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LaShell Robinson, Head of Global Feasibility and Trial Equity at Takeda, discusses strategies to address underrepresentation in phase III psoriasis trials. She also talks about Takeda's partnerships with community stakeholders like Inside Edge, Black Health Matters, and Hispanic Communication Global Network to help improve engagement, address operational barriers by incorporating multicultural imagery, and prioritize sites with diverse patient demographics. She also explains how they've enhanced investigator diversity through educational sessions and multilingual toolkits, and how Takeda aims to expand these efforts globally.

Podcasts

LaShell Robinson, Head of Global Feasibility and Trial Equity at Takeda, speaks about the company's strategies to engage patients in underrepresented populations in its phase III psoriasis trials. 

Addressing Disparities in Psoriasis Trials: Takeda's Strategies for Inclusivity in Clinical Research

AstraZeneca's Shubh Goel, head of immuno-oncology, gastrointestinal tumors, US oncology business unit, AstraZeneca, discusses:

Results of the NIAGARA trial in bladder cancer

The significance of the five-year overall survival data from the HIMALAYA trial, specifically the long-term efficacy of the STRIDE regimen for unresectable liver cancer

In this episode of the Pharmaceutical Executive podcast, Shubh Goel, head of immuno-oncology, gastrointestinal tumors, US oncology business unit, AstraZeneca, discusses the findings of the NIAGARA trial in bladder cancer and the significance of the five-year overall survival data from the HIMALAYA trial, particularly the long-term efficacy of the STRIDE regimen for unresectable liver cancer. 

Key Findings of the NIAGARA and HIMALAYA Trials

Fran Gregory, Vice President of Emerging Therapies at Cardinal Health, gives insight into:

Recent trends in the cell and gene therapy space

How CAR-T therapies have been gaining momentum and what kind of progress we can expect in the next 10 years

Personalized medicine and gene editing advances

The biggest hurdles within cell and gene therapy space and how do we begin to jump over them

The biggest foci for cell and gene therapy for the next 5 years

Fran Gregory, PharmD, MBA, is Vice President of Emerging Therapies at Cardinal Health, where she is responsible for Cell & Gene Therapies and Biosimilars. Fran’s background includes extensive clinical, HEOR, trade and commercial experience in the payer/PBM, specialty pharmacy, and biotech manufacturer spaces. Her career reflects a consistent focus on complex, innovative, high-value pharmaceuticals, and a passion for breaking down barriers to access and care. Throughout her career she has paved the way in innovation by starting two of the first and largest specialty pharmacies in the US, launching two of the first cell and gene therapies in the world, and bringing several first-to-market biosimilars to patients. As a pharmacist, she has an acute focus on patient outcomes, and is an expert in value-based care models, reimbursement and payment strategies, as well as navigating successful teams in healthcare to deliver solutions to bring advanced therapies and treatments to patients.

The editors of Pharmaceutical Executive bring you the latest commercial insights to master the science of success. Podcast episodes examine current trends, key conferences, and critical topics in the bio/pharmaceutical industry.

Fran Gregory, VP of Emerging Therapies, Cardinal Health discusses her career, how both CAR-T therapies and personalization have been gaining momentum and what kind of progress we expect to see from them, some of the biggest hurdles facing their section of the industry, the importance of patient advocacy and so much more. 

Cell and Gene Therapy Check-in 2024

A new study shows that varenicline combined with brief remote counseling significantly improves vaping abstinence in in patients between 16 and 25 years of age. 

Study Finds Varenicline Significantly Boosts Vaping Cessation Rates in Youth 

In the Phase III HARMONi-6 trial, ivonescimab plus chemotherapy significantly outperformed tislelizumab plus chemotherapy as a first-line treatment for advanced squamous non-small cell lung cancer.

Akeso’s PD-1/VEGF Bispecific Antibody Demonstrates Meaningful Progression-Free Survival Benefits in Advanced Squamous NSCLC

Results from the dual Phase III TRIUMpH trials demonstrated the statistical superiority of tegoprazan over lansoprazole in patients with erosive esophagitis and non-erosive reflux disease. 

Sebela’s Novel Potassium-Competitive Acid Blocker Demonstrates Promise in Gastroesophageal Reflux Disease

In the Phase III ARISE trial, Cobenfy administered as an adjunctive treatment to atypical antipsychotics for patients with inadequately controlled schizophrenia did not achieve statistically significant improvements. 

Bristol Myers Squibb’s Cobenfy Falls Short in Phase III Trial as Add On Therapy for Schizophrenia 

Results from the Phase IIb SunRISe-1 study demonstrated a one-year duration of response with the TAR-200 intravesical gemcitabine-releasing system in patients with Bacillus Calmette-Guérin-unresponsive high-risk non-muscle-invasive bladder cancer. 

Johnson & Johnson’s Novel Intravesical Gemcitabine Shows Durable Response in BCG-Unresponsive Bladder Cancer

Rare Disease Therapies Drive Shift in Drug Development Focus

, a session leader from “U.S. Pharmaceutical Transitions: What’s Next?” shared key insights on the current and future state of the market. The conversation highlighted the rapid growth of specialty pharmaceuticals—now comprising over half of the total market—and examined critical industry developments ranging from biosimilar uptake and GLP-1 drug disruption to persistent challenges like retail pharmacy closures and drug shortages. With an eye toward long-term trends and underexplored risks, the discussion underscored how therapeutic priorities and commercial strategies are evolving in a complex and increasingly constrained healthcare landscape.

Pharmaceutical Executive: Today, you hosted the ‘U.S. Pharmaceutical Trends, Issues, and What’s Next’ session. Can you provide a brief synopsis of what the session entailed?

The session focused on where the pharmaceutical market stands today—what’s growing, what’s not, and several hot-button issues shaping the industry. Specialty pharmaceuticals are now outpacing traditional drugs and currently account for 52% of the market. A particularly noteworthy development is the emergence of a private-label biosimilar to Humira, which is already making an impact. Immunology remains a high-growth area, alongside oncology and HIV treatments.

We also covered key industry challenges and developments, including ongoing uncertainty around tariffs, widespread drug shortages, the wave of retail pharmacy closures, and recent product launches. These were the central themes discussed.

PE: You shared a lot of insights about total market performance—what surprised you most about how different therapeutic classes are performing right now?

One of the more striking trends is the continued shift toward treatments for rare diseases, including orphan drugs and cell and gene therapies—a movement that’s been building over the past two decades. While biosimilars are making progress in the biologics space, there remains a significant gap: out of 128 biologic molecules eligible for biosimilar development, work is currently only underway on 12. That discrepancy is something to watch closely, as it may have a major impact on future access and affordability.

The “U.S. Pharmaceutical Transitions: What’s Next?” sessionoffered a timely look at current market dynamics, with a focus on growth areas, emerging challenges, and broader industry shifts. Specialty pharmaceuticals now make up 52% of the total market, with strong momentum in immunology, oncology, and HIV treatments. Notably, the introduction of a private-label biosimilar to Humira is making a measurable impact.

Discussion also touched on macro-level issues such as drug shortages, retail pharmacy closures, and new product launches. Tariff uncertainties and economic pressures are contributing to instability, particularly in retail pharmacy, where closures are occurring at a rate of approximately four per day. Pharmacies are struggling with staffing shortages and expanded responsibilities, such as administering immunizations, with no near-term policy solutions in sight.

The session also explored the expanding footprint of GLP-1 drugs, which have disrupted both therapeutic and commercial landscapes. Retail pharmacies often lose money dispensing them, prompting a shift to 30-day fills and increasing reliance on manufacturer-direct programs like LillyDirect and novoCare. Although GLP-1s are being explored for additional indications such as sleep apnea and cardiovascular conditions, broader coverage remains limited, especially under Medicare and Medicaid.

In terms of long-term trends, panelists noted the increasing focus on rare diseases, orphan drugs, and cell and gene therapies—high-cost treatments for smaller patient populations. While biosimilars are gaining ground in biologics, development is lagging; out of 128 eligible biologic molecules, only 12 have active biosimilar programs.

An underappreciated trend is the role of pharmaceutical rebates in subsidizing health insurance premiums. A decline in rebates could lead to higher premiums, potentially reshaping payer dynamics. As the market evolves, industry leaders must navigate pricing pressure, access challenges, and shifting therapeutic priorities.

Videos

Doug Long, VP, industry relations, IQVIA, examines shifting pharmaceutical trends, including specialty growth, biosimilar gaps, reimbursement pressure from GLP-1s, and persistent access challenges.

Video Series

Latest Conference Coverage

Asembia 2025: Rare Disease Therapies Drive Shift in Drug Development Focus

LaShell Robinson, head of global feasibility and trial equity at Takeda, discusses how Takeda will ensure that diversity and inclusion remain a priority in its clinical trials moving forward.

Long-Term Sustainability for Diverse Trial Initiatives

 video interview, LaShell Robinson, head of global feasibility and trial equity at Takeda, discusses strategies to address underrepresentation in clinical trials, particularly in phase III psoriasis trials. Robinson also discusses the company's partnerships with community stakeholders like Inside Edge, Black Health Matters, and Hispanic Communication Global Network to improve engagement, address operational barriers, and prioritize sites with diverse patient demographics. Takeda has also enhanced investigator diversity through multilingual toolkits and educational sessions, and aims to expand these efforts globally by adapting strategies to regional needs.

Pharmaceutical Executive: What are the long-term sustainability plans for these initiatives? How will Takeda ensure that diversity and inclusion remain a priority in its clinical trials moving forward?


We talked a little bit about our US efforts, but we're also intending to expand globally and this trial is included with that. We're also making sure that, region by region, we're adapting the trial to what is best for that region and the perspective that each of those patient populations or communities may bring. We try to get to the community before they've thought of a clinical trial so that they're empowered with that information. Region by region, that may differ. I'm really looking forward to the learnings that we'll get as we're connecting in different areas of the world around clinical research, and how we can make it a reality to connect.

As we may see a change, we are committed to patients, or what I like to call, PTRB: patient, trust, reputation, business. That is our corporate philosophy and those are always our four priorities when we are looking at developing a treatment. When we think about the reflection of disease in our clinical trials and making sure that it accurately reflects the patient population, it really becomes about what's best for patients and making sure that we have the best scientific research to do that.

LaShell Robinson, head of global feasibility and trial equity at Takeda, identifies some of the key barriers in achieving representative clinical trials for psoriasis, and they are overcoming them.

Barriers in Achieving Representative Psoriasis Trials

Pharmaceutical Executive: What are some of the key barriers you've identified in achieving representative clinical trials, and how are you overcoming them?

For the Hispanic and Asian communities in particular, one of the largest aspects was the lack of translated materials in their native language. One of the gaps that we closed was providing translated materials, and we're hoping to really help overcome the barrier around health literacy. As a standard practice, since 2023, we automatically translate all of our clinical trial materials that are patient facing into Spanish. This is before a site requests it and it has helped close the gap and had a positive impact on connecting with the Hispanic community. Moving forward, that's the same strategy that we are looking to expand for other multilingual audiences.

Second, to better reach and engage with underserved populations, we have partnerships with Inside Edge, Black Health Matters, and Hispanic Global Communications Network. Since they have a lot of trust already built with the community, it made it very nice to partner with them to do some of these educational aspects. We also use that as a mechanism to record videos. For example, we heard from sites that they wanted another mechanism to talk about clinical research in a general aspect and a video would be nice. We worked with Inside Edge and one of our principal investigators that was on the trial to develop a video in English and Spanish that they could use with potential patients to just get them comfortable with research and share that information.

In the development of our patient facing materials, specifically for psoriasis, we knew imagery was one of the core issues, so we wanted to make sure that our patient materials also included multicultural visuals. That came into play largely when we started seeing that our Asian population was lagging behind in the execution of the trial. We had already had this discussion and proactive planning, so we were able to use the imagery to enhance our social media and digital outreach, and connect with our sites that we knew were in closer proximity to the Asian patient communities to start addressing that lagging behind in real time. That turned out well, because we were able to meet that particular trial metric.

All of these different aspects, when we're talking about challenges and barriers, become very important to be able to have that proactive plan. You can address these issues in real time and look at them from a holistic standpoint, instead of waiting till the end of the trial, or waiting until the trial is already in flight, and then trying to fit a solution on the fly.

LaShell Robinson, head of global feasibility and trial equity at Takeda, identifies some of the key barriers in achieving representative clinical trials for psoriasis, and how they are overcoming them.

Barriers in Achieving Representative Psoriasis Clinical Trials

LaShell Robinson, Head of Global Feasibility and Trial Equity at Takeda, talks about how Takeda aims to expand their efforts globally, adapting strategies to regional needs.

Ensuring Representative Psoriasis Clinical Trials on a Global Scale

Pharmaceutical Executive: Beyond the US, how is Takeda addressing the need for representative clinical trials globally, considering the 60 million people worldwide living with psoriasis? Are there unique challenges in different regions?

The US definitely has very specific aspects around trial diversity, and that doesn't necessarily translate to every single region. Takeda  intends to expand those efforts by drawing on that US strategy framework, but making sure that it's adapted to what is most important per country or region, specifically when it comes to talking about the patient perspective. What does underrepresented or underserved mean in a particular community? That could vary in the US. It could be race, ethnicity, gender, or even location.

When we start looking at the other parts of the world, it could even be how they're accessing healthcare. Gender or sex tends to be a factor that consistently plays a role that we're seeing regardless of geography, so that will be one of our areas of key focus as we start to broaden this into other geographies. While we have this global problem, it's extremely important that we're taking the time to understand what it means from a patient perspective in that region, and that's how we'll start expanding globally.

PE: Can you provide examples of how Takeda is ensuring that the data collected from these more diverse trials is translated into improved real-world outcomes for patients?

The first part was ensuring that our trials were representative of real-world patient populations. It can become difficult to even talk about outcomes if we don't have that inclusion. We're looking at this as a way to serve the patients that we aim to treat so that we have better outcomes from patients. We've studied how different populations may respond to different therapies, but that's usually after the drug is approved. We wanted to make sure that we're taking that into account in the context of our trial.

One of the aspects that we're really looking forward to is Takeda is also working to share the epidemiology data that we've collected to better help and support further research and understanding in the area. Stay tuned for that, because directionally, that can really help different areas of understanding in this area. 

LaShell Robinson, head of global feasibility and trial equity at Takeda, talks about Takeda's aims to expand its efforts globally by adapting strategies to regional needs.

LaShell Robinson, Head of Global Feasibility and Trial Equity at Takeda, discusses strategies to address underrepresentation in clinical trials, particularly in phase III psoriasis trials.

Addressing Disproportionate Underrepresentation of Racial & Ethnic Groups in Psoriasis Trials

 video interview, LaShell Robinson, head of global feasibility and trial equity at Takeda, discusses strategies to address underrepresentation in clinical trials, particularly in phase III psoriasis trials. Robinson also discusses the company's partnerships with community stakeholders like Inside Edge, Black Health Matters, and Hispanic Communication Global Network to improve engagement, address operational barriers, and prioritize sites with diverse patient demographics. Takeda also enhanced investigator diversity through multilingual toolkits and educational sessions, and aims to expand these efforts globally by adapting strategies to regional needs.

Pharmaceutical Executive: What specific strategies is Takeda implementing to address the disproportionate underrepresentation of racial and ethnic groups in clinical trials, particularly in its Phase III psoriasis trials and beyond?

As the head for global feasibility and trial equity, my day-to-day job is focused on making sure that all of our trials have the representation of patients that reflect the real world, including these psoriasis trials. It goes without saying that our ambition from the very beginning was to ensure that our phase III trials for psoriasis had better reflection of the real-world patient population, because we did see a gap. When we were looking at the real-world data, we noticed that things like under diagnosis, longer treatment paradigms, or longer pathways to diagnosis also impacted the people that were represented in our clinical trials. We wanted to ensure that from the very beginning, we were really thinking about this and implementing our tested, tried and true PAVE strategy, which is our strategy to make sure that underrepresented and underserved populations are included in our trial.

First, our P is partnering with community stakeholders. We partnered with organizations such as Inside Edge, Black Health Matters, and Hispanic Communication Global Network to ensure that we have better engagement with communities that were underrepresented and underserved, specifically for psoriasis. These organizations have tried and true relationships, and we were able to leverage that to connect with patients in a more authentic manner, not only educating them about psoriasis, but also around the importance of them participating in clinical research and how that helps contribute to better science.

We have the A, which is addressing operational barriers that impede patient access. This is where we spend a lot of time around planning, operations, and looking at the protocol. One of the things that we did immediately was incorporate multicultural imagery. We know that most of the imagery for plaque psoriasis is of patients that are white. The impact that has is it creates a longer diagnosis pathway, we see gaps in the teaching around dermatology, etc. We wanted to make sure that we had multi-racial and multiethnic imagery with skin-of-color and multiple images represented so that we could start closing that gap. We also did careful site selection, where we prioritized sites that had a broader reach and patient representation in our feasibility process. We even went so far as to ask our sites to tell us about the demographics of their patients so we could prioritize the selection.

Then V is verifying that any goal we had around representation was truly reflective of the patient data. One of the things we saw at the very beginning is even though psoriasis is one of the most common skin disorders, there's a lack of epidemiology data that is specific to race, ethnicity, and gender breakdown. One of the other elements that we did in addition to looking at literature review and epidemiology was ask our sites what their real-world patient population is and used that to actually develop a goal that we felt was more reflective of the patients with psoriasis.

Then finally E was enhancing the diversity of our investigative sites. This is where we really deepen the education and sharing the best practices to support more accurate diagnosis of psoriasis in skin-of-color at the very beginning. We offered sites multilingual toolkits where they could actually discuss what the general sense of clinical research is, and how it transmits to our particular trial. We even had an investigator do a session at  an investigator meeting. This is where we pull all of our sites and those participating together to really educate them on the protocol, give them the opportunity to ask questions, and use the opportunity to share best practices around diagnosing skin-of-color. In fact, we had two sessions that were dedicated to that, so that at the end of the day our sites were very well prepared. We're also thinking a bit differently around how they could connect patients that are traditionally left out of research.

LaShell Robinson, head of global feasibility and trial equity at Takeda, discusses strategies to address underrepresentation in clinical trials, particularly in phase III psoriasis trials.

Addressing Underrepresentation of Racial & Ethnic Groups in Psoriasis Trials

Jennifer Kyle, CEO and founder of Condor, discusses the inspiration behind creating a financial cloud platform specifically for biopharma R&D. 

Inspiration and Problem Solving: Condor's Core Functionalities

video interview, Jennifer Kyle, CEO and founder of Condor, discusses the inspiration behind creating a financial cloud platform for biopharma R&D. During the beginning of her career in auditing and finance, she repeatedly encountered the same financial management challenges across various biotech companies. This led her to develop Condor, a platform that integrates clinical and financial systems, providing a unified system of record for accruals, vendor oversight, budgeting, and forecasting. Future plans include advanced AI and machine learning for benchmarking and deeper integrations with clinical systems.

Pharmaceutical Executive: What inspired you to create a financial cloud platform specifically for biopharma research and development? What problem were you aiming to solve?

 I would say I didn't go looking for this, it found me pretty organically. My background is in finance and accounting. I started my career at Ernst and Young auditing a lot of biotech companies and going through IPOs. Initially, I thought I wanted to be a CFO, so I left Ernst and Young and went into the industry to climb the corporate ladder, gain experience, and eventually get that corner office at the top. Along that journey, I realized that I didn't know what I wanted to do.

I was really good at solving problems at the intersection of operations, FP&A, and accounting. I loved making business processes more efficient and effective. I loved to come in, add value, and make things better—that was like my superpower. I saw a lot of companies that had all of these financial projects on  backlog, but didn't really have time in their day-to-day to clean things up. I love doing it and I'm good at it, so I decided to offer my services and help as many companies as I can. That's really where it started.

I got a phone call from a clinical stage, publicly traded biotech company saying they needed somebody to come in to help clean up their clinical accruals. They wanted to use the information to help with some financial planning. Additionally, they had to become 404b SOX compliant, so they needed to step up their internal controls and create a process around compliance. That turned into a comprehensive workbook that connected a lot of the clinical stakeholders and data to the financial and accounting processes, workflows, and compliance. That was such a success at this biotech company that I kept getting called to other biotech companies to go do the same thing.

Over and over, I realized it was the same problem. I wondered if there was any software out there to automate this, because surely there had to be something better than an Excel spreadsheet.

I talked to big pharma companies, small biotech companies, and what I realized was two things: the big organizations build a homegrown database, the small organizations use an Excel spreadsheet, and nobody understands the process. Not the auditors that audit it, not the clinical folks that are managing their contracts and vendors, and certainly not the finance people. I just decided at that point that there's a market for it, and If I didn't build it, somebody else would.

So, I said, ‘Why not? Let's do it.’ That's when my career completely shifted from going from accounting to finance, to business process consultant, to tech entrepreneur, and it's been a really fun ride. Every company is trying to solve the same challenges over and over, and I'm just stoked that we can offer the market some solution here.

Jennifer Kyle, CEO and founder of Condor, discusses the inspiration behind creating a financial cloud platform specifically for biopharma R&D.

Advancing Hearing Solutions

Last month, Regeneron announced updated results from the Phase I/II CHORD trial in children with otoferlin-related hearing loss. Out of 11 children who received DB-OTO, 10 showed notable hearing improvements at different decibel levels, with some achieving nearly normal or normal hearing thresholds. Pharmaceutical Executive spoke with Jonathon Witton, AuD, PhD, VP, auditory global program head, in more detail about the trial results, the performative potential of DB-OTO, and the company’s work with the hearing loss community.

How has Regeneron engaged with the hearing loss community, including patients and clinicians, to understand and optimize DB-OTO’s potential impact?

Since the beginning of this particular program, we have spent a lot of time talking with clinicians in the field. Obviously, I have been one of them. Our goal is to understand the biggest unmet needs for patients—where we can solve problems for them. We’ve also spent significant time engaging with advocacy groups, the patient community, and families during the early stages, as we were determining where to focus our technology development efforts. I think when you learn about what’s really important to families, the answers can be surprising.

For example, one thing that families talked a lot about was the importance of their children being able to hear 24/7, which is something we often take for granted. We continue to hear even while we sleep. In fact, we sometimes wake up because something alerts us. Our ears play a critical role in our safety. We’ve repeatedly heard from parents that when children are fitted with prostheses for their hearing loss, they often need to remove them for activities like sporting events, and this raises safety concerns for parents.

When I initially thought about these programs, I focused on developing technology to improve speech understanding, noise perception, and the ability to appreciate music. I was looking at all of this complex processing from a scientific perspective. But when I spoke with families, I found that what mattered most to them was ensuring their kids’ safety and being able to hear things like an approaching car. These are insights you gain through early conversations. A key part of our culture is making these personal connections.

Another point worth mentioning is that this is just the beginning for us. We’re starting with a program that impacts a smaller number of patients each year—fewer than 50 children are born annually with deafness caused by a mutation in this specific gene. But we’re not stopping there. We’re committed to continuing the development of these kinds of therapeutics, both for childhood deafness and adult-onset hearing loss.

Deafness can affect individuals early in life, and it also has a significant impact later in life, with noise-induced hearing loss and age-related hearing loss. This is another area we’re investing in, with the goal of developing treatments for these patients as well. I believe it’s a great opportunity to address pressing public health challenges, and we’re excited to lead the way.

 Regeneron envisions DB-OTO reshaping the standard of care for hearing loss by shifting from symptomatic management with prostheses, such as hearing aids and cochlear implants, to a molecularly targeted therapeutic approach. Historically, clinicians diagnosed hearing loss without understanding its genetic cause, but advances in genetic testing have revealed that over half of childhood hearing loss cases stem from genetic protein deficiencies. DB-OTO aims to correct this by delivering functional DNA to specific inner ear cells, enabling them to produce the missing protein, potentially restoring hearing to near-normal levels.

The transformative potential of DB-OTO is evident in early clinical trials, where 10 out of 11 patients demonstrated hearing improvements, with some achieving normal or near-normal sensitivity. This represents a paradigm shift—clinicians will now need to incorporate molecular diagnostics into routine care, ensuring that patients receive precise, targeted treatments rather than generalized symptom management.

Regeneron has secured multiple regulatory designations, including the FDA’s RMAT designation, which facilitates early and frequent discussions with regulators. These interactions are crucial in navigating the complexities of gene therapy development, accelerating timelines for approval and commercialization.

Challenges remain, particularly in clinical development and manufacturing. Ensuring scalable, high-quality production of gene therapies is as critical as generating robust clinical data. Regeneron has invested heavily in manufacturing capabilities to meet these demands.

Beyond clinical development, Regeneron actively engages with the hearing loss community, including advocacy groups, clinicians, and families. Insights from these discussions have highlighted practical concerns, such as ensuring continuous hearing for safety. This patient-centric approach shapes the development of DB-OTO and future gene therapies, with plans to expand treatments to broader populations, including adults with age-related and noise-induced hearing loss. Regeneron aims to pioneer gene therapy solutions for a currently untreatable public health challenge.

Jonathon Whitton, AuD, PhD, VP, auditory global program head, Regeneron, discusses how the focus of technology development is shifting toward ensuring safety and continuous hearing for children.

Understanding the Real Needs of Families: Advancing Hearing Solutions for Children and Adults

Artiva Biotherapeutics, CEO, Fred Aslan, MD, explains the strategic advantages of pursuing both a company-sponsored trial and an investigator-initiated trial simultaneously.

Dual Trial Strategy: Company-Sponsored and Investigator-Initiated

 video interview, Fred Aslan, MD, CEO of Artiva Biotherapeutics, discusses the company’s current trials for autoimmune indications in the US. The first is a company-sponsored trial focusing on lupus, while the second is an investigator-initiated trial exploring four conditions: lupus, rheumatoid arthritis, pemphigus vulgaris, and vasculitis. This second trial is uniquely being conducted in a community setting to assess the practicality of outpatient treatment.

Pharmaceutical Executive: What are the strategic advantages of pursuing both a company-sponsored trial and an investigator-initiated trial simultaneously, and how do they complement each other and differentiate AlloNK from autologous cell therapies or other existing treatments for these conditions?


In a company sponsored trial, you generally work with a CRO and open multiple different sites, sometimes in different countries. One of the advantages is that the data is often considered more robust because it’s not dependent on what is happening on one site. You can look across a variety of different sites with different patient populations in different countries. It's a powerful tool to give you more confidence on the signal that you're generating.

An investigator-initiated trial is very compelling because it can be a very efficient way to work with a site where there is already an investigator that's interested in generating data for you. One of the specific advantages of working in this investigator-initiated trial goes back to the notion that this is a community rheumatologist testing our drug. Today, a patient receiving auto CAR-T cannot get this done in a community setting and instead must go to a FACT-accredited center. There are approximately just 100 of these centers around the country. We have 1000s of different outpatient clinics where one can get treated today for an autoimmune disease.

Auto CAR-T needs to be done at these tertiary care centers because it is a more complicated procedure. They must perform apheresis on the patient and send them home while they manufacture the product. The patient must come back to be hospitalized. After they're treated and leave the hospital, they must hang around the center for at least 30 days in case they need to be rushed back.

A site like the one we're working with for this investigator-initiated trial is a community site. They would not have the capability to run an auto CAR-T trial. This specific trial gives us an opportunity to demonstrate that AlloNK can be used in an outpatient setting (assuming the trial goes as we expect). The physician at this site works independently as a strict rheumatology community practice and can treat his patients in his infusion chairs, send them home after they receive the therapy, and manage their outpatient therapy.

Part of our differentiation compared with autologous CAR-T cell is that we believe that our therapy can become accessible to a much larger population of autoimmune patients. The only way to get to that population is really to make this therapy available in community sites. The only way for community sites to be administering this drug is if it's something that they can handle as a site. Having a trial in a community site gives us a lot of insights as to the feasibility of getting this done with our product.

Artiva Biotherapeutics' CEO Fred Aslan, MD, explains the strategic advantages of pursuing both a company-sponsored trial and an investigator-initiated trial simultaneously. 

Fred Aslan, MD, CEO, Artiva Biotherapeutics, identifies data points Artiva is prioritizing to demonstrate that results from these trials will translate to similar efficacy across a range of autoimmune conditions.

Translating AlloNK Efficacy Across Autoimmune Diseases

 video interview, Fred Aslan, MD, CEO, Artiva Biotherapeutics, discusses the company’s current trials for autoimmune indications in the US. The first is a company-sponsored trial focusing on lupus, while the second is an investigator-initiated trial exploring four conditions: lupus, rheumatoid arthritis, pemphigus vulgaris, and vasculitis. This second trial is uniquely being conducted in a community setting to assess the practicality of outpatient treatment.

Pharmaceutical Executive: Given the different underlying disease mechanisms in autoimmune diseases, what specific data points are you prioritizing to demonstrate that results from these trials will translate to similar efficacy across a range of autoimmune conditions?


One of the things that iscompelling about this academic study isthat the investigator didn't just treat patients with lupus, he treated patients with lupus with-and-without lupus nephritis, along with patients with myositis or scleroderma. Since then, there's been other conditions that have also been investigated by this academic group. What’s remarkable about that initial readout is this notion that a deep B-cell depletion could actually be transformational in patients across multiple different conditions, which is something that was interesting and not seen before.

Now the challenge (and good news) for the industryis that there arethese indications that one can interrogate and pursue. The one commonality across all these diseases, which we believe is the mechanism of action of how these therapies are working, is the depletion ofthe B-cells. When these cells reconstitute, they do so with a naïve phenotype.

Artiva Biotherapeutics’ CEO Fred Aslan, MD, identifies data points the company is prioritizing to demonstrate that results from certain trials will translate to similar efficacy across a range of autoimmune conditions.

R&D/Clinical Trials

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