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2020/09/01

Big Ideas Yield Transformative Results for SMA Patients

Celebrate SMA Awareness with us!  Stuart Peltz (Founder and CEO, PTC Therapeutics) shares his perspectives on the dynamics of R&D in the context of the journey that led to the accelerated approval this August of  Evrysdi™ (risdiplam), the first oral treatment for SMA. What can we learn from the collaborative partnership between Roche, PTC Therapeutics, and the SMA Foundation?

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2020/08/17

One-Stop Cell and Gene Therapy Solutions for Global Innovators: A Conversation with David Chang, CEO of WuXi ATU

In the past few years, the field of cell and gene therapy has witnessed major breakthroughs. It has become one of the hottest fields in the biotech industry, with the hope that it will eventually revolutionize the way we treat diseases and, ultimately, become a mainstream treatment option. Cell therapy refers to the transfer of healthy or modified human cells to heal or replace damaged tissue or cells, while gene therapy refers to the modifying of genes or replacing of defect genes with healthy ones to treat, cure or prevent a disease or medical condition. Though distinct, the two are closely associated with each other and often considered one field. Though still in their infancy, cell and gene therapies are developing at an explosive rate. There are currently hundreds of related programs in the clinic. This means that, over the next several years, many cell and gene therapies are likely to enter the market. There are clearly good prospects for the future of cell and gene therapy, but the road ahead contains obstacles as well. These include a prolonged and dynamic R&D cycle, safety issues, and manufacturing cost and limitations. Only by addressing and overcoming these challenges can we unleash the full potential of cell and gene therapy to help patients in need. Dr. David Chang, CEO of WuXi ATU, WuXi AppTec’s cell and gene therapy Contract Development and Manufacturing Organization (CDMO), recently sat down with the WuXi AppTec Content Team to share his observations on cell and gene therapy’s current development and insights into the field’s path forward. Prior to joining WuXi AppTec, Dr. Chang held senior positions at several leading companies, such as Celgene (acquired by Bristol-Myers Squibb in 2019) and Genentech. He also has extensive experience in the biotech and cell and gene therapy industries. What’s the current state of cell and gene therapy globally? Dr. Chang: Although the earliest research occurred decades ago, it is only in recent years that we have been able to bring effective therapies to the market. The biggest and by far most famous breakthrough in cell therapy is of course CAR-T (Chimeric Antigen Receptor T-Cell Immunotherapy). In 2017, Novartis’ Kymriah, developed for the treatment of certain types of ALL (acute lymphoblastic leukemia) patients, and Yescarta, developed by Gilead’s Kite Pharma for large B-cell lymphoma, were successively approved by FDA. These approvals demonstrated the potential of such therapies for cancer treatment. Gene therapy has also been gaining momentum for a long time, and over the past five years several drugs have been approved by regulators in Europe and the U.S. to treat severe inherent genetic diseases caused by specific genetic mutations, such as spinal muscular atrophy (SMA) and β-Mediterranean anemia. These recent developments are a great encouragement to the industry— to drug developers, to investors, and of course they are tremendously encouraging to patients as well. Keep in mind that, for any new therapy, a breakthrough from zero to one is the hardest. Given these successes, it’s no surprise that enthusiasm for cell and gene therapy has grown significantly lately, with many innovative companies being involved. The current pace of development is explosive. Hundreds of drugs are in the pipelines around the world, and many of them are already in the late clinical stage. To what extent will these types of therapies change the future of medicine?  Dr. Chang:  Cell and gene therapies have enabled people to effectively treat and even cure hitherto very difficult-to-treat cancers and rare genetic diseases, thus rewriting the life stories of many patients. I believe that they will become one of the mainstream treatments in the future. Cell therapy has the potential to be one of the most important treatments in our fight against cancer. For example, current CAR-T therapies have proven effective in treating relapsed/refractory ALL. These specific CAR-T therapies can, in theory, be applied to treat a lot of other cancers, and there’s a lot of relevant research going on right now. As for gene therapy, it has been applied to treat a wide range of diseases, such as cardiovascular disease, eye diseases, nervous system diseases, metabolic diseases and solid tumors, in addition to long-standing efforts in genetic blood diseases such as sickle cell disease and hemophilia. For cell and gene therapy to really take off, what challenges and problems need to be tackled? Dr. Chang: The potential is huge, but the challenges should not be underestimated. To keep our forward momentum there are still a series of bottlenecks to overcome. The industry needs to continuously improve R&D pipeline and lifecycle management, optimize the relevant manufacturing and logistics processes, and reduce costs. I personally believe the major bottleneck is not in R&D, but rather in manufacturing as well as in test and release. One of the major issues to consider in cell and gene therapy is how to scale out or up the manufacturing while reducing the cost at the same time. So, what exactly are the manufacturing challenges for cell and gene therapy? Dr. Chang: In gene therapy, there is a big gap in the production of non-replicable lentiviral vectors and AAV vectors. Depending on the genes of interest, production sometimes requires large-scale fermentations to meet a single patient’s treatment needs, making it commercially challenging or even infeasible. Therefore, yields and efficiency of virus vectors manufacturing are a major challenge for the whole industry. Cell therapy also faces the limitations in commercial manufacturing. Autogenous cell therapy is an individualized treatment that activate, modify and expand the patient’s own immune cells, so a single batch is made for one patient only. This means that the manufacturing scale will never be as large as that of traditional small-molecule or biologics products, and thus is much more costly. At the same time, cell therapy, especially allogeneic cell therapy, which utilizes donor cells, faces the challenge of graft-vs-host disease. It’s necessary to ensure that the modified cells transmitted back to the patient do not generate an immune response (similar to what happens with organ transplant rejection). Finally, cell therapy faces strict requirements for timely and reliable transport of labile raw material and cell products, as well as complexity in product testing and release. As an enabling platform for gene and cell therapy, what solutions and advantages does WuXi ATU have to address these challenges? Dr. Chang:  Through the establishment of an advanced therapies CDMO platform, we offer real one-stop service to our partners to help with their research, manufacturing, and test and release. Most existing CAR-T manufacturing processes are not closed and thus require substantial open, manual manipulation under a biosafety hood, which may lead to errors, high costs, and risk of contamination. To specifically address these problems, WuXi ATU has recently introduced an advanced closed automated CAR-T platform in its base in Philadelphia to meet the manufacturing needs of our partners. Relying on our two bases respectively in the US and China, we can not only meet the diverse manufacturing needs of customers from different regions, but also ensure the timeliness of transportation that CAR-T requires. In gene therapy manufacturing, currently, we have established high-yield, highly scalable AAV and lentiviral platforms which can supply cost-effective and commercially feasible viral vectors in a timely manner. WuXi ATU also has the capability to provide international standard oncolytic virus product development and manufacturing services to customers worldwide. In November, we signed a strategic partnership agreement with Gene Medicine, a Korean gene therapy biotech, to provide services covering oncolytic virus product development, manufacturing and clinical trials for its key programs. In addition to the mainstream approaches of introducing genes into cells, we are also establishing more efficient and promising non-viral technologies such as electroporation, which utilizes electric fields to allow genes to pass through the cell membrane. Most importantly, WuXi ATU also provides the integrated analytical method development and testing services to our manufacturing clients.  With the complexity of testing and releasing advanced therapies products in a timely and reliable manner, this integrated testing and releasing service will greatly support our clients to supply their products to meet either clinical or commercial needs. Through improving efficiency and reducing costs, we’ll support our clients to enhance the accessibility of cell and gene therapy – and help more of our partners’ innovative therapies to reach patients as broadly as possible as soon as possible. The rapid development of cell and gene therapy has attracted a lot of outside attention. People have high expectations. From the perspective of someone on the inside, what do you see when you look at the near future?  Dr. Chang: There is no denying that cell and gene therapy still face challenges. Actually, every new therapy faces or faced a similar situation. Even regulators need to learn alongside industry. This process is both normal and necessary. I believe that in another five to ten years, with the accumulation of relevant knowledge and experience, the whole industry will become more mature, efficient and standardized. I firmly believe that the future for cell and gene therapy is very bright, and WuXi ATU stands ready to join hands with global innovators to move it to the next stage.

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2020/07/08

TG Therapeutics’ Early Oncology Research Leading to New MS Therapeutic

At TG Therapeutics in New York City the company didn’t start out looking for new therapies for Multiple Sclerosis (MS). TG began as an oncology focused company but as the data came in from their research the company and President and CEO Michael Weiss believed their assets could be applied in additional disease spaces and are developing a new therapy for MS. More than 2.5 million people suffer from MS worldwide. It is a long-lasting disease that can affect your brain, spinal cord, and the optic nerves in your eyes. It can cause problems with vision balance, muscle control, and other basic body functions. MS is a rare autoimmune disease. Normally, antibodies produced by the immune system help protect the body against viruses, bacteria, and other foreign substances. In people who have MS, the immune system attacks the body. There is no known cure for MS, but Mr. Weiss believes that real progress has been made in developing new treatment options for MS sufferers, and that TG may be offering its own new therapy for MS. WuXi AppTec Communications as part of its special series on MS spoke to Mr. Weiss about the advanced therapeutic approaches TG is developing to treat this debilitating disease. Mr. Weiss founded and has served as the Company’s Executive Chairman, President and CEO since December 2011. Prior to TG, Mr. Weiss founded and was the CEO of ACCESS Oncology (1999) which merged with Keryx Biopharmaceuticals (2004) for which he served as Chairman and CEO until mid-2009. Previously, Mr. Weiss spent several years as a lawyer and then investor in biotech venture capital. Mr. Weiss continues to have a passion for helping early stage biotech companies and serves as Vice Chairman of Fortress Biotech and Chairman of Checkpoint Therapeutics and Mustang Bio.  WuXi AppTec: Many biotech companies are involved in cancer research; how did you chose to target MS as well? Michael Weiss: At TG our number one goal and priority is to create the best possible treatment solutions for patients with B-cell diseases. While we began with a focus on oncology, we’ve known that many autoimmune diseases also result from aberrant B-cell activity, so from very early on we had a vision to also bring our drug candidates into autoimmune disease. When we saw the early data evaluating CD20s in MS we knew this was a great place to start helping patients. WuXi AppTec: What makes MS so difficult to develop effective therapies for? Michael Weiss: One of the biggest challenges in treating MS is that the cause remains unknown. It’s still unclear exactly why this autoimmune CNS disease develops in some people and not in others Most available data points to some combination of genetic and environmental factors sharing the responsibility. In addition, signs and symptoms of MS patients vary widely. That said, while this lack of critical information has so far made targeting the root cause of MS extremely difficult, we believe CD20 treatment is extremely beneficial to patients and is a significant step forward for the treatment landscape. WuXi AppTec: Have you encountered any patient recruitment challenges in your MS program that were different from your experience with other trials you’ve conducted in the past? Michael Weiss: The ULTIMATE trials enrolled incredibly well with a total of approximately 1100 patients across both trials and target enrollment achieved approximately 9 months ahead of schedule. WuXi AppTec: How much progress has been made in MS drug research and development over the last 20 years? Michael Weiss: Progress has been significant to say the least. Think about it: 30 years ago, there were no approved MS drugs in the United States at all. Ten years ago, there were still no oral treatments available. The use of anti-CD20 monoclonal antibodies in treating MS was only first approved in 2017. We’re witnessing several MS treatments in development, and while a true cure remains elusive, I have hope this research will serve to further advance novel therapies and provide new treatment options for the millions of MS patients worldwide. WuXi AppTec: We have been talking about MS research in general. I would like to switch to your specific drug program. What phase is it in? What is its MOA? And what results have you seen so far? And will it treat all stages of the disease? Michael Weiss: Our ULTIMATE I and ULTIMATE II Phase 3 trials in relapsing forms of multiple sclerosis (RMS) are two independent, global, randomized, multi-center, double-blinded, double-dummy, active controlled trials comparing ublituximab to oral teriflunomide. The primary endpoint for each study is annualized relapse rate following 96 weeks of treatment and the trials are designed to support submission for full approval of ublituximab in relapsing forms of MS. As I had touched on earlier, ublituximab is a novel glycoengineered monoclonal antibody that targets a specific and unique epitope on the CD20 antigen found on mature B-lymphocytes. Its mechanism of action is similar to the approved anti-CD20 monoclonal antibodies, but the innovation of ublituximab is that it has been bioengineered to remove certain naturally occurring sugar molecules from the anti-CD20 antibody in a process called glycoengineering. By removing the sugar molecules we enhance the potency of ublituximab, with data showing 50-100x greater activity than non-bioengineered anti-CD20 antibodies. The ULTIMATE I & II trials are fully enrolled and being conducted under Special Protocol Assessment with the FDA. We are targeting top line data from these trials in the second half of this year. WuXi AppTec: Why do you think your approach can be more successful than other companies’ drug programs in this disease? How will it fit in the MS market space? Michael Weiss: Right now, we expect that ublituximab will be the third CD20 to market, following ocrelizumab, which is currently selling, and ofatumumab, which is expected to enter the market in the next six months or so. To my knowledge, there are no other CD20s in development or late-stage development for MS. We believe ublituximab may offer several advantages over the other CD20’s for MS, including the convenience of being a one-hour infusion every six months, versus the current 3-4 hour standard of care with ocrelizumab, at what we expect to be a competitive price designed to optimize patient access. WuXi AppTec: Sounds very promising! Finally, when do you think your drug may be available to patients? Michael Weiss: Assuming all goes well, we plan to possibly prepare a regulatory filing next year. If that is the case, we may be looking at FDA approval in late 2021/2022.    

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2020/05/19

Disease-Modifying RNA Therapies from Recode Therapeutics Target Rare Lung Diseases

The recent merger of TranscripTx and ReCode Therapeutics created a company that combines RNA drug expertise with a non-viral lipid nanoparticle (LNP) delivery platform to develop new genetic therapies for treating the underlying causes of two rare lung diseases: cystic fibrosis and primary ciliary dyskinesia (PCD). The new company retained the ReCode Therapeutics name and made headlines with an oversubscribed $80 million Series A round of financing in March 2020. David Lockhart, Ph.D., former TranscripTx CEO, is the president and CEO of the merged company. He said an essential element in the success of any new genetic therapy depends on a safe, effective and targeted delivery vehicle. The non-viral LNP delivery platform, Lockhart observed, was “a big part of getting the merged company funded. It can be used to package different types of genetic therapy payloads and can be tuned to deliver them in different ways, get them to where you want them to go, and deliver them safely and repeatedly.” Cystic fibrosis is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene which encodes the CFTR protein. An absent or defective protein leads to airway hydration problems, inflammation, lack of mucociliary clearance and frequent airway infections – impairing lung function and leading to life-threatening respiratory failure. The rare disease affects more than 70,000 people worldwide. ReCode is developing a transfer RNA (tRNA) therapy to treat a subpopulation of cystic fibrosis patients with so-called nonsense mutations. The tRNA therapy is delivered into lung cells to enable production of the functional CFTR protein and restore lung function. PCD is caused by mutations in a number of different genes that result in dysfunctional cilia on lung cells. When working properly, the hair-like cilia wave back and forth to clear mucus and harmful bacteria from the lungs. When the cilia are defective, mucociliary clearance is impaired, and inflammation and infections damage lungs creating chronic breathing difficulties. Only 3,000 cases of PCD have been confirmed in North America, but according to the PCD Foundation, limited awareness and under diagnosis of the disease means the number of patients could be closer to 25,000. PCD is sometimes considered less severe than cystic fibrosis. But children with PCD suffer from similar complications as those with cystic fibrosis and face shortened life spans due to respiratory failure. In addition, there are no treatments available. ReCode has developed a messenger RNA (mRNA) therapy delivered into cells to produce normal versions of the proteins that make cilia, restoring proper ciliary function on cells throughout the airways and lungs. ReCode’s cystic fibrosis and PCD RNA therapeutics are nebulized and turned into an aerosol that patients will inhale into their lungs. The therapies are in preclinical development and the company expects to file Investigational New Drug applications with the US Food and Drug Administration (FDA) in 2021 to begin clinical trials in both programs. As part of its ongoing series exploring the challenges of rare disease drug development, WuXi AppTec Communications discussed with Lockhart how ReCode’s RNA therapies represent a significant advance in the treatment of cystic fibrosis and PCD. Lockhart has more than 25 years of drug discovery and development experience. Before joining ReCode, he held scientific and executive positions at Amicus Therapeutics, Ambit Biosciences and Affymetrix. Lockhart received his Ph.D. from Stanford University and was a postdoctoral fellow at the Whitehead Institute in the Biology Department at the Massachusetts Institute of Technology. WuXi AppTec: What are the major differences between developing drugs for rare diseases and for more common diseases? David Lockhart: As you know, approximately 7,000 rare diseases have been identified. Obviously, for some rare diseases one of the challenges is that there are so few patients that it’s hard to find enough of them for clinical trials. You have to have clinical sites all over the world and even then, it can be very challenging, especially with “ultra-rare” diseases. Also, rare diseases are often under-diagnosed because many physicians have never seen a patient with a particular disease. So in addition to small patient populations, there is not a lot of information about some of the diseases. WuXi AppTec: What are some of the regulatory challenges you face, such as determining the appropriate endpoints, or outcomes, for clinical trials? David Lockhart: The challenges differ based on the disease. We’re fortunate that for cystic fibrosis and PCD the drug development and regulatory paths are fairly well-established and clear. That’s because of the work done by Vertex Pharmaceuticals to get its drugs approved for cystic fibrosis. The clinical path and primary endpoints, and even secondary and supportive endpoints, are very similar for cystic fibrosis and PCD. But in other rare diseases, if no drugs have been approved, no primary endpoints have been developed for clinical trials and it may not be clear what the clinical endpoints should be. As a drug developer, you may have to create endpoints of your own, then validate them and convince the FDA they make sense. That can be a very difficult process. And then for some rare diseases, the endpoints can be difficult to achieve. For example, if the primary endpoint involves slowing progression of the disease, it can take a long time to see a difference between the treated group and placebo group or an untreated historical control group. For our company, we’re very fortunate to have a primary endpoint that has already been used as the basis for approval of other cystic fibrosis drugs. Just as important, that endpoint has shown rapid improvement in lung function within weeks, not just a slowing of progression over a year or two or longer. That’s a huge advantage for our programs in cystic fibrosis and PCD. WuXi AppTec: What are some of the business challenges, such as raising funds for rare disease drug development? David Lockhart: The primary thing we look for is unmet medical need; that we’re really going after a problem in need of a solution and that patients are in need of a therapy. But then there also are practical matters of selecting diseases with enough patients to run clinical trials and enough patients in the world to make the drug commercially viable. In the context of raising money, the latter is an important part of the conversation. People need to know this is a disease that’s treatable, that a drug can get approved and that with approval the drug can be commercially successful. WuXi AppTec: How important are government incentives, such as the FDA’s orphan drug designation, for rare disease drug research and development? David Lockhart: The short answer is they are really important. With the orphan drug designation there is guaranteed market exclusivity. If your drug gets approved, and has orphan drug status, you’ll know you have a good position commercially. The orphan drug designation also is important because there’s additional help from the FDA. There are more interactions with the agency’s regulators and specific encouragement to meet with them early and often to look at clinical trial design and to make sure things are on track. By the time you get to the more advanced clinical development stages you already know the regulators involved. You’ve established a relationship, and you’re able to work together to develop the drug. WuXi AppTec: How much progress has been made in cystic fibrosis research and drug development? Is a cure in sight? David Lockhart: I can take those two questions one at a time. There’s been significant progress in cystic fibrosis, especially recently. The discovery of the gene and protein that cause cystic fibrosis occurred about 30 years ago. The hope early on was that therapies could be developed relatively rapidly. The disease turned out to be a lot more difficult to treat than people had hoped during the early days. But the good news is there have been multiple drugs approved recently for cystic fibrosis from Vertex Pharmaceuticals. The company has had a series of different small molecule drugs approved that are appropriate for different genetic subsets of the cystic fibrosis population. As a result of all that work, there are therapies that should benefit approximately 90 percent of people with cystic fibrosis. So that’s good progress, and with our targeted genetic therapies we want to reach the 10% of patients who don’t have treatment options yet. But note I said benefit, not cure. The drugs lead to improvement in lung function. They can lead to other improvements in cystic fibrosis symptoms and quality of life as well. But they are not a cure. People still have the disease. They have to keep taking the drugs for life, but it’s certainly better than no therapy at all. There’s also a new type of therapy that has moved into clinical studies from Translate Bio. It’s not a small molecule. It’s a nebulized mRNA therapy that is inhaled into the lungs. The mRNA therapy instructs cells to make the CFTR protein required for normal lung function. Translate Bio has just started clinical studies, so it’s not clear yet whether it’s going to work and how well it will work. But it is encouraging that a new type of drug is going into clinical studies. Do I expect a cure? It depends on what you mean by cure. A cure is a therapy that when administered the patient effectively no longer has the disease. I think that’s still a ways off. But the next best thing is to have treatments that basically make it so the symptoms of the disease are greatly reduced and that people can live long full lives. I think that actually is possible. I don’t know if that counts as a cure, but it certainly counts as a huge improvement in which you basically turn the disease into a manageable condition. Patients still have the underlying cause. They still have the mutation in the CTFR gene. But they can take a medicine and have a long, full, rich life. WuXi AppTec: What is your company’s approach for treating cystic fibrosis and PCD, and how far have you progressed? David Lockhart: For both cystic fibrosis and PCD we have been able to show we can deliver our RNA therapies into human cells using the cell-based model that’s considered to be the gold standard for studying the human airway and testing new approaches. We have been able to show that we can deliver our RNA therapies into those human cells and correct the functional deficiencies. In the case of cystic fibrosis, we show that we get recovery of the CFTR protein function. In PCD, our therapy generates cilia that function normally on the cells. We also have shown we can deliver these therapeutic agents into the lungs of animals and we can do that repeatedly and safely. Now the next step is to do those same types of delivery experiments in larger animals with lungs that are more like those in humans. WuXi AppTec: What kind of RNA therapies are you developing and how do they work? David Lockhart: They are a little bit different for the two programs. For cystic fibrosis we’re delivering tRNA, which helps decode the instructions in mRNA to make proteins. Our tRNA therapy makes it so the CFTR mRNA with nonsense mutations can be translated into a functional version of the CFTR protein, rather than producing a truncated and non-functional protein. The nonsense mutations in the CFTR gene occur in about 5% to 10% of cystic fibrosis patients. These are people with cystic fibrosis who are not helped by the Vertex small molecule drugs. The nonsense mutations in the CFTR gene lead to a premature stop in making the CFTR protein. When the protein is being made from the natural mRNA, the cell machinery comes to a halt so that instead of making the full length protein needed in the airway cells, only part of the protein is made, and it tends to degrade very rapidly. It’s as if the person does not have the needed protein. Our tRNA drug reads the nonsense mutation in the mRNA and makes it possible for the cellular machinery to continue making a functional version of the CFTR protein. Basically, it reinterprets the mutated mRNA so the CFTR protein can be made. It’s very clever, and it makes great sense. It’s a new type of drug. In PCD, people are born with mutations in particular genes responsible for making cilia that don’t move as they should. We deliver into the cells the full-length, normal mRNA that encodes the properly functioning cilia protein. The deficient mRNA is still there, we just add in the correct copy to make the correct version of the protein so that the ciliary machine can work properly. WuXi AppTec: How important are patient advocacy groups and patients in your drug development efforts? Are they helping with clinical trial design? What roles do they play, other than participating in clinical trials, of course? David Lockhart: The patient advocacy groups are extremely important. Sometimes one of the benefits of working on rare diseases is that the patients are very knowledgeable. They’re very engaged. They know about their disease, about the genetics, and about what works and what doesn’t. They know their doctors well. The patient advocacy groups and foundations also set up patient registries. They often are involved in natural history studies. The registries and natural history studies are very helpful in identifying patients, monitoring them and tracking them on how their disease progresses over time. All of these elements are immensely helpful in terms of designing clinical studies, populating the studies, interpreting the studies, and knowing what endpoints to use and what’s likely to happen in those endpoints over time. Fortunately for both cystic fibrosis and PCD there are very well-established foundations and patient groups. It’s an incredible network that involves not only patients, but also caregivers, families, doctors and nurses. WuXi AppTec: In general, what technological breakthroughs will be game-changers for drug developers over the next five years? Will they have an impact on improving the efficiency of drug approvals and lowering the cost? David Lockhart: We’re getting smarter about drug discovery and development all the time. We have made significant progress in developing new kinds of therapies. But the biggest breakthrough in terms of creating better drugs and developing them more rapidly and more efficiently is the delivery. It’s really the delivery side of the equation that can be the most challenging. We need better delivery vehicles that are safe and versatile, meaning they can be used with different types of payloads, such as RNA drugs and proteins, and even the combination of the two. We need to be able to target the drugs so that when they’re delivered they go to the right places, in the right amounts. They go to the right tissues. They go to the right organs and the right cell types. We will speed up drug development and make it more cost-effective when we have a better tool box for delivery of all the new types of genetic therapies.  

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2020/05/19

Powerhouse Collaborator, MassBio’s President & CEO, Bob Coughlin Optimistically Embraces Industry Changes

MassBio is a 35-year standing life sciences trade association consisting of over 1,300 member companies. Today, their programs, services, and industry-critical mass influence, allows them to be truly patient-driven. By utilizing their unparalleled network of innovative companies and industry thought leaders, MassBio is able to advance policy, promote education, and push for innovative cross-industry collaborations. Leading MassBio is President and CEO, Bob Coughlin. Bob focuses on promoting industry growth through the advancement of Massachusetts’ leadership in the life sciences, adding value to the healthcare system, and improving patient lives. Another vital focus for Bob and MassBio is on ensuring collaboration and innovation between the government, industry, and academic sectors. Before joining MassBio, Bob served as the Undersecretary of Economic Development within Governor Deval Patrick’s administration, where he prioritized both healthcare, economic development issues, and advocated for the Massachusetts life sciences industry. Prior to that, he was elected as State Representative to the 11th Norfolk district for three terms. He has also held senior executive positions in the environmental services, capital management and venture capital industries. Currently, Bob serves on the board of directors for The Schwartz Center for Compassionate Healthcare. He is also a member of the Scientific Advisory Board at Boston Children’s Hospital. Recently, Bob sat down with Rich Soll and the WuXi AppTec Content Team to discuss MassBio, their role in the COVID-19 response, industry response takeaways, future preparations, and Bob’s insights into our path forward. WuXi AppTec: What makes Massachusetts unique in the life sciences? Bob Coughlin: We have a critical mass of companies, we have a critical mass of highly educated people, and we have 122 colleges and universities in this state, which is one-sixth the size of California. The high talent pool is a key resource for the life sciences here. We not only educate our own here, we educate the best and brightest from all over the country and around the world. WuXi AppTec: Where does MassBio fit in? Bob Coughlin: Our mission at MassBio is to leverage Massachusetts’ leadership role in the life sciences. We ensure that academia, industry and government are working collaboratively, so together, we can continue to drive innovation. We do that through many different programs and services we offer. Many folks think of us as a lobby entity, but in fact, government affairs amounts to only 15-20% of what we do. Our Innovation Services group works with the amazing tech transfer offices, including five of the top six NIH-funded academic medical centers in the country. They manage our MassCONNECT mentoring program where we funnel those companies, along with other under-the-radar startups, into our Pharma / Partnering days for engagement in business development activities. We help companies grow and raise much-needed capital through traditional fundraising forms or partnerships with large Pharma companies. We help recruit Pharma companies to this area to the extent that today, 18 of the top 20 Pharma companies have a significant physical presence here. We worked closely with the state government on economic development, in a life sciences initiative, which started in 2008 and resulted in a $1 billion investment over 10 years. This really put us in a position to succeed. We offer professional development forums and business services to member companies. For example, MassBio Edge enables our members to procure services and goods at bulk rates, thus offering significant savings, so our membership can work more effectively and collaboratively to address the unmet needs of patients. WuXi AppTec: MassBio has been very involved in taking action during the COVID-19 crisis. Bob Coughlin: Yes, when COVID-19 was coming, we realized early on that this was a particularly tricky virus that could perhaps turn into a pandemic, which it did. Our partners in the health care system were saying, “We are unable to keep our supply chain flowing as it relates to PPE, ventilators, lab supplies, etc. We’re not going to be able to order a lot of the things that come from supply chain partners overseas, because those folks overseas were shut down.” It then became revealing to me — we live in a world of “just in time” inventory. As a consequence, our health care providers were not going to have the proper protection required to care for patients infected with COVID-19. This became our call to action. WuXi AppTec: What action did you take? Bob Coughlin: We have 1300 members in MassBio and there are 500 members in Mass Medic, our counterpart in the medical device space. We thought that if we could inventory in short order what was existing in our member company supply rooms, we may be in a position to help fill some of those voids during the crisis. That led to the creation of the Massachusetts Life Sciences Emergency SupplyHub, known as the SupplyHub, where we could inventory everything our members were able to donate to health care workers. Then, on the needs side, we were able to relay requests from frontline health care providers to the state’s Command Center. First, within 24 hours of initiation, we had close to 200 member companies donate what they had in inventory. Second, more than 500 people volunteered their time to help with supply chain procurement opportunities while also procuring supplies for the hospitals. Third, and most spectacularly, was the giving of commitment by member company volunteers. They would go to the battlefield when frontline health care workers needed relief due to fatigue or sickness. This included doctors, nurses, pharmacists, lab technicians and others. Information was then provided daily to the State and to the Command Center, which then triaged the supply inventory based on needs. Through partnership with the National Guard and Massachusetts Emergency Management Agency (MEMA), the supplies (PPE) were distributed to the sites. The state took over all the operations a few weeks ago, which allowed us to set our energies on getting people back to work. WuXi AppTec: What did you do there especially to be compliant with social distancing? Bob Coughlin: We are working on a program called RESET. Phase 1, which is now completed, was ensuring the buildout of the SupplyHub with sufficient PPE; you can find that information on our website. The next phase focuses on returning more non-essential employees to work. WuXi AppTec: What area do you consider to be of strength of Massachusetts? Bob Coughlin: Our strength is true innovation. Diagnostics, therapeutics, and vaccines go hand in hand, yet who would have thought that in our lifetime the most important thing to the world economy would be a vaccine? In Massachusetts, we have a critical mass in all three areas; there is no better place than here to solve this problem. WuXi AppText: What do you think is the most important lessons learned so far? Bob Coughlin: We have serious supply chain issues in the US that hamper our progress. Despite significant growth in large molecule manufacturing in Massachusetts, certain materials are not readily available so that hinders progress. Yet, this serves as a stark reminder to how essential the life sciences are to the economy in Massachusetts. WuXi AppTec: Yes, health of the life science industry is critically important in Massachusetts. Bob Coughlin: I’ve lived it every day for the last 18 years. My son has cystic fibrosis, but he now has a therapy that works – but it took 18 years to get there. That is why this industry means so much to me—it’s personal. People who once took health for granted think much differently now if they are living through the COVID-19 pandemic, especially if a loved one has suffered from infection. WuXi AppTec: What are two critical areas MassBio has been addressing? First, collaboration is more important than ever because we are in this together; everyone needs to contribute if we are to succeed. The one thing that I’ve noticed, and was inspired by, is the level of collaboration not only between various sectors in the life sciences, but also between the private sector and government. Second, the COVID-19 situation today is unprecedented and we need to better prepare for the future. People are very scared and worried; there is so much misinformation. To help elected officials better communicate with their constituencies, MassBio has established various kinds of briefings, some daily, for elected officials and government officials, especially ones where the complex science needs to be distilled down. WuXi AppTec: Looking to the future– will things be different? Bob Coughlin: Yes, a new norm will result: one that depends on virtual connections and measurable social distancing. I would imagine, until an effective vaccine is in place, we will incentivize infectious disease R&D. We will see more cross-state collaborations and more transparency between elected officials, their constituencies, and supporting industries like ours. I’m going to continue to be an optimist, because I know that this time period is going to change the way we work long-term. I think companies are going to realize that people can work remotely and increase productivity. And really, I think we’re going to be more flexible and nimble as an industry. I think that’s a great thing.   WuXi AppTec: What should be our priority? Bob Coughlin: The most important thing in the world right now is a vaccine; I don’t think you can put a price on it. Our industry has really been showing compassion and willingness to solve problems, and not prioritizing returns. They’re doing the right thing. Closing out, I think we’re going to continue to see more collaborations between companies and private-public partnerships. At this time, many people would say drugs are too expensive. I would say that drugs are too expensive to invent. If this is forcing us all collectively to get better at how we do what we do, it will be less expensive at the end of the day; we’ll be able to bring even more value to the health care system.      

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