Every month, MassBio spotlights a member company and its efforts in advancing the life sciences industry and supporting the patients we serve. In December we spoke with Garo Armen, Ph.D., the Co-founder, Chairman and Chief Executive Officer of Agenus, Inc. and the Founder and Chairman of COAF (Children of Armenia Fund). Both organizations draw their inspiration from the life and legacy of Armen’s grandmother, who taught him about the power of the immune system and the importance of leaving no one behind.

Tell us about your organization, its mission, and current activities.

Every day at Agenus, our eyes are set on our mission: to end the suffering of cancer patients. For over 29 years, Agenus has been dedicated to the discovery, development, and manufacturing of immuno-oncology (I-O) products. Our research and innovation engine has built a comprehensive pipeline of novel immunotherapies that empower the body to fight cancer, including checkpoint inhibitors, immune activators, and tumor microenvironment conditioning agents.

Our flagship program, botensilimab, represents a potential paradigm shift in immunotherapy. As a next-generation immune activator, botensilimab was designed to detect cancer, attack the cancer, and train the body’s immune cells to boost both innate (our natural defense mechanisms) and adaptive (our immune system’s ability to adjust and learn to defend us) immune responses in cold tumors. We are investigating botensilimab across multiple clinical trials, including a phase 2 study in colorectal cancer.

How do your organization’s activities help patients now and into the future?

In recent years, the medical community has started to unlock the power of immunotherapy; however, for many patients, I-O has yet to offer any benefit. This is especially true in cold and late-stage cancers.

Cold tumors, like colorectal, are notorious for “hiding” from the immune system, preventing the body from identifying and destroying them. The first I-O revolution brought benefit to “hot” tumors, including melanoma. Agenus is poised to bring a second revolution that could extend the benefit of I-O to cold tumors to help a larger patient population.

Botensilimab’s potential to turn an unresponsive tumor to a responsive one—turning cold to hot—could offer tremendous benefit to patients. In advanced cancer, patients who have failed prior therapies are left with extremely limited options and low chances of benefit. Now, we are even seeing botensilimab’s potential in earlier settings as well as in combination with chemotherapy agents, which could prevent cancers from ever reaching those advanced stages.

What do you see as the biggest challenge facing the life sciences industry today?

The life sciences industry continues to stand at the forefront of rapid innovation and change, giving rise to some of the most cutting-edge technologies and therapeutics, including immunotherapy. However, the fast-paced nature of this environment presents a unique challenge – the need for every area within the industry to keep pace.

Harnessing the power of the immune system remains an emerging frontier in oncology, and this gives rise to challenges in the lack of consistency in how I-O products are developed, tested, and regulated today. Our knowledge of the immune system is still evolving, and as new therapies continue to emerge, the knowledge gaps between the industry, healthcare professionals, and patients continue to grow. To address these challenges, the industry must find innovative solutions to enhance education and foster partnership with the oncology community so that the very best treatment approaches can be directed at ending cancer.

What’s next for your organization / what are you focused on in the coming year?

Agenus remains laser-focused on delivering the breakthrough potential of our novel investigational immune activator, botensilimab. We believe botensilimab represents a paradigm shift in I-O, with its ability to activate the immune system in multiple cold, traditionally unresponsive tumors and its versatility to be used in a range of treatment settings, including prior to surgery.

Our immediate priority in the coming year is to file our Biologics License Application (BLA) with the U.S. FDA for botensilimab in MSS colorectal cancer. Looking ahead, we plan to further advance botensilimab in other tumor types and expand into earlier lines of therapy.

At Agenus, we recognize the importance of the individuality of each patient battling their cancer and our goal is to make our treatments available to help as many patients as possible.

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Every month, MassBio spotlights a member company and its efforts in advancing the life sciences industry and supporting the patients we serve. In November, we spoke with Jean-Paul Kress. As CEO, Jean-Paul Kress leads MorphoSys in its mission by focusing on translating scientific innovation into medicines that can redefine how cancer is treated. Since becoming CEO in 2019, Jean-Paul has guided MorphoSys through a strategic and cultural transformation to its current status as a global biotechnology company with an approved medicine and several late-stage development opportunities. As part of this evolution, Jean-Paul championed the company’s acquisition of Constellation Pharmaceuticals in 2021. Jean-Paul received his M.D. from Faculté Necker-Enfants Malades in Paris, and graduate and post-graduate degrees in biochemistry and in molecular and cellular pharmacology from Ecole Normale Supérieure in Paris. Jean-Paul is based in Boston.

Can you tell us about your organization, its mission, and current initiatives?

At MorphoSys, our mission is clear – More life for people with cancer.

We are a global biotechnology company based in Boston and Munich, Germany. Originally a research and development technology provider, MorphoSys has been transformed by me and my team into a company focused on the clinical development and commercialization of innovative oncology medicines.

We have an advanced pipeline with strong growth opportunities across a wide range of difficult-to-treat, debilitating and often deadly cancers. Our largest and most immediate opportunity lies with pelabresib, our investigational BET inhibitor, which has the potential to become a foundational first-line therapy for myelofibrosis patients. 

How does your organization’s activities help patients now and into the future?

Everything we do at MorphoSys is aimed at making a profound difference for cancer patients and their loved ones.

In 2020 and 2021, our immunotherapy tafasitamab was granted accelerated approval as Monjuvi^® in the U.S. and conditional approval as Minjuvi^® in the European Union, respectively, for use with lenalidomide in certain patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). These milestones enabled MorphoSys to forge strong relationships with the healthcare providers and specialists dedicated to treating these patients, bringing us closer than ever to the community.

We have the opportunity to do this again with pelabresib, which has the potential to truly improve the standard of care for patients with myelofibrosis. Before the end of this year, we will share topline results from the Phase 3 MANIFEST-2 trial of pelabresib in combination with ruxolitinib in the first-line setting. Previously presented Phase 2 data plus the enthusiasm expressed by oncologists and other experts underscore the promise of this investigational therapy. And we’re not stopping there. Beyond myelofibrosis, we are also exploring pelabresib’s therapeutic potential in other myeloid diseases, including essential thrombocytopenia and lower-risk myelodysplastic syndromes.

What do you see as the biggest challenge facing the life sciences industry today?

Increased market volatility in recent years has led to a period of turbulence in the life sciences industry. Cautious behavior from investors and the financial community has resulted in many distressed biotechnology companies.

Despite this turbulence, MorphoSys has remained stable and successful by transforming into a late/commercial-stage biotechnology company. We developed a cohesive business model and strategy, choosing to focus on oncology based upon previous experience – including successfully taking a medicine from the clinic to patients. 

For companies to be successful, it is crucial to strategically prioritize unique strengths and deliver differentiated, late-stage clinical results. In this market, access to capital is tied to a company’s ability to generate tangible innovation. Not only is this in the best interest of patients, it also propels companies like ours to the next level. 

What’s next for your organization / what are you focused on in the coming year?

Our primary focus is to deliver the topline results from our Phase 3 MANIFEST-2 study of pelabresib for first-line treatment of patients with myelofibrosis by the end of this year.

Based on strong Phase 2 findings, we are very optimistic about the outcome of this pivotal study. We are therefore proactively preparing our regulatory submissions, so we’re able to quickly apply for approvals in both the U.S. and Europe. Following the release of Phase 3 data, we look forward to discussing the results with regulatory agencies. Our hope is to bring this therapy to patients as quickly as possible.

Beyond pelabresib, we have made exceptional progress with our pipeline in a range of difficult-to-treat cancers and, as a result, have a rich set of catalysts from our pivotal studies over the next two years. Pivotal topline data readouts are expected in 2024 for the Phase 3 inMIND study of tafasitamab in combination with lenalidomide and rituximab for relapsed/refractory follicular lymphoma or marginal zone lymphoma and in 2025 for the Phase 3 frontMIND study of tafasitamab plus lenalidomide and R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone) as a first-line therapy for DLBCL.

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Every month, MassBio spotlights a member company and its efforts in advancing the life sciences industry and supporting the patients we serve. In October, we spoke with Jit Basak, Founder and CEO of Catailyst Inc. Jit, a first-time entrepreneur, a former medicinal chemist, and a former FINRA registered investment banker is a passionate and visionary leader who strives to create innovative solutions for complex problems in the life science industry. Earlier in his career, Jit spent nine years in scientific roles at both midsize biotech and large pharmaceutical companies: Chemocentryx, Inc. and Pfizer, Inc. Later, he was a Transaction Lead of the Corporate Development team at Shire/Takeda; Inotek Pharmaceutical as well. He holds numerous degrees: post-doctoral research at the University of Chicago, an MBA from the University of Connecticut School of Business, a Ph.D. from the University of Illinois at Chicago in synthetic organic chemistry, and a master’s degree from the Indian Institute of Technology Kanpur, India.

Tell us about your organization, its mission, and current initiatives.

Catailyst produced a database and data visualization platform that analyzes scientific, clinical, and business announcements through a combination of automation and artificial intelligence. The company was founded in 2019 and stayed stealth for three years while it developed its proprietary extractive artificial intelligence methods leveraging deep learning models and NLP methods. We focused on extractive artificial intelligence to ensure the integrity and reliability of our data. This includes methods of identifying, extracting, and categorizing relevant scientific and business information without human intervention. Today, Catailyst processes 500+ press releases, 100+ clinical trials-related information, and hundreds of SEC filings every day making data access for drug discovery easy, efficient, and at a fraction of the cost.

How do your organization’s activities help patients now and into the future?

We help our biotech and pharmaceutical researchers and executives accelerate their drug discovery and development by providing access to accurate and up-to-date scientific and business information through a web-based portal at an affordable price. In addition, our platform provides customizable real-time alerts, superior search functionalities, and state-of-the-art visualization tools. We aspire to have a lasting effect on medicine creation for investment decisions, portfolio prioritization, new strategic initiatives, clinical trial planning, and transaction executions. Our goal is to help accelerate the research and development of new medicines.

What do you see as the biggest challenge facing the life sciences industry today?

There are several challenges that the industry is facing today.

1. Increased generic competition for Drugs and Biologics, Pricing pressure from the Inflation Reduction Act (IRA): differentiated price controls for Biologics (13 years) vs. small molecules (9 years) and many other forces have created shifts in strategies for Pharma and investors both in recent years. Undoubtedly, indication prioritization and science selection have become more than ever important for long-term sustainability.
2. Today, business leaders are constantly asking questions: a) Is there a real unmet need? b) if the new drug can exceed current market standards; c) how to design trials to establish a clear differentiation of better clinical outcomes.
3. The cost of finding accurate data to support such a decision is currently very high and labor intensive due to overwhelming data flow and lack of an integrated data source. We often manage these situations with multiple subscriptions, or a series of internet searches to assemble information. Manual data verification and curation become highly time-consuming, causing delays in business decisions.

What’s next for your organization / what are you focused on in the coming year?

We aim to transform the way life science data and intelligence are accessed, analyzed, and utilized by biotech/pharma professionals, investors, and consultants. To date, we have efficiently and economically managed the company and have not required a formal capital raise till 2022. Now, we are in the middle of our first seed round and have raised more than a quarter of a million dollars, which gives us a good runway to propel the company forward. Our low-cost business model involved a partnership-based approach and non-dilutive government funding: a foundational partnership with a data science company Xelpmoc, a sales and marketing partnership with Launchit ventures, and MLSC funding. Since our initial launch of the product, we have been constantly improving the platform using customer insights. Most exciting to share is that we recently integrated visualization and clinical trials analytics. We will continue to build a few additional features: predictive data analytics, stock market data integration, and layering measured generative AI to the platform. Today we are ready to take it to all users.

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Tell us about your organization, its mission, and current initiatives. How do your organization’s activities help patients now and into the future?

Firalis Molecular Precision (FMP) operates as a veritable biomarker investigation agency for biotech and pharmaceutical companies, ultimately improving the development, effectiveness, and personalization of therapeutics. Our expertise revolves around biomarkers, those biological breadcrumbs that indicate disease presence, severity, or response to therapy. The identification and understanding of these biomarkers are the backbone of targeted therapies and personalized medicine, a realm in which many pharmaceutical and biotech companies are striving to make their mark. We explore the -omics, or the complete sets of biological components within the body – genes (genomics), proteins (proteomics), lipids (lipidomics) and metabolites (metabolomics) – with utmost precision. This deep analysis reveals the intricate pathways of disease and offers a profound understanding that directly informs drug discovery and development. In addition, FMP offers cutting-edge sample storage services and manages significant volumes of data generated by –omics investigations via bioinformatics and statistics to extract comprehensive insights from clinical samples.

What do you see as the biggest challenge facing the life sciences industry today?

Artificial Intelligence (AI) and Machine Learning (ML) are transforming the -omics fields, enhancing data analysis, accelerating biomarker discovery and drug development, and facilitating predictive modeling and integrative analysis. However, this revolution isn’t without challenges. Concerns around data privacy and ethics loom large as AI and ML techniques increasingly involve patient data. Lack of standardization in data collection, analysis, and interpretation can lead to inconsistencies. Overfitting models due to the complexity of biological systems and vast omics data might yield inaccurate predictions. Thus, while AI and ML hold tremendous promise for advancing omics and personalized medicine, addressing these challenges is critical to harnessing their full potential effectively.

What’s next for your organization / what are you focused on in the coming year?

Looking ahead, Firalis Molecular Precision’s ambition is to expand its global presence, thereby furnishing comprehensive support for drug development — from research to Phase 3 clinical trials — across the globe. Our endeavor aims to streamline partners’ processes, enhance analytical reproducibility, expedite turnaround times and give access to the last technological innovations. The key milestone of the next years will be the opening of a lab facility in the US to get closer to our US partners and customers.

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Every month, MassBio spotlights a member company and the great work they’re doing to advance the life sciences industry and support the patients we serve. For July, we spoke with GraVoc Vice President ‑ Information Security and IT Nate Gravel. In this role, he oversees the development and delivery of GraVoc’s managed IT and cybersecurity services. Nate has a B.A. from Trinity College (Hartford, CT) and a M.A. from Middlebury College (Middlebury, VT).

Tell us about your organization, its mission, and current initiatives

Founded in 1994, GraVoc is a technology consulting company located in Peabody, Massachusetts. Since its inception, GraVoc has been committed to helping customers in the life sciences industry solve business problems and reimagine their operations through the development, implementation, and support of technology-based solutions, including:  software development – analysis, design, customization, and implementation of software applications; IT managed services – LAN/WAN design, implementation and support services; information security – advisory, regulatory compliance, audit, and cybersecurity testing; web and media – website and web application development and digital marketing.   Today, GraVoc partners with many life sciences organizations to ensure that their technology only accelerates the important work they are doing.

How does your organization’s activities help patients now and into the future?

While many of the solutions developed by GraVoc are not patient-facing, they can be considered, directly or indirectly, patient-enabling.  In short, we try to eliminate technology-related barriers for our partners in life sciences so they can, in turn, spend more time helping their patients.

What do you see as the biggest challenge facing the life sciences industry today?

Like organizations across all industries, life sciences organizations face significant challenges now and into the future with regard to anticipating and planning for how technology will affect their development and growth. The life sciences industry often finds itself at the intersection of groundbreaking research and development and transformative technology.  For all life sciences organizations, navigating this intersection remains crucial to the success and longevity of both the businesses themselves and to the patients they serve.

What’s next for your organization / what are you focused on in the coming year?

GraVoc’s focus will remain on providing value to the life sciences industry through partnership.  By staying in front of and advising on technology trends, GraVoc can allow customers to focus on the problems they’ve set out to solve.  We see this role as a small but significant contribution to the important work they are doing.

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Every month, MassBio spotlights a member company and the great work they’re doing to advance the life sciences industry and support the patients we serve. For June, we spoke with MarathonLS CEO Rob MacDougall. Rob founded Marathon Life Sciences in 2018 after working in the life science and pharmaceutical industries for over 20 years. Rob’s experience and knowledge in product distribution, service management and business development allows him to lead a team of passionate, creative individuals eager to make the jobs of laboratory professionals more concentrated and improve the lives of patients.

Tell us about your organization, its mission, and current initiatives

MarathonLS is a Boston-based laboratory operations support partner enabling the race for innovation. We do this by offering our customers tailored solutions through our lab equipment service, vendor managed inventory (VMI) program and software, and a vast portfolio of plastic lab consumables and equipment.

We understand the critical work being conducted in labs and we are committed to ensuring that these labs are never disrupted due to equipment breakdowns and supply chain challenges. MarathonLS is focused on providing our customers peace of mind through our 24-hour response time, in-stock products, and transparency and clear communication. We are continuously innovating to deliver solutions to our customers to address unmet needs in the marketplace. MarathonLS is able to offer your lab customizable support to ensure your lab is running efficiently at all times.

How does your organization’s activities help patients now and into the future?

MarathonLS ensures labs do not miss a step in their race for innovation. Marathon understands that critical research and innovation is being conducted by our customers and so it is our mission is to ensure these laboratories are always running efficiently. Marathon’s service team regularly performs preventative maintenance on lab equipment and educates lab professionals on the importance of equipment upkeep to mitigate disruptions throughout research. They also maintain the fastest response time in the industry on emergency repair requests to further ensure these disruptions do not occur. Marathon’s newest initiative, Marathon Pacer, is a full-service vendor managed inventory program that works alongside lab operations teams to streamline procurement through on-site and off-site support, software and brand agnostic product offerings. Pacer customers have not had a single product stockout since implementing Pacer.

Marathon is backed by a team of individuals who are agile and flexible to adapt to a dynamic industry that literally save lives. 

What do you see as the biggest challenge facing the life sciences industry today?

Prioritization of resources. With so much opportunity on the horizon, prioritizing resources such as personnel, equipment, funding, and time in a biotech lab is extremely complex and difficult. Because all of the work biotech labs do is critical to patient wellbeing, it is hard to prioritize resources based on importance. In other industries, it is easier to assign more resources to high-priority projects and fewer resources to lower-priority project, but what do you do when these projects are all priorities?

What’s next for your organization / what are you focused on in the coming year?

MarathonLS is in a phase of innovation and rapid development. We have introduced several programs to holistically serve our customers with lab products, asset management and inventory control. The newest introduction to Marathon’s offerings has been Marathon Pacer which is our tailored vendor managed inventory (VMI) solution, which provides transparency, flexibility and predictability. It also aligns with the objectives of all key stakeholders including: operations teams, scientists and finance teams. Within the next few months, Marathon will be introducing a portfolio of lab equipment and is committed to transitioning our portfolio of consumables to be made in the USA with over 70% already transitioned. We are constantly listening to the feedback of our customers and recognizing gaps in the market. From these insights, we are continuously building solutions to make it easier for scientists to conduct their work.

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Every month, MassBio spotlights a member company and the great work they’re doing to advance the life sciences industry and support the patients we serve. For May, we spoke with Joanne Smith-Farrell, Chief Executive Officer of Be Bio. Dr. Smith-Farrell is a mission-driven executive whose career has been devoted to leading teams conquering cancer and other life-limiting diseases. She currently serves as the Chief Executive Officer of Be Bio where she leads committed scientists, drug developers, business builders, and cell therapy operational specialists, united in one common mission – to fully unleash the power of engineered B cell medicines to change countless patient stories and provide previously impossible solutions across a wide range of diseases. Prior to joining Be Bio, Joanne held executive roles building businesses and developing medicines at innovative biopharma companies including bluebird bio, Merck, Pfizer, and Gene Logic.

Tell us about your organization, its mission, and current initiatives

Be Biopharma (Be Bio) is an innovative biotechnology company pioneering engineered B cell medicines, a new class of cellular medicines to treat a broad range of diseases.

Building on the research of Be Bio’s scientific co-founders at Seattle Children’s Research Institute, the company was founded on one simple, but powerful idea: what if we could engineer B cells to make any therapeutic protein we wanted – leveraging the biology of this extraordinary cell to produce therapeutic proteins at constant levels for years with a single infusion – redosably, allogeneically (off-the-shelf), and without toxic preconditioning? 

Today, Be Bio is helping to make this vision a reality by building a broad portfolio across rare disease and oncology and moving our programs towards the clinic.

How does your organization’s activities help patients now and into the future?

When you walk into Be Bio’s office, the first thing you see is: Eyes Locked on the Patient.

Why? Because we believe harnessing B cells will lead to a fundamental and transformative breakthrough that will change countless patient stories. Over millions of years, nature perfected B cells to produce the proteins we need to defend against pathogens and rogue cells. At Be Bio, we are engineering B cells to turn this power against today’s most challenging diseases. Our goal is to create medicines that function as protein factories and engraft in a patient’s bone marrow to produce therapeutic proteins for years with a single dose. The versatility and durability of B cells is extraordinary – we have the opportunity to develop previously impossible therapeutics and establish a new standard of care for patients.

What do you see as the biggest challenge facing the life sciences industry today?

A common challenge facing companies in the cell and gene therapy space is navigating manufacturing complexity. Recognizing this, we decided early on to establish end-to-end manufacturing capabilities. Today, all non-GMP manufacturing, process development, and analytical development are done in our facilities. GMP manufacturing for our first products is handled through a strategic partnership we formed with Resilience, Inc. in 2022. 

This partnership is deeply collaborative, and together we are driving innovation and reliability in cell therapy manufacturing to ultimately ensure this new class of B cell medicines is safely and efficiently created for patients.

What’s next for your organization / what are you focused on in the coming year?

Be Bio’s first development candidate is in rare disease, and we are focused on advancing this potentially transformative medicine to the clinic. Our proprietary cutting-edge platform allows us to rapidly engineer research candidates and we are fueling a multi-product pipeline of both autologous and allogeneic products.

Business development is also a focus. We have a strong, multi-candidate pipeline and our platform enables a versatile and efficient product engine across therapeutic areas. Potential partners recognize the impact we could make together, and we’re excited about the possibilities to develop medicines with some of the industry’s leading companies. 

Most importantly, we are nearing the hundred-person mark, and continue to build a team and culture where intensity and humanity are held in intentional balance. We are collectively aware that our mission is only possible because of our committed and talented team of B cell believers who walk through walls to unlock the potential of B cell medicines.

For more information, visit: be.bio.

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Every month, MassBio spotlights a member company and the great work they’re doing to advance the life sciences industry and support the patients we serve. For April, we spoke with Sharon Cunningham, CEO and Co-founder of Shorla Oncology. Named Ireland’s Best Young Entrepreneur 2019 and recognized as one of the 100 most influential CEO’s in Ireland by the Business & Finance Media Group 2020, Sharon is a PwC trained Chartered Accountant (ACA) with an MBA from University College Dublin Michael Smurfit Graduate Business School and a BSc in Finance from University College Cork Ireland.

Tell us about your organization, its mission, and current initiatives

At Shorla Oncology we develop and commercialize innovative oncology drugs. We focus on indications where existing treatments are limited, in shortage or inadequate for the target population.

Our mission is to make existing oncology treatments better through formulation re-innovation. 

Our development strategy of rethinking approved pharmaceuticals in oncology represents a focused approach that can dramatically reduce clinical burden, cost and risk while maximizing speed to market. This month, we announced FDA approval for Nelarabine Injection for the Treatment of T-cell Leukemia. We plan to have at least two products on market and 4+ products at a late stage of development by the end of 2024.

How does your organization’s activities help patients now and into the future?

Drug shortages pose a significant public health concern impacting patient’s health outcomes leading to delays in treatment or substitution with less effective therapies when the preferred drug product is not available. Our growing portfolio will bring accessible, affordable and life-changing treatments to patients, delivering a major contribution to patient care.

What do you see as the biggest challenge facing the life sciences industry today?

The global socio economic landscape and its impact on investment dollars. Research and development is a lengthy and capital intensive process which requires continuous financial support. However, the impact of global volatility has caused uncertainty for dealmakers and problems for life sciences companies in recent times.

What’s next for your organization / what are you focused on in the coming year?

Launch our recently FDA approved drug in the U.S., secure a product in-licencing deal for a synergistic oncology product, close our Series B funding round, grow our team in the U.S. and Ireland and continue to advance our pipeline.

For more information, visit: shorlaoncology.com.

If you’re interested in being featured in MassBio’s Member Spotlight, please see guidelines here.

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Every month, MassBio spotlights a member company and the great work they’re doing to advance the life sciences industry and support the patients we serve. For March, we spoke with Yvette Stallwood PhD., Head of Lonza Early Development Services. Yvette completed her PhD at the University of Birmingham (UK) and has a background in Virology, Cell & Molecular Biology. She leads Lonza’s Early Development Services. Her team is focused on de-risking the lead selection, optimization and early development phases of biotherapeutic proteins and vaccines with a particular focus on developability assessment and small scale protein expression.

Tell us about your organization, its mission, and current initiatives

Lonza’s vision is to bring any therapy to life.  We optimize scientific innovation and manufacturing technology to enable our customers to serve their patients and consumers. We provide a wide range of services and products from active pharmaceutical ingredients to innovative dosage forms for the pharma, consumer health and nutrition industries. Our offerings and scale mean we can provide our customers a one-stop solution of development and manufacturing services and technologies, from early phase drug discovery to commercial supply, and from drug substance to drug product, across a variety of molecule types.

In 2021, we supported more than 780 preclinical and clinical small and large molecules, more than 245 commercial small and large molecules and produced around 250 billion capsules.

How does your organization’s activities help patients now and into the future?

By combining technological insight with manufacturing, scientific expertise and process excellence, we help our biotech and pharmaceutical customers to deliver new and innovative medicines that help treat a wide range of diseases. Our purpose is to enable a healthier world, and this motivates us to deliver for our customers and their patients, every single day.

What do you see as the biggest challenge facing the life sciences industry today?

My answer is threefold;

1. Despite improvements in technologies, attrition rates remain high, therefore keeping costs associated with drug development high. Reducing attrition rates is critically important, specifically the identification and mitigation of potential issues early in development.

• Biotech funding goes through cycles. During downturns, additional pressure is placed on resources and there is greater focus on the early de-risking to help reach clinical proof-of-concept as efficiently as possible. At Lonza, about 60% of our early phase customers are small or emerging biotech companies. Backed by decades of experience, we offer flexible solutions tailored to their milestones and needs.

• Drug candidates are increasingly innovative and complex. More complex therapies, including cell and gene therapies, are seeing very high demand along with a growing appetite to increase manufacturing speed.

There are many  innovations which aim to address these challenges, from the technologies being used to develop and manufacture to the types of biopharmaceuticals themselves. We are seeing an increased use of bioinformatics, protein engineering, and risk mitigation platforms. Their use helps to reduce costs and accelerate development, especially from gene to IND, which – in the end – benefit the patient most.

What’s next for your organization / what are you focused on in the coming year?

We will continue to offer our clients world-class services and products that can support them through the process of bringing their therapies to life. Late discovery to IND phase of drug development is mostly carried out by small biotech companies and we will continue to offer personalized development programs to meet our customers timelines, molecule needs and investors’ requirements. Drug development has become increasingly competitive and leaves little room for error or delay. Selecting and optimizing the right lead candidate is critical and helps to maximize the chances of a successful First-in-Human campaign.  For over 15 years, Lonza’s Early Development experts have been supporting biotech and pharma customers in assessing, de-risking, and optimizing biotherapeutic drug candidates. Building on this expertise, we will expand our Early Development Services footprint this May by opening a new lab in Cambridge, Massachusetts (US) close to Kendall Square. The site will mirror the already trusted early non-GMP expression and in vitro immunogenicity assessment services that we offer in the UK, making it easier for North American customers to access our services.

For more information, visit: In Silico & In Vitro Services | Early Drug Development | Lonza

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Every month, MassBio spotlights a member company and the great work they’re doing to advance the life sciences industry and support the patients we serve. In February, we spoke with Paul Bolno, President and CEO of Wave Life Sciences since 2013. During his tenure, Wave has grown from a team of five into a fully integrated clinical-stage genetic medicines company, with a broad pipeline of preclinical and clinical programs supported by scalable, in-house manufacturing capabilities. Prior to joining Wave, he was Vice President, Worldwide Business Development with positions as Head of Asia BD and Investments and Head of Global Neuroscience BD, as well as Head of Oncology BD at GSK. Prior to his time at GSK he was Director of Research at Two River Group. Dr. Bolno earned a medical degree from MCP-Hahnemann School of Medicine and an MBA from Drexel University and was a general surgery resident and cardiothoracic surgery postdoctoral research fellow at Drexel University College of Medicine.

Tell us about your organization, its mission, and current initiatives 

At Wave Life Sciences, we’re driven by a resolute sense of urgency and are on a mission to bring life-changing medicines to people affected by devastating genetic diseases. We’ve built a unique oligonucleotide platform (PRISM^TM) that combines novel chemistry with the means to optimally address disease biology through multiple RNA-targeting modalities – editing, splicing, and silencing (including siRNA and antisense). In 2022, we delivered on the initial promise of our platform with the first data showing target engagement across all three of our clinical programs: WVE-N531 for Duchenne muscular dystrophy, WVE-003 for Huntington’s disease and WVE-004 for amyotrophic lateral sclerosis and frontotemporal dementia. Simultaneously, we continued to advance our leading RNA editing capability and named our first clinical candidate: WVE-006 for alpha-1 antitrypsin deficiency. Finally, we recently announced a strategic collaboration with GSK to advance transformative oligonucleotide therapeutics, which will bring multiple new programs aimed at novel, genetically-validated targets into our pipeline over the next four years.

How will your organization’s activities help patients now and into the future? 

Since the beginning of Wave we have been patient focused, having strong engagement with the patients and families we serve. Our pipeline addresses devastating diseases where scientific progress has been extremely limited. We have also pioneered innovative clinical trial designs to incorporate the feedback of patients and their families and reduce the burden on those who are already bravely enduring the challenges associated with these diseases. For example, our WVE-003 and WVE-004 studies have adaptive designs to rapidly optimize dose level and frequency based on early indicators of target engagement, and Wave was also among the first companies selected for FDA’s Complex Innovative Trial Designs pilot program in 2019.

While our current clinical pipeline includes candidates for monogenic disorders, we are particularly excited about our growing capability to leverage RNA editing for upregulation and modification of protein-protein interactions. This approach has the potential to reach significantly larger populations, such as those with cardiometabolic, liver and renal diseases.

What do you see as the biggest challenge facing the life sciences industry today? 

As an industry we have made incredible strides with understanding the relationship between certain genes and diseases, yet those insights have not meaningfully increased the universe of targets that are considered “druggable.” For this reason, there is significant overlap among RNA and DNA therapeutics companies on the genetic targets they are working on. Our belief is that combining novel nucleic acid chemistry with novel targets is crucial for realizing the full potential of genetic medicines.

This is the precise reason behind our recently announced collaboration with GSK. To continue to build a pipeline of transformational oligonucleotide therapeutics and gain access to new areas of disease biology, we recognized the need to partner with a company that has significantly invested in genetics and genomics to validate novel targets. Likewise, GSK recognized a need for a complementary discovery capability to translate these insights into medicines. This collaboration has potential to deliver a substantial number of first-of-their-kind therapeutics to patients and families.

What’s next for your organization / what are you focused on in the coming year? 

In 2023, we’re focusing on advancing WVE-003, WVE-004 and WVE-N531 to additional clinical data milestones. These data will include relevant biomarkers and safety/tolerability to inform next steps for each program. We also expect to bring the first-ever RNA editing therapeutic into the clinic with WVE-006. WVE-006 represents a novel treatment approach for AATD and is designed to correct the single point mutation on the SERPINA1 Z RNA transcript, thereby addressing both lung and liver manifestations of the disease. The WVE-006 first-in-patient study would both validate WVE-006 as potential best-in-class approach for AATD, as well as the viability of RNA editing as a new therapeutic modality. We will continue our preclinical research on RNA editing for upregulation and modulating protein-protein interactions, and expect 2023 to be a year where we share more on potential therapeutic applications. Finally, we’re excited to progress our GSK collaboration leveraging novel targets and our PRISM platform.

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