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An Interview with Ray Tabibiazar, M.D., CEO & Chairman of SalioGen Therapeutics

As part of WuXi AppTec’s ongoing efforts to collaboratively foster new thinking and actionable approaches in advancing breakthroughs for patients, we have launched a new interview series in 2022 – “Delivering on the Promise of New Modalities” – so leading voices of R&D can share how their approaches are addressing the barriers standing in the way of breakthroughs. Up next in our interview series, Ray Tabibiazar, M.D., a seasoned executive in the healthcare and biopharma industries, imparts his perspective as CEO & Chairman of SalioGen Therapeutics. SalioGen is advancing a new category of genetic medicine through its unique Gene Coding approach, a genome engineering technology that leverages proprietary mammal-derived enzymes. Earlier this year, SalioGen closed a $115 million Series B financing to advance the company’s goals of transforming the treatment paradigm for patients with inherited diseases and beyond. Thank you for taking the time to sit down with us, Ray. What major bottlenecks or barriers is SalioGen aiming to address? Ray: At SalioGen, we aim to address inherited disorders by addressing some of the limitations of currently available approaches. Current therapeutic approaches include gene editing and viral vector-based gene therapies. These modalities, however, often have limited clinical applicability. Many of the inherited disorders are caused by many different mutations and oftentimes involve a large gene (>5 kb sequence). Gene editing is designed to fix only a few point mutations at a time, and viral gene therapies can only accommodate gene sequences of a few thousand base pairs at most. Additionally, viral gene therapies carry the risk of causing potentially severe immune responses in patients and gene editing carries the risk of causing damage to the DNA that it changes. Altogether, the field of genetic medicine is limited in the number of inherited disorders it can address effectively and safely. How is SalioGen’s approach differentiated and what might be advantages of the approach? Ray: SalioGen is developing a new category of genetic medicine called Gene Coding, which is designed to turn on, turn off or modify the function of any gene in the genome. The cornerstone of Gene Coding is SalioGen’s portfolio of mammal-derived enzymes collectively called Saliogase, which can take a DNA sequence of any length and integrates it into the genome at a precise, pre-defined location. To our knowledge, SalioGen is the only company developing a mammalian genome engineering enzyme. Gene Coding machinery is delivered using a cell specific lipid nanoparticle; therefore, we don’t anticipate any risk of causing the virus-related immune responses that some gene therapies can cause. Furthermore, Saliogase works without causing double-stranded breaks when it inserts new DNA to the genome. It therefore doesn’t trigger any of the error-prone DNA repair pathways that may cause DNA damage and other unwanted downstream effects. Saliogase can also insert DNA sequences of any length, unlocking the potential to address most inherited disorders regardless of the size of the gene or genes involved in the disease. All these features are designed to make Gene Coding applicable to a broad range of inherited disorders. The physical components of the technology are also easily reproducible and scalable, ensuring they can be manufactured to reach large patient populations with prevalent inherited disorders. What challenges are you facing currently and how are you addressing these?  Ray: With the recent explosion of new biotech companies, there has been a shortage across the sector including limited capacity at partner CDMOs and a shortage of non-human primates for GLP toxicology studies. We are working to develop in-house CMC capabilities to maintain control over our manufacturing process. Pipelines of genetic medicines are on the rapid rise. What trends do you see coming in the next few years?  Ray: There has been a clear trend toward the accelerated development timeline for the new fields in genetic medicine. RNA-based therapeutics were largely developed over the course of roughly the past twenty years, with siRNA therapeutics and recently with mRNA therapeutics. On the other hand, the more recent newcomer of gene editing has taken hold in about half the time, in the past 11 years since the first papers on the CRISPR-Cas9 system as we know it were first published. We anticipate that by 2030, we’ll see increased availability of different modalities of genetic medicine for inherited disorders. In addition to currently available products like viral vector-based gene therapies, RNAi and antisense oligonucleotides, perhaps modalities like Gene Coding and other non-viral genome modification platforms, mRNA-based gene therapies and epigenetic therapies will be further advanced and available for patients. Definitely a great outlook and great opportunities for patients.  Any closing thoughts you like to share with our audience? Ray: Looking forward, we expect to see a shift in drug approvals away from single-purpose compounds and toward therapies based on platform technologies. Successful platform technologies will each be capable of yielding multiple successful therapies, each of which can be developed in parallel. Platform technology-based development is becoming more common and is significantly more streamlined than the historically more common trial-and-error, one-molecule-at-a-time path of drug development, and that may push us toward a greater volume of drug approvals by 2030. Thank you Ray, it’s been an insightful discussion into the new breakthroughs SalioGen is bringing to the field of genetic medicine. We wish you luck in your future endeavors. Ray: Thanks for having me.   Ray Tabibiazar CEO & Chairman, SalioGen Therapeutics Ray is a seasoned executive with leadership experience in the healthcare and biopharma industries, including venture capital, pharmaceuticals and diagnostics. As a clinician-entrepreneur and as managing director of 526 Ventures, he has focused on creating new ventures in the form of new companies or spinouts to translate innovative science into commercially viable products. Previously, Ray led Aravive Biologics as President and CEO, served as Senior Vice President, Corporate Development and Business Strategy at Twist Bioscience, a venture partner at Bay City Capital, and other senior executive roles. Prior to moving to industry, Ray was a practicing cardiologist and an adjunct faculty member at Stanford University, having trained as a physician scientist at Harvard Medical School and a cardiologist at Stanford Medical Center. Ray graduated with his medical degree from Harvard Medical School.

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Delivering on the Promise of New Modalities: WuXi AppTec Interview Series

As part of WuXi AppTec’s ongoing efforts to collaboratively foster new thinking and actionable approaches in advancing breakthroughs for patients, we have launched a new interview series in 2022 – “Delivering on the Promise of New Modalities” – so leading voices of R&D can share how their approaches are addressing the barriers standing in the way of breakthroughs. Ray Tabibiazar CEO & Chairman, SalioGen Therapeutics David Main President & CEO, Notch Therapeutics Susan Dillon CEO, Aro Biotherapeutics

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Delivering on the Promise of New Modalities: An Interview with David Main, President & CEO, Notch Therapeutics

As part of WuXi AppTec’s ongoing efforts to collaboratively foster new thinking and actionable approaches in advancing breakthroughs for patients, we have launched a new interview series in 2022 – “Delivering on the Promise of New Modalities” – so leading voices of R&D can share how their approaches are addressing the barriers standing in the way of breakthroughs. Our next installment of this interview series features David Main, President & CEO of Notch Therapeutics based in Vancouver, Canada. Notch has unlocked the ability for large-quantity production of T cells and other cells from any source of stem cells to bring best-in-class cell therapies for cancer and other immune disorders. In February of 2021, Notch Therapeutics closed $85 million Series A financing to develop their pipeline of renewable stem-cell derived cancer immunotherapies. Thank you for joining us today David. Looks like Notch’s pipeline will cover a number of therapeutic areas such as cancer and autoimmune diseases. What are your considerations behind those?  David: Notch is primarily interested in treating cancer. Current cancer therapy challenges, which have remained unchanged for decades, include trying to improve treatment specificity and reduce toxicity. But now, significant cancer research and therapy development is focused on the immune system’s response, with cell therapies offering transformational benefits over existing standards of care. Where chemotherapies act on the cancerous tumours themselves, immunotherapies focus on harnessing immune cells to identify and destroy cancer cells directly. Cell therapy does bring its own set of challenges. Access to a uniform and unlimited supply of cells is a critical limiting factor. Further, administering cells that are not produced by a particular patient, could introduce new toxicities such as potential rejection or triggering other immune issues. What is your approach helping to address these challenges? How is it different from existing approaches? David: The principles of cell therapy treatment for cancer are well established. We can take immune cells from a patient, do some engineering, and then give these cells back to the patient, to attack the cancer. However, this approach is limited to very few patients. Not all patients are well enough to donate their own immune cells. Due to a long manufacturing process, by the time we are ready to give the patient back their cells, their disease could have progressed. Even the variability in the manufacturing process means the cells may not work as expected. Thus, Notch’s approach is to create immune cells from renewable cell sources. Our proprietary technology platform enables the development of uniform, T cells from any source of pluripotent stem cells. These cells are now not dependant on an individual patient and can be “sitting on the shelf”, ready to treat the patient. Any potential risks or challenges associated with this approach? David: The benefits of cell therapy have been demonstrated scientifically but our approach is in progress and not yet proven.  We must move forward to demonstrate in clinical trials the cells we produce work to kill cancer and have an acceptable safety profile. To advance our technology, we have already attracted significant interest from companies and investors who view Notch’s technology platform as a means to maximize the benefit for future generations of cell therapies. In 2019, we partnered with Allogene Therapeutics to apply Notch’s T cell production platform to develop CAR-targeted, iPSC-derived or natural killer (NK) therapies for hematologic cancer indications. And in 2020, we closed an oversubscribed Series A financing. This year, we are focused on delivering a clinically representative process—including equipment, procedures, materials, and cell lines—to produce batches of T cells for in vivo testing. This is a critical step in Notch’s move towards IND (Investigational New Drug) studies and ultimately clinical trials. Look forward to your development David. Entering clinic will be an exciting milestone. Looking at the cell and gene therapy field at large, what do you see as a critical challenge to overcome?      David: We will need to work on reducing costs. Applying new technology will help. Also because costs arise not only from how we do research, but from the amount of research required for a drug to be approved, reducing costs requires not only excellent technology, but a strong commitment from and collaboration with Regulators. We are supportive of a strong regulatory progress. We all want to know the drugs going into our body, or that of a loved one, are safe and effective. However, with the current regulatory process, we typically see an annual increase in the body of knowledge and the addition of more regulations. It takes a strong-willed person to say it’s time to streamline the process by reviewing everything at once and then reforming the approvals process.  Dr. Richard Pazdur, Head of Oncology Center of Excellence at the FDA, is one such person. He demonstrated the commitment to revisiting potential cancer drug development and approval process and looked for ways to expedite paths to approval. What’s your outlook for the future of cell and gene therapies? Say 2030? David: I expect cell therapy and gene therapy will be two prominent treatment modalities. Cell and gene therapies are allowing us to harness a natural process to treat disease and to treat diseases at the gene level.  This means we can think cures, and not just treating symptoms. It means eradicating chronic diseases. So, in 2030, I think we will have several cell therapies and a number of gene therapies approved, and these will be the mainstay approach to treating disease. And I also believe that 100 new drugs are achievable by 2030. There is a proliferation of innovation within the industry and the progressive way we tackle treatments is accelerating every year. Thank you David for sharing Notch’s approaches in help advancing the potential of cell and gene therapies. Best of luck in your endeavor. David: Thank you.     David Main President & CEO of Notch Therapeutics David Main is President and Chief Executive Officer of Notch Therapeutics. Previously, as Chairman and CEO of Aquinox Pharmaceuticals, a company he co-founded in 2006, Mr. Main oversaw the advancement of the company’s lead product from target validation through Phase 3 clinical trials. He also led the transition of Aquinox from a private company to a NASDAQ-listed public company with approximately $300 million raised in equity capital and then completed the successful merger of Aquinox with Neoleukin Therapeutics. Prior to his leadership of Aquinox, Mr. Main served as President and CEO of INEX Pharmaceuticals and as a Vice President of QLT. He formerly served as the Chair of LifeSciences BC (formerly BC Biotech), BIOTECanada, and Accel-Rx as well as a Director of Mr. Main began his career as a licensed pharmacist at the Royal Columbian Hospital in New Westminster, B.C. He holds a BSc (Pharmacy) and an MBA from the University of British Columbia (UBC).

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Delivering on the Promise of New Modalities: An Interview with Sue Dillon, CEO of Aro Biotherapeutics

New modalities offer unprecedented therapeutic potential for patients, yet they also pose unique challenges. As part of WuXi AppTec’s ongoing efforts to collaboratively foster new thinking and actionable approaches in advancing breakthroughs for patients, we have launched a new interview series in 2022 – “Delivering on the Promise of New Modalities” – so leading voices of R&D can share how their approaches are addressing the barriers standing in the way of breakthroughs. Our first episode of this interview series features Sue Dillon, an industry veteran and co-founder & CEO of Aro Biotherapeutics based in Philadelphia. Aro’s proprietary Centyrin technology platform enables precise receptor-mediated delivery of RNA drugs to address intracellular gene targets. In Jan 2021, Aro announced $88 million Series A financing to advance development of Centyrin-Targeted genetic medicines to clinical development. Congratulations Sue on Aro’s series A. What gaps is Aro trying to address in the field? Sue: We are focused on developing tissue – targeted genetic medicines for patients with rare genetic muscle diseases and immune diseases with high unmet medical need. Our approach achieves selective and efficient targeting of RNA medicines, such as small interfering RNA and antisense oligonucleotides, to specific tissues thus enabling first-in-class medicines that precisely modify disease related genes. As a drug class, oligonucleotides have demonstrated dramatic clinical proof of concept for modulating disease related genes in the liver, or when locally delivered to the CNS or to the eye directly. Although this approach offers the possibility of addressing previously “undruggable” disease targets, the gap facing the field has been lack of efficient targeting and delivery of this exciting class of drugs to the many extra – hepatic tissues that cannot be addressed with local administration. Our initial work is focused on targeting siRNA drugs to skeletal muscle, heart, immune cells, and tumors. Tell us a bit more about Aro’s Centryn platform and how is it different from others out there? Sue: We are pioneering a new class of drugs called Centyrin – siRNA conjugates. Centyrins, Aro’s proprietary antigen-binding platform, are small, simple, and highly stable proteins.  Centyrins have been shown to bind target antigens with high specificity and high affinity, and Aro has identified Centyrins that bind to cell surface receptors which internalize post binding, thus carrying the Centyrin inside the cell. By chemically linking Centyrins with siRNAs or other oligonucleotides, we can target these gene modifying RNAs to particular tissues in vivo.  Centyrins have a number of unique properties – small size, low immunogenicity and relative simplicity of chemical conjugation and manufacturing among others – that makes them uniquely suited for targeted delivery of oligonucleotides. Using Centyrin oligonucleotide conjugates, we have shown robust gene modulation in target tissues with little to no effect in non-target tissues and have recently achieved disease relevant pharmacology in animal models. Any challenges or risks that you foresee with Aro’s novel approaches? Sue: We are pioneering a new class of drugs that is comprised of a protein component produced by recombinant technology in bacteria (Centyrin) covalently linked to a chemically synthesized oligonucleotide. There are no drugs of this type that have achieved FDA approval and as such, we are engaging with the FDA early to de-risk the preclinical and manufacturing IND enabling studies that will support our First-in-Human trial. Furthermore, given the nature of our molecules, there are complexities associated with species cross-reactivity for the oligonucleotide and Centyrin that complicates development.  We rely upon certain precedents (ADCs, oligonucleotides) that are not perfect analogues but can still provide valuable information. Additionally, we have recruited an excellent team of experienced scientists and developers and identified external consultants to create development plans for our first product candidate. Importantly, our learnings from our first programs will be deployed against future programs, which will reduce the risks associated with development of new pipeline assets. Look forward to hearing more on Aro’s exciting developments as you advance programs to clinic. It is exciting to see now patients may have more and better treatment options as we gain more insights into new modalities and understand better how to overcome the challenges. How do you see the field will continue to evolve? Sue: While small molecules and antibodies dominate the approval landscape of 2021, there are major shifts in R&D investment given new scientific breakthroughs. In recent years, the first siRNA medicine, cell therapy, and gene therapy have all achieved FDA approval. Just as it took several years for antibody drugs to become a mainstay in the pharmaceutical industry, we are at the very early stages of these new modalities. Across many of these new modalities, targeted delivery of genetic medicines to diseased tissues, while sparing normal tissues, is recognized as a barrier to achieving maximum efficacy and ideal safety profiles. We are very excited to potentially play a meaningful role in solving this issue, as Centyrins have the potential to achieve specific targeting to various tissues for a variety of drug payloads including oligonucleotides, and also more complex drug moieties including mRNA, gene editing technologies and cell therapies. As new modalities continue to broaden and brighten new horizons of therapeutic intervention, do you see our industry collectively may be able to achieve 100+ new drug approvals at 50% of today’s cost? Sue: I am optimistic about the industry’s ability to deliver 100+ approvals by 2030. The application of artificial intelligence and machine learning toward analyzing complex data sets and making associations across data will advance understanding about the root causes of disease and enable new classifications of disease and identification of new drug targets. At the same time, advances in precision medicine will improve success rates by identifying patients who are most likely to respond to a given therapeutic based on linking therapeutic MOA with the patient’s genetic profile. Additionally, accelerated clinical trials and the potential for singular (n=1) trials of highly customized therapeutics should shorten development timelines and reduce costs. Manufacturing costs and capacity of GMP approved sites for genetic and cell therapies are currently a significant challenge and must be addressed to make these medicines more cost effective and widely available. Thank you Sue for your sharing Aro’s approaches in advancing breakthroughs for patients. Sue: My pleasure.     Sue Dillon Co-Founder, President & CEO, Aro Biotherapeutics Sue Dillon, PhD, brings 30 years of experience in executive leadership roles at pharmaceutical and biotech companies to Aro Biotherapeutics. During her more than 16-year career at Johnson & Johnson, Sue led global Immunology R&D, and achieved numerous regulatory approvals for innovative antibody products for autoimmune diseases including REMICADE®, SIMPONI®, STELARA® and TREMFYA®. She also built a robust Immunology development portfolio through internal discovery and external licensing, and championed research into the emerging areas of the microbiome and immune repertoire profiling. Multi-disciplinary teams under Sue’s leadership were twice recognized with the Prix Galien Award for PROMACTA® and STELARA®, each first-in-class medicines. Sue received her PhD in Immunology from Thomas Jefferson University in Philadelphia and completed a postdoctoral fellowship in Immunology at Duke University. She was named by FierceBiotech as one of the “Top Women in Biotech” in 2013.

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Cydan Sets the Pace for Rare Disease R&D

As part of our ongoing interest in rare disease R&D, we interviewed Niels Svenstrup, the Senior Vice President of Development at Cydan, a virtual rare disease accelerator focused on the development of treatments for rare genetic disorders. Tune in to hear discussion on Cydan’s unique disease –agnostic model and their prosperous child orphan drug companies, Imara and Tiburio. Gain insight into the future of rare disease R&D, Cydan’s essential collaborations, and more. We invite you to watch now.

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