Archive | Interviews RSS feed for this section

Get in the Groove and Tune Up Your Pitch at Two Groundbreaking RESI Events from Life Science Nation  

13 Nov

By Dennis Ford, Founder & CEO, Life Science Nation (LSN)

DF-News-09142022Life Science Nation (LSN) is proud to embark on our inaugural RESI London, taking place December 4-6, 2024, and the milestone 50th RESI JPM, scheduled for January 14-16, and 21, 2025, offer unparalleled opportunities for companies to jumpstart their fundraising efforts and shape their future in the industry. RESI London: A European Springboard, imagine stepping into the heart of the UK’s vibrant life science ecosystem at RESI London, co-located with Genesis 2024. This event isn’t just a conference; it’s a launchpad for companies looking to make their mark in Europe. Here, you’ll have the chance to refine your pitch for a discerning European audience, engage with elite investors and licensing partners, and showcase your innovation in the Innovator’s Pitch Challenge (IPC). The hybrid format, combining an in-person day with two days of virtual partnering, ensures you can maximize your reach and flexibility. RESI JPM: Where 2025 Global Partnering Roadshows Take Flight. As the calendar turns, RESI JPM in San Francisco beckons, offering a global stage amidst the energy of JPM Healthcare Conference week. This flagship event is your ticket to connecting with over 600 investors spanning the “4 Ds” – Drugs, Devices, Diagnostics, and Digital Health, as well as R&D Tools and Services. Picture yourself pitching to a panel of industry luminaries, forging connections that could define your company’s future, and kicking off the year with momentum that could carry you through 2025 and beyond.

The Art of Refinement

Remember the old joke about getting to Carnegie Hall? “Practice, practice, practice.” The same principle applies here. By participating in both RESI London and RESI JPM, you’re not just attending events; you’re embarking on a journey of refinement. Each interaction, each pitch, each conversation is an opportunity to hone your message, gain invaluable feedback, and build a global network that could be the key to your success.

LSN’s Global Partnering Ecosystem Is Your Secret Weapon

Behind these events lies LSN’s global partnering ecosystem. This isn’t just a network; it’s a sophisticated matchmaking system that spans the “4 Ds” and beyond, including R&D Tools, Services, and AI. With curated data profiles ensuring precise matches and a series of worldwide partnering events, LSN offers you a comprehensive toolkit for navigating the complex landscape of life science innovation and investment.

A Strategic One-Two Punch

Attending both RESI London and RESI JPM isn’t just about being present; it’s about strategic positioning. Use RESI London as your European warm-up, refining your approach for a continental audience. Then, take those lessons learned and amplify your impact at RESI JPM, where you’ll be primed to make a lasting impression on the global stage. This continuous engagement across two major markets isn’t just networking; it’s momentum-building at its finest. The time to act is now. By securing your place at both RESI London and RESI JPM, you’re not just registering for events; you’re investing in your company’s future. With LSN’s tailored support guiding you every step of the way, you’ll be equipped to navigate the challenges of the life science landscape and transform your innovative ideas into reality. Don’t let this opportunity pass you by – your next big breakthrough could be just one connection away.

Register for  RESI London Register for RESI JPM

RESI-London-2024-1100px

RESI-JPM-2025-1100PX

Amplified Sciences, Winner of the RESI Boston Pitch Challenge

5 Nov

Interview with Diana Caldwell, CEO of Amplified Sciences By Caitlin Dolegowski, Marketing Manager, LSN

DianaDiana Caldwell CaitiCaitlin Dolegowski

In this interview, we sit down with Diana Caldwell, CEO of Amplified Sciences and the first-place winner of the Innovator’s Pitch Challenge (IPC) at RESI Boston. Amplified Sciences stood out with their innovative approach, impressing both judges and attendees. Today, Diana shares insights into her company’s journey, their groundbreaking technology, and the experience of pitching at RESI. Join us as we dive into the story behind Amplified Sciences and what this win means for their future.

Interested in showcasing your innovation? Applications for the Innovator’s Pitch Challenge at RESI London and RESI JPM are open until this Friday, November 8th. Don’t miss the opportunity to pitch in front of a dedicated panel of investors, gain valuable feedback, and make lasting connections. Apply now to take your innovation to the next level.

Apply Pitch at RESI London Apply Pitch at RESI JPM

RESI-London-2024-1100px

RESI-SF-2025-Banner-1100px

How to Succeed in Company Presentations/Pitches at Partnering Conferences: The Drive to Win, and What It Takes

22 Oct

Interview with Dennis Ford, Founder & CEO of Life Science Nation (LSN) By Claire Jeong, CCO, VP of Investor Research, Asia BD, LSN

DF-News-09142022Dennis Ford Claire-newClaire Jeong

In the high-stakes world of fundraising, competition is fierce, and success demands more than just a good idea—it requires strategy, persistence, and grit. At Life Science Nation (LSN), participant feedback helps us fine-tune our events to serve their needs better. Recently, concerns were raised about the fairness of the Innovator’s Pitch Challenge (IPC) at RESI Boston. In this forum, companies pitch to a panel of investor judges and audience, with some alleging that participants may have “gamed the system.” These observations point to a larger conversation about business dynamics—where determination often trumps traditional notions of fairness. In this candid interview, LSN’s CEO, Dennis Ford, explores the fine line between integrity, innovation, and the entrepreneurial hustle that drives results in a competitive environment.

Claire Jeong (CJ): What differentiates CEOs who succeed in fundraising from those who struggle, particularly in the context of RESI and the Innovator’s Pitch Challenge?
Dennis Ford (DF): The RESI IPC is a microcosm of the real-world fundraising landscape. Some CEOs adopt a proactive approach, while others wait for investors or licensing partners to approach them. The passive ones tend to wait for things to happen to them, while the proactive CEOs are out there making things happen. Just like in the real world, the ones who seize every opportunity are the ones who tend to succeed.

CJ: What mindset shift do you believe is critical for startup CEOs transitioning from academia to a competitive fundraising environment?
DF: Many startup CEOs, particularly those coming from academia, think that paying an entry fee, being selected to pitch, and setting up a poster is enough. They expect investors to naturally flock to them. However, that’s only a minimal effort. To truly excel at a partnering event like RESI, you need to actively seek out opportunities—engage with everyone who walks by, identify investors, and make sure they know who you are. This transition mirrors the shift from academia to the business world, where you’re no longer a scientist—you’re a salesperson, marketer, and business developer.

CJ: How important is balancing compelling science with a determined business development strategy in the fundraising process?
DF: Striking the right balance is essential. In fact, we’ve seen some of the more aggressive participants in the IPC go as far as asking people for their RESI dollars, getting colleagues to donate theirs, and working the system to gain attention. This is the point of the IPC – getting CEOs to put themselves out there and speak to as many attendees as possible. It gives companies many opportunities to practice their pitch, network, and push CEOs to tell as many people as possible their story. While it might not seem “fair” to some, this aggressive approach mirrors the cutthroat nature of fundraising. CEOs need to understand that they’re no longer just researchers—they’re operating in a startup environment where they need to fight for every dollar, every collaboration, and every opportunity.

CJ: Can you share examples of how startup CEOs who took a more aggressive approach achieved success at RESI or in their broader fundraising efforts?
DF: The CEOs who reframe their mindset and adopt a more aggressive approach are often the ones who succeed. The combination of preparation, hustle, and compelling science gives them a real chance. If a CEO lacks either preparation or hustle, the likelihood of failure increases. That’s why the failure rate in life science startups is so high. The process of translating science into a viable product is hard, and having a team willing to fully commit is even harder. But when you have that combination, you’re much more likely to succeed.

CJ: What advice would you give to CEOs who are hesitant to take an aggressive approach when engaging with investors at events like RESI?
DF: The hesitation is understandable, but this isn’t an environment where waiting around will get you anywhere. If you want to succeed, you have to go after it relentlessly. Yes, the market can be frustrating, and investors can be difficult, but those who adapt and push beyond their comfort zones are the ones who will secure funding. CEOs should view it as a way to practice their elevator pitch and refine their story – speaking to 100 people in the quest to gain RESI cash will help companies refine their pitch a lot more than if they stick to only speaking to people with whom they have scheduled meetings.

CJ: How does participation in the RESI IPC competition reflect the real-world challenges life science startups face in fundraising?
DF: The RESI IPC mirrors the fundraising marketplace perfectly. We’ve run about 50 of these competitions over the last twelve years, and the pattern is always the same—some participants hustle hard, others take a more passive approach, and many learn and improve as they go. What’s interesting is that the startups who win IPC prizes—based on votes from both RESI dollars and judges—tend to be the ones who also succeed outside of the competition. They’ve become guerrilla marketers and street fighters, combining that tenacity with a solid product and milestones. Success in the technology and innovation space has always required that mix of drive and substance, and it always will.

The RESI Innovator’s Pitch Challenge is not just a competition; it’s a testing ground for the broader fundraising landscape. It rewards those who balance science with the hustle, pushing past the perceived limits of fairness to carve out their path in the startup ecosystem. The lesson? If you want to succeed, you must be willing to fight for it—aggressively, intelligently, and relentlessly.

Apply Pitch at RESI London Apply Pitch at RESI JPM

RESI-London-2024-1100px

RESI-SF-2025-Banner-1100px

Conversation with John Qiao, Founder & CEO of RadioClash: Innovator’s Pitch Challenge Success at RESI Boston

22 Oct

Interview with John Qiao, Founder & CEO of RadioClash By Caitlin Dolegowski, Marketing Manager, LSN

John-QiaoJohn Qiao CaitiCaitlin Dolegowski

In this interview, we spoke with John Qiao, Founder and CEO of RadioClash, who secured third place in the Innovator’s Pitch Challenge at RESI Boston this past September. John shares insights into the company’s journey, their fundraising efforts, and their experience pitching to investors at the conference.

Apply Pitch at RESI London Apply Pitch at RESI JPM

RESI-London-2024-1100px

RESI-SF-2025-Banner-1100px

EYWA Biotech: Pioneering Psychedelic APIs for Mental Health Treatment

16 Oct

Interview with Victoria Paz, Co-founder and CEO of EYWA Biotech By Caitlin Dolegowski, Marketing Manager, LSN

Victoria-Paz
Victoria Paz
CaitiCaitlin Dolegowski

EYWA Biotech is leading the charge in developing psychedelic APIs through cutting-edge synthetic biology techniques. In this interview, Victoria Paz, a key figure at EYWA, shares insights into the company’s mission, their innovative approach to solving mental health treatment challenges, and their experience at the Innovator’s Pitch Challenge during RESI Boston. From their fundraising journey to the impact they envision for the healthcare industry, this conversation sheds light on how EYWA Biotech is pioneering solutions in the biotech space.

About the Company

Caitlin Dolegowski (CD): Can you give us a brief introduction to EYWA Biotech and the problem you’re aiming to solve?
Victoria Paz (VP): Eywa Biotech is focused on using synthetic biology and gene engineering to produce GMP-certified psychedelic APIs like psilocybin, DMT, 5-MeO-DMT, and bufotenin. We aim to solve the challenge of providing a reliable, high-quality, and sustainable supply chain for psychedelic compounds used in research and therapeutic applications. Our goal is to address the mental health crisis by ensuring consistent access to these innovative compounds for conditions such as treatment-resistant depression and PTSD.

CD: What inspired you to start EYWA Biotech, and what differentiates your technology from others in the field?
VP: The inspiration behind Eywa Biotech came from the urgent need for new solutions in mental health treatment. Current production methods for psychedelic compounds are often inefficient and environmentally unsustainable. Eywa’s approach stands out because we use synthetic biology to create scalable microorganisms that produce these compounds in fewer steps, cutting down production time and reducing environmental impact. This enables us to deliver high-purity APIs for research and clinical use at a lower cost.

CD: How do you envision your solution impacting the healthcare industry in the next few years?
VP: We believe that Eywa Biotech’s innovations will play a key role in transforming mental health treatment by providing a reliable supply of high-quality psychedelic compounds. Our technology has the potential to make therapeutic psychedelics more accessible to researchers and healthcare providers, accelerating the development of novel mental health treatments. By doing so, we hope to contribute to a broader acceptance of psychedelics in medicine and support the advancement of personalized treatments for conditions like depression and PTSD.

Fundraising Status

CD: Where are you currently in the fundraising process?
VP: We recently closed our pre-seed round, which has allowed us to build and scale our initial production capabilities. We are now preparing to launch our seed round by the end of next year to support further scaling, commercialization, and the development of new formulations and preclinical trials.

CD: What type of investors are you seeking, and what do you hope to achieve with this round of funding?
VP: We are looking for strategic investors who have a strong understanding of biotechnology, mental health, and pharmaceuticals. Ideally, these investors will bring not only capital but also expertise in scaling biomanufacturing and navigating regulatory landscapes. With this round, we aim to scale our production to an industrial level, expand into new markets, and advance our pipeline of formulations and APIs.

CD: How have potential investors responded to your pitch so far, and what has been the most frequent feedback?
VP: Feedback from investors has been positive, especially regarding our innovative approach to scaling psychedelic API production and our focus on sustainability. The most common feedback has been around ensuring that our go-to-market strategy is well-defined, especially in terms of navigating regulatory challenges and market entry in different regions.

Experience at RESI Boston

CD: How was your experience participating in the Innovator’s Pitch Challenge at RESI Boston?
VP: Participating in the Innovator’s Pitch Challenge at RESI Boston was an invaluable experience. It provided us with a platform to showcase Eywa Biotech’s vision to a diverse panel of investors and industry leaders, allowing us to refine our message and receive valuable feedback.

CD: What was the most valuable aspect of presenting to a dedicated panel of investors at the event?
VP: The most valuable aspect was the insightful questions and constructive feedback from the panel, which helped us better understand the investment priorities of various stakeholders in the biotech space. It also offered a great opportunity to build connections with potential investors who align with our long-term vision.

CD: How did the RESI Boston conference contribute to your overall fundraising journey and networking?
VP: The RESI Boston conference significantly boosted our fundraising journey by allowing us to engage directly with high-quality investors and potential partners. It expanded our network within the biotech and life sciences community, helping us identify potential strategic partners for future collaborations.

CD: Did you find the feedback from investors during the pitch challenge helpful, and if so, how?
VP: Absolutely, the feedback was extremely helpful. It highlighted the importance of clarifying our regulatory strategy and demonstrating market readiness. This has guided us in refining our pitch to better address investors’ concerns about market entry timelines and scalability.

CD: Would you recommend the Innovator’s Pitch Challenge to other early-stage biotech companies, and why?
VP: Yes, I would highly recommend it. The Innovator’s Pitch Challenge provides a unique opportunity to receive direct feedback from experienced investors, gain visibility in the industry, and connect with like-minded entrepreneurs. It’s an ideal platform for any early-stage biotech company looking to validate their business model and accelerate their growth.

RESI-London-2024-1100px

RESI-SF-2025-Banner-1100px

Talon Biomarkers’ Role in Precision Drug Development

19 Sep

Interview with Pratip Chattopadhyay, Founder and CEO of Talon Biomarkers By Caitlin Dolegowski, Marketing Manager, LSN

Pratip-Chattopadhyay
Pratip Chattopadhyay
CaitiCaitlin Dolegowski

In this interview, Pratip Chattopadhyay, Founder and CEO of Talon Biomarkers, shares the journey behind the company’s founding and its mission to improve cancer recurrence predictions through advanced immune cell characterization. As a sponsor at the upcoming RESI Boston, Pratip delves into Talon’s cutting-edge technologies, strategic collaborations, and what excites them about connecting with early-stage companies in the biotech space.

Caitlin Dolegowski (CD): Can you tell us about Talon Biomarkers’ founding story and mission?

Pratip Chattopadhyay (PC): Talon Biomarkers was born from my personal frustration with understanding cancer recurrence risks and a broader need in drug development for precise cell-type markers that predict therapeutic outcomes. My career has been dedicated to developing the technologies that accurately characterize immune cells, and Talon is where we connect these technical capabilities to drug discovery and informed patient treatment decision-making.

CD: What sets Talon Biomarkers apart from other biomarker discovery companies?

PC: At Talon, we believe that every patient sample and clinical trial holds valuable insights—there’s always something to learn, even when outcomes aren’t as expected. We leverage cutting-edge, high-parameter immune monitoring technologies to identify predictors of therapeutic efficacy and disease progression. While we operate as a CRO, what truly sets us apart is our deep scientific commitment. We collaborate closely with our clients’ translational teams, not just delivering data but helping to analyze and interpret it, ensuring every experiment reveals actionable insights.

CD: How do you collaborate with clients on experimental design and data interpretation?

PC: We’ve developed unique, automated approaches to accelerate experimental design and execution, ensuring that our work is robust and impactful. Our process starts with understanding the client’s needs through one-on-one consultations and tailoring our strategies to fit their budget and goals, delivering actionable insights.

CD: Can you discuss your strategic partnership with LASE Innovation and how it enhances your services?

PC: Our partnership with LASE Innovation showcases Talon’s tech development expertise. We help companies validate, demonstrate, and introduce new technologies to the scientific community. LASE, for instance, has an exciting technology that transforms static flow cytometry results into dynamic time-lapse insights on immune responses, pushing the boundaries of traditional immune monitoring.

CD: What types of early-stage companies are you most excited to connect with at RESI Boston?

PC: We’re eager to connect with companies developing drugs that affect the immune system or involve immune responses in disease settings. The immune system often serves as a sentinel for many diseases and drug reactions, and we’re excited to demonstrate how our tools can accelerate discoveries.

CD: What emerging trends do you see in immune monitoring and biomarker research?

PC: One major trend is the simultaneous measurement of multiple immune cell parameters, maximizing insights from valuable patient samples. Another is the advancement in complex data analysis. As thought leaders, we guide our clients toward the best biomarker discovery approaches that yield actionable insights.

CD: How can interested companies best engage with Talon Biomarkers during and after RESI Boston?

PC: We’d love to meet you at Table 20, and don’t miss our Wednesday afternoon workshop at 2PM on the third floor of the Westin, St George Room D! If you can’t attend RESI, you can schedule a one-on-one meeting through our website. We look forward to connecting!

RESI-Boston-2024-September-1100pxw

RESI-London-2024-1100px

RESI-SF-2025-Banner-1100px

Innovating for a Cure: Insights from MDA’s Leaders on the Future of DMD Research 

29 Aug

Interview with Scott Kozak, Vice President of Research & Development, with contributions from Dr. Angela Lek, Vice President of Research at Muscular Dystrophy Association (MDA) By Caitlin Dolegowski, Marketing Manager, LSN

scott-kozakScott Kozak dr.-angela-lekDr. Angela Lek CaitiCaitlin Dolegowski

I had the opportunity to interview Scott Kozak, Vice President of Research & Development, with contributions from Dr. Angela Lek, Vice President of Research, both at the Muscular Dystrophy Association (MDA), a title sponsor at upcoming RESI Boston. As leading figures in MDA’s mission to advance treatments and find cures for neuromuscular diseases, Scott and Angela bring a wealth of experience and a shared dedication to improving patient outcomes. In this interview, they discuss their professional backgrounds, the latest advancements in research, and offer valuable advice for early-stage companies in the field of Duchenne muscular dystrophy (DMD) research.

Caitlin Dolegowski (CD): Scott, can you share a bit about your background and what led you to your current role as Vice President of Research & Development at the Muscular Dystrophy Association?

MDA: Sure. I have been in the pharmaceutical industry for 35 years. Most of that time has been concentrated on licensing & business/corporate development. I have held senior positions in Big Pharma & Specialty Pharma before concentrating on the entrepreneurial route, co-founding 3 companies along with my own consulting company. As an Entrepreneur In Residence for Yale Ventures, The University of Connecticut Center for Entrepreneurial Innovation, and Connecticut Innovations, I have helped many start-ups with business plans and fundraising as well as helping university faculty & students start companies. My role at MDA was a perfect fit for the experience I have gained throughout my years in the industry. I support the Research areas from a business perspective, which covers strategy, negotiation and other partnering activities for MDA Grants, Venture Philanthropy, MOVR, our patient registry of Real-World Data (RWE), and KickStart, a program encompassing MDA in-licensing IP and partnering for the development of therapeutics for the first time.

CD: What advice would you give to early-stage companies looking to access grants for DMD research, and what resources or programs does MDA offer to support them in this process?

MDA: Advice: It is never too early to reach out to MDA’s research team to initiate conversations about your drug or device development plans. We can provide disease-specific insights, patient perspectives, guidance on trial design as well as help connect you with the right experts in the field. We also highly recommend engaging with TREAT-NMD’s Advisory Committee for Therapeutics (TACT) to receive a comprehensive review on your drug development plans by a multidisciplinary team of experts in the neuromuscular disease space: https://www.treat-nmd.org/what-we-do/tact/

Resources/programs: MDA Venture Philanthropy (MVP) awards early-stage companies with promising therapies in development to treat neuromuscular conditions: https://www.mda.org/science/mda-venture-philanthropy

CD: As we explore innovative approaches in DMD treatment, can you explain the significance of Myosana’s non-viral delivery platform and how it differs from current viral delivery methods?

MDA: Myosana’s non-viral platform differs from AAV-based delivery methods because the therapeutic transgene is not packaged in an existing biological delivery vehicle. Instead, the platform includes proprietary components to deliver DNA that is stable in circulation and can assist in movement from blood vessels to muscle by targeting the GLUT4 transporter that is enriched in skeletal and cardiac muscles. The GLUT4 transporter naturally undergoes trafficking from internal stores to the muscle surface and then back again. The Myosana platform is designed to hitch a ride on GLUT4, leading to internalization of the cargo. Once inside the cell, the DNA cargo is optimized for nuclear targeting and expression.

Myosana’s non-viral delivery platform to deliver full-length dystrophin is mutation agnostic and is anticipated to benefit the entire DMD patient population. In addition, the technology overcomes known hurdles associated with current viral delivery methods utilized in gene therapy such as adeno-associated viruses (AAV) by: 1) not limiting gene size; 2) potential for repeat dosing due to lack of immune response; 3) enriched cardiac and skeletal muscle targeting via the GLUT4 receptor; 4) a predicted decreased cost of goods and better scalability.

CD: What are the potential advantages of delivering the full-length dystrophin gene compared to current microdystrophin approaches?

MDA: Full-length gene delivery of dystrophin at sufficient doses is anticipated to restore full functionality of the dystrophin protein at the sarcolemma, thus achieving a better result than current microdystrophin gene replacement therapy developed for DMD. Microdystrophin gene constructs currently in clinical development are approximately a third the size of full-length dystrophin and can at best achieve a milder Becker Muscular Dystrophy phenotype.

CD: How might Myosana’s technology address some of the limitations of existing gene therapies for DMD, such as gene size restrictions and the inability to repeat doses?

MDA: Currently, patients who test positive for pre-existing AAV antibodies are currently not eligible to receive AAV-based therapies due to immune-related safety concerns. This also precludes patients from being able to receive AAV-based therapies more than once. Myosana’s non-immunogenic platform is not anticipated to exclude any patients based on pre-existing immune concerns. Importantly, current gene therapy technologies are not optimized for durability, hence the ability to re-dose presents a very attractive option for Myosana’s platform.

Myosana’s non-viral delivery platform can be adapted to deliver gene cargoes without the size limitation of AAV. This opens doors for gene replacement therapy across a range of NMDs with genes that exceed the size limit for AAV packaging (e.g. Titin, Nebulin, LAMA2, Dysferlin, RYR1). The lack of size limitation also makes fine-tuning regulation of the transgene expression theoretically feasible by allowing for the use of native promoters and or additional regulatory elements.

CD: Could you elaborate on the mutation-agnostic nature of this approach and its potential to benefit the entire DMD patient population?

MDA: Despite FDA approval for exon skipping drugs for Duchenne (eteplirsen, golodiresen, casimersen, vitolorsen), there is still significant unmet need for the DMD patient population. These specific drugs target only a subset of patients with mutations in hotspot exons of the DMD gene, excluding those with pathogenic mutations that do not fall in exons 45, 51, 53. The recently approved Elevidys gene replacement therapy delivers a truncated version of the dystrophin gene that aims to achieve a milder Becker-like phenotype in patients. The ability to deliver sufficient quantities of full-length dystrophin may be necessary for the maximum achievable benefit and should be helpful to all DMD/BMD patients regardless of their disease-causing mutation.

CD: How does MDA’s support for Myosana’s research align with the organization’s history of funding DMD research, dating back to the identification of the dystrophin gene in 1986?

MDA: MDA has a strong track record in funding DMD research, dating back to the 1980s when we funded Dr. Louis Kunkel who discovered the dystrophin gene as the disease-causing gene responsible for DMD. Over the years, MDA has funded a range of DMD projects ranging from basic science research to clinical trials. Importantly, MDA supported Dr. Jeffrey Chamberlain and his research into understanding the function of dystrophin and whose pivotal findings have enabled the identification of the most functionally important regions of dystrophin. This vital knowledge has enabled the generation of miniaturized dystrophin gene therapies pursued by several companies. MDA also funded Dr. Jerry Mendell who conducted early AAV-based gene therapy trials and pioneered systemic delivery for muscular dystrophies. We continue to support cutting edge therapy development, identifying new technologies that can fill an unmet need in DMD. Myosana was selected for investment via our MVP program committee because their platform represents an alternative option to viral-based gene therapies and potentially holds tremendous therapeutic benefit through the restoration of the full-length dystrophin gene.

CD: What are your expectations for the development of genetic treatment options for DMD patients in the coming years?

MDA: Our expectation for genetic treatment options is to ensure that every patient is eligible for treatment that can restore dystrophin to therapeutic levels that is sustained over many years, thus allowing patients to life longer and more independent lives.

RESI-Boston-2024-September-1100pxw

RESI-London-2024-1100px

RESI-SF-2025-Banner-1100px