Disease Advisory Council. She worked with researchers and clinicians in rare disease at the University of North Carolina Gene Therapy Center where she engaged with patient advocates supporting research in Gene Therapy.

Tara currently serves as Associate Chair of the North Carolina Rare Disease Advisory Council and Network. The council was created as a result of co-authoring legislation with a North Carolina patient advocate to create a Rare Disease Advisory Council signed into law, August 2015. Tara has created a unique rare disease ecosystem within the state that brings together patients, caregivers, academics, clinicians, foundations, industry and any entity in rare disease.

Tara is also the President and Founder of Rare Disease Innovations Institute (RDII). RDII is a global non-profit focused on educating, engaging and equipping the rare disease community to achieve a higher quality of life, accelerating diagnosis and enabling access and treatment. Through this non-profit and the rare disease network, RDII has developed exclusive toolkits for other states to build upon the learning and success of North Carolina enabling creation of successful councils and unique rare disease networks. The toolkits are state tailored to the unique rare disease population. RDII also partners with rare disease advocacy groups, academia, foundations, healthcare systems, state and federal government and industry to build disease specific models to support targeted disease populations and the challenges to accessing care and managing their disease. These models are currently developed in North Carolina and can be modelled globally by the RDII team.

Jennifer Doudna is the Li Ka Shing Chancellor’s Chair and a Professor in the Departments of Chemistry and of Molecular and Cell Biology at the University of California, Berkeley, as well as an investigator of the Howard Hughes Medical Institute.

Best known for her co-discovery of CRISPR-Cas9 genetic engineering technology, with collaborator, French scientist Emmanuelle Charpentier, Doudna has been instrumental in changing the face of genomics research. In fact, it is considered to be one of the most significant biological discoveries in history and has the potential to chang the face of healthcare.

In addition to her scientific achievements, Doudna is also a leader in public discussion of the ethical and other implications of genome editing for human biology and societies and advocates for thoughtful approaches to the development of policies around the use of CRISPR-Cas9. She has received many prizes for her discoveries, including the Japan Prize (2016), the Kavli Prize (2018), and the LUI Che Woo Welfare Betterment Prize (2019). In 2015, Doudna was named by Time Magazine as one of the 100 most influential people in the world.

Doudna has received many prestigious awards and fellowships including the 2000 Alan T. Waterman Award for her research on the structure as determined by X-ray crystallography of a ribozyme and the 2015 Breakthrough Prize in Life Sciences for CRISPR-Cas9 genome editing technology (with Charpentier). She has been a co-recipient of the Gruber Prize in Genetics (2015), the Canada Gairdner International Award (2016) and the Japan Prize (2017).[15]. Outside the scientific community, she has been named one of the Time 100 most influential people in 2015 (with Charpentier) and she was listed as a runner-up for Time Person of the Year in 2016 alongside other CRISPR researchers.

Here’s more about what Jennifer is doing in her lab currently.

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Maria FardisPhD, MBA, President & Chief Executive Officer, Iovance Biotherapeutics, Inc.Dr Fardis has extensive experience in drug development and novel cancer treatments. Prior to joining Iovance Biotherapeutics, Dr.Fardis was chief operating officer at Acerta Pharma working on the development of CALQUENCE® (acalabrutinib), until the company’s acquisition by AstraZeneca. Before joining Acerta, Dr Fardis held the position of chief of oncology operations and alliances at Pharmacyclics where she oversaw development of IMBRUVICA® (ibrutinib). She was a key contributor in the creation of a broad clinical program for ibrutinib, as well as NDA and MAA submissions. Previously, Dr Fardis held a number of key scientific and management roles at Gilead Sciences. At Gilead, she was involved with multiple therapeutic areas including antivirals, oncology, and cardiovascular therapeutics and worked on the development and life cycle management of LETAIRIS® (ambrisentan). She received her PhD in organic chemistry from UC Berkeley and holds an MBA.

Currently, Fardis is CEO and President at one of the most innovative biotechnology companies in the world. She leads a team that are using tumour infiltrating lymphocytes (TILs) to Enables and enhances a patient’s own robust immune response to treat cancer. This is opening doors to treating solid tumours, whereas the clinical successes so far have been with haematological malignancies.

Fardis joined Iovance as CEO back in 2016, when it was named Lion Biotechnologies. She has since grown the company from 20 people to around 100 in 2019 with plans to increase by a further 25% to execute her regulatory and commercialisation plans. Iovance’s lead candidate, lifileucel, is now at pivotal trial stage after receiving fast track designation from the FDA.

Catriona JamiesonM.D., Ph.D., Professor of Medicine, Division of Hematology-Oncology, UC San Diego Moores Cancer Center

Catriona Jamieson, M.D., Ph.D., is Professor of Medicine in the Division of Hematology-Oncology, Deputy Director at UC San Diego Moores Cancer Center, Koman Family Presidential Endowed Chair in Cancer Research, Chief of the Division of Regenerative Medicine, Deputy Director of the Sanford Stem Cell Clinical Center, Co-Leader of the Hematologic Malignancies Program, and Director of Stem Cell Research at UC San Diego Moores Cancer Center.

Jamieson specialises in myeloproliferative disorders (MPDs) and leukaemia. Myeloproliferative neoplasms are a family of uncommon but not rare degenerative disorders in which the body overproduces blood cells. Myeloproliferative neoplasms can cause many forms of blood clotting including heart attack, stroke, deep venous thrombosis, and pulmonary emboli and can develop into acute myelogenous leukaemia. Although some effective treatments are available, they are laden with serious side effects. In addition, individuals can become resistant to the treatments. Dr Jamieson studies the mutant stem cells and progenitor cells in myeloproliferative neoplasms. These cells can give rise to cancer stem cells. Cancer stem cells may lie low to evade chemotherapy and then activate again later, causing disease progression and resistance to treatment. Her goal is to find more selective, less toxic therapies.

More about Catriona’s research

Find out more about Iovance’s TIL platform

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Jeanne F. LoringPhD, Director of the Center for Regenerative Medicine and Professor Emeritus at the Scripps Research Institute, La Jolla, California

Another California-based scientific leader makes the list. Jeanne Frances Loring is an American stem cell biologist, developmental neurobiologist and geneticist. She is also the director of the Center for Regenerative Medicine and professor at the Scripps Research Institute in La Jolla, California. Loring joined The Scripps Research Institute in 2007, where her research is currently focused on human pluripotent stem cells, a remarkable cell type made by reprogramming adult cells to an embryonic state, making them capable of developing into all of the cell types in the body. She has published over 100 peer-reviewed scientific articles, which have been cited nearly 5,000 times and holds five patents on transgenic methods, Alzheimer’s disease and stem cells.

After completing her doctoral work, Loring spent five years studying and lecturing on embryology and neurobiology at UC Davis before moving to Hana Biologics in 1987. As staff scientist at Hana Biologics, Loring’s work included study of cell therapy for Parkinson’s disease. Loring remained in the biotech industry and began to focus more on genomics, working as a senior scientist at GenPharm International (1989–1995), senior research fellow at Molecular Dynamics (1995–1997), senior director at Incyte Genomics (1997–2001) and as chief scientific officer and founder at Arcos BioScience (1997-2003).At GenPharm, Loring worked on gene editing in mouse embryonic stem cells, and creation of mouse models for human disease. Loring founded Arcos Bioscience in part to work on human embryonic stem cells and derived nine of the human embryonic stem cell preparations that were approved for federal funding by President George W. Bush in 2001. Arcos merged with Cythera, now renamed Viacyte and in 2004 Loring moved to academia, serving as the founding co-director of the Stem Cell Center at the Burnham Institute (now called the Sanford Burnham Prebys Medical Discovery Institute).

Loring is also an advocate for patient education and against stem cell tourism and has frequently spoken

out on these subjects including commentaries in ethics journals with bioethicist Mary Devereaux. She has also commented on the ethics of stem cell research in articles with ethicist Jonathan Moreno and pro-life advocate Christine Scheller. She often guest blogs on the stem cell blog, The Niche, describing her experiences, such as attending an FDA public meeting on Huntington's disease and Parkinson's disease. For her outspoken support of patients and advocacy of stem cell research she was awarded the Stem Cell Person of the Year award in 2015 and received a 2015 Stem Cell in Action Advocacy Award from the Genetics Policy Institute.

“Women in science are the greatest risk takers because, unlike men, they can never rest on their previous accomplishments”

More about Jeanne’s research

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Martina Schussler-LenzChair of the Committee for Advanced Therapies at the European Medicines Agency (EMA)This January, Martina has been re-elected as chair, for EMA’s Committee for Advanced Therapies (CAT) for a second three-year mandate.

Alongside this role, Dr Schüssler-Lenz is the Deputy Head of the Advanced Therapy Medicinal Products Section at the Paul-Ehrlich Institute (PEI) in Langen, Germany. Before becoming chair, she served as vice-chair of the CAT from March 2014 to February 2017.

Overall, Martina has 30 years of professional experience. She completed her clinical training in Mainz and Berlin (Board Certified Internist), and worked as a postdoctoral fellow in tumor biology and immunology in New York (Memorial Sloan Kettering Cancer Center) and Barcelona (Instituto Municipal de investigacion Medica).

The CAT is responsible for assessing the quality, safety and efficacy of advanced therapy medicinal products (ATMPs) and following scientific developments in the field. ATMPs are medicines for human use that are based on genes or cells. They offer ground-breaking new opportunities for the treatment of disease and injury. The Committee prepares a draft opinion on each ATMP application submitted to EMA, before the Agency’s committee for human medicines (CHMP) adopts a final opinion on the marketing authorisation of the medicine concerned.

Upon her re-election, Dr Schüssler-Lenz stated “Over the next three years, I will continue the work we have already undertaken to strengthen the CAT’s interaction with EMA's committee for human medicines, as well as other committees and working parties and to make the Committee’s expertise more visible. Strong interaction with all relevant players in the EU medicines regulatory network and with stakeholders will ultimately facilitate access to advanced therapies that can transform patients’ lives."

Christi ShawCEO, Kite, a Gilead Company

Christi Shaw serves as Chief Executive Officer of Kite, Gilead's cell therapy company. Based in Santa Monica, California, Kite is pursuing the ambitious goal of a cure for cancer with industry-leading pipeline and manufacturing capabilities. In her role, Christi is responsible for all cell therapy operations around the world.

Christi holds a bachelor's degree in business administration from Iowa State University and an MBA from the University of Wisconsin. Before joining Gilead in 2019, Christi was President at Lilly Bio-Medicines; her leadership has spanned a broad range of therapeutic areas, including oncology.

In 2016, Shaw founded the More Moments More Memories Foundation with her sister Shelley. The foundation assists patients with cancer and their families and was born of a personal experience with cancer, having lost their mother in 1996, their sister Sherry was diagnosed with multiple myeloma. Sherry was running out of options and has had to travel many states away to gain access to new solutions to try and save her life. Christi and Shelley put their careers on hold to support their sister through a clinical trial, in addition, both Christi and Shelley are pharmaceutical execs who had the knowledge to advocate for their sister. However, most families in similar situations do not have that ability. The Shaw sisters are passionate about their belief that money or lack of awareness should never stand in the way of creating more moments and more memories for families.

Find out more about the CAT

More about Kite More about the More Moments More Memories Foundation

Emily WhiteheadCancer Patient and Emily Whitehead Foundation

Emily’s journey since being diagnosed with standard-risk pre-b acute lymphoblastic leukaemia (ALL) in May 2010 when she was five years old has undoubtedly had a huge influence on families and regulators alike. Hers is not a straightforward story but one of relapses and bone marrow transplants and the prospect of a hospice.

However, neither Emily nor her parents gave up and were able to enrol Emily in a brand-new CAR-T trial at Children’s Hospital of Philadelphia (CHOP) in April 2012. In fact, Emily was the first child ever to receive a CAR-T treatment and has thankfully been in remission since May 2012. Since then, Emily has been involved in the regulatory authorisation of Kymriah with the FDA.

Emily’s father, Tom and Bruce Levine, Dr Bruce Levine, Barbara and Edward Netter Professor in Cancer Gene Therapy at the University of Pennsylvania discuss more at Phacilitate Leaders World 2019.

Emily’s treatment gained worldwide attention, and her story has appeared in The New York Times, Forbes, PARENTS Magazine, the Stand-Up-to-Cancer Telethon, HBO Vice and a PBS documentary.

Tom, Kari and Emily travel internationally to share Emily’s story and to raise awareness for childhood cancer research.

Cutting edge treatments like the T-cell therapy are needed to help cure children whose cancer returns, but funding is limited. Childhood cancer receives less than 4% of the National Cancer Institute budget for research in the US. Many researchers rely on grants from non-profit organizations and private donors who are dedicated to raising funds for childhood cancer research.

The Emily Whitehead Foundation’s focus is on funding the most promising research such as the T-cell therapy that Emily received and other immunotherapies.

“We have been given so much and we hope to give back much more in return. With your help we can make a difference!”

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Learn more about the Foundation

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Dr. Samantha Yammine, PhD (she/her) is a neuroscientist, science communicator and digital media producer who shares all kinds of brain-blowing science anywhere and everywhere. She goes by Science Sam on Instagram, where she’s built a strong community of people (currently 43.4k!) who love her unique style of socially conscious science storytelling.

Samantha earned her PhD from the University of Toronto researching how stem cells build the mammalian brain before birth and maintain it throughout adulthood. In addition to her doctoral research, Samantha co-authored a crowd-funded research study exploring the effects that #ScientistsWhoSelfie on Instagram can have on public perceptions of scientists.

Samantha is passionate about empowering people to explore science by making it more familiar, accessible and inclusive. She does this in part by sharing interactive science commentary and research updates on social media through short videos, emoji-filled captions and engaging pictures. She’s created space for continued, multi-way dialogue about science that transcends geographical borders and the traditional gate-keeping of academia. A leader in digitising science on social media, Samantha founded Science Sam Media in 2019 to continue to grow the reach and impact of her science communication initiatives.

Samantha always takes an audience-first approach to share complex topics in clear and entertaining ways. She is a frequently requested keynote speaker and event host who loves engaging folks with anything science, anywhere and everywhere – she's hosted a livestream with an astronaut aboard the International Space Station, presented her research on the streets at Toronto's busiest intersection, talked ringside with Muay Thai fighters about performance anxiety and showed off model and real brains at 1am in a Yurt during an all-night art festival. She leads interactive workshops on science communication, personal branding, and social media to teach scientists and STEM professionals the skills they need to share their science creatively.

She's produced, scripted, and hosted educational videos, and regularly appears as a guest expert in the popular media including appearances on TVO Kids, Leafs Nation Network, Netflix, CBC GEM, and engaging audio with AsapSCIENCE and CBC Radio. Samantha is a member of the Editorial Committee for the World Congress of Science and Factual Producers and the Program Committee for the Royal Canadian Institute for Science. She was the Founder and a Co-Producer of the first ever #ScienceIsADrag event, which she continues to run in Toronto in partnership with RCIScience, Geith Maal-Bared and Shawn Hercules.

Learn more about Sam

Dr Samantha YamminePhD, Neuroscientist and Science Communicator

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Ran ZhengCTO, Orchard Therapeutics

Ran Zheng brings joined Orchard in March 2016 after 16 years at Amgen and 25 years of experience in technical operations. Zheng oversees multiple related functions for Orchard, including process development, manufacturing, quality, facilities and engineering, supply chain and CMC program management.

Prior to joining Orchard, Ran spent 16 years with Amgen in roles of increasing responsibility in operations. Most recently, she served as vice president of development supply chain, overseeing development labs and pilot plants, end-to-end supply chain and manufacturing operations across multiple geographic locations to enable Amgen’s global clinical development, product launch, commercial supply and technology innovation.

Ran holds a B.S. from Beijing Forestry University and an M.S. in microbial engineering and chemical engineering from the University of Minnesota.

Orchard Therapeutics is an important player in the advanced therapies industry and a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.

Orchard’s portfolio of autologous, ex vivo, hematopoietic stem cell gene therapies includes Strimvelis, the first such treatment approved by the European Medicines Agency for severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD) and transfusion-dependent beta-thalassemia (TDBT), as well as an extensive preclinical pipeline.

“We are committed to serving the rare disease

community by pioneering ground-breaking

innovation and driving the industrialisation of

gene therapies. Our vision is to make the

incurable, curable.”

Find out more about Orchard’s pipeline

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Claudia ZylberbergCEO, Akron BiotechClaudia is a biotech leader with more than 30 years in the field. She founded Akron Biotechnology in 2006, a manufacturer of novel cGMP-grade products for the gene and cell therapy industries. She also co-founded AssureImmune, an adult stem cell bank, for personalised medicine. She holds numerous patents and peer-reviewed publication records. She has developed products to support the industry growth, like cryomedias and others.

Zylberberg has several patent-pending technologies and grants through NIH and the Department of Defense. In early years, Dr Zylberberg worked at Nabi Biopharmaceuticals, specializing in human plasma derived products. She is a board member of ISCT, the Alliance for Regenerative Medicine ARM, and the National Academy of Sciences (Regenerative Medicine Forum). Other advisory positions include ISO US TAG, BioFlorida, ISSCR, and Cord Blood Association.

In the early days, Akron was primarily a distributor of raw materials and components for the biotechnology industry. Over time, it has expanded its product portfolio to fill key industry gaps and expanded its core capabilities to offer greater value to its growing set of clients.

From its humble beginnings, Akron has evolved into a company with a strategic focus on developing and commercializing enabling tools, technologies and services for the regenerative medicine industry. As a global supplier, Akron manufactures and distributes components and raw materials for cell therapy discovery, development and commercialization, meeting industry needs worldwide. The company’s unique business model emphasises flexibility, quality, and unparalleled service—from research and development through clinical trial, market authorization and commercial product.

Claudia has been an important mentor for the Women in Advanced Therapies mentorship programme and also launched an update of her children’s book in January 2020 at the Future of Medicine Day hosted by Frost Science Museum and Phacilitate. The book, You’re Full of Genes, takes children on a journey as they participate in games and activities that help them learn about how cells, enzymes and the genetic code work to form their identities.

"This project began as a labour of love, but as the cell and gene therapy industry propels forward at an unprecedented rate, I've found it is necessary to help educate the public, beginning with our youth," Zylberberg said.

Sales proceeds will support four foundations working on scientific advancements and education in the field: the CCRM Foundation, the ARM Foundation for Cell & Gene Medicine, the American Society of Gene & Cell Therapy (ASGCT) and The Duke Pediatric Bone Marrow Family Support Program.

“As key contributors to this great revolution in

medicine, women are innovating at all levels

to bring new treatments to so many in need. We

are part of the big engine and recognizing

their (women and others’) valuable

contributions is the way to stimulate many more

to join the cause.” Here's more about You’re Full of Genes

WWomen Offering Mentoring, Education and Networking (W.O.M.E.N) is a mentorship and networking programme hosted by Phacilitate for the advanced therapies community. The goal of W.O.M.E.N in advanced therapies is to build the foundations for a community to help recruit, retain, develop and promote women, creating leaders of tomorrow and encouraging new women to join the sector. We meet face to face at Phacilitate meetings across the year, which act as both an education and networking opportunity. A panel of women in C-level positions in the industry will discuss actions others can take to support themselves in reaching board level. Mentors and mentees are paired and continue their relationship virtually or in person throughout the year. If you’d like more information or to join, please do get in touch!