Changing the Lives of Patients with

Myotubular and Centronuclear Myopathies (CNM)

Careers & Culture

Patients are at the core of what we do. We believe all people deserve safe and effective treatments that can improve the quality and duration of their lives. To achieve this, we continue to strengthen and grow our relationships with patient advocacy groups, key opinion leaders and practitioners, to establish Dynacure as a patient-focused leader in the treatment of rare neuromuscular disorders like CNM.

We’re looking for passionate, dedicated and fun people to help us achieve our vision and ambitious goals. We are a global company who fosters collaboration between our employees, industry partners, patient advocacy groups and clinicians as we develop, and ultimately commercialize, life-saving antisense medicines.

Join us on our mission!

Current Dynacure Opportunities

Meet Some of our Inspiring Women Leaders

Lori McKenna Gorski

Head of Corporate Communications & Patient Advocacy

I live in the Boston area and have been working in biotechnology and life sciences for 25 years. I was introduced to rare diseases in my role at Genzyme (now Sanofi Genzyme), a pioneer in rare disease drug development, and where I had global leadership roles in both patient advocacy and corporate communications. Since then, I have had the privilege of working with so many wonderful advocates from several dozen rare disease communities around the world. I also serve on the board of an organization called Our Odyssey, which supports young adults impacted by rare and chronic conditions.

I have had the chance to work in many different roles at Dynacure – which is not uncommon at smaller companies that are growing quickly. Dynacure is focused on developing treatments for neuromuscular diseases, beginning with myotubular and centronuclear myopathies (MTM-CNM).  I am responsible for engaging with the patient community and making sure their perspectives, insights and experiences as patients and caregivers are infused within our work at Dynacure.  This covers a wide range of perspectives from how we understand and talk about the disease to how we design our clinical studies. I also oversee corporate communications at Dynacure and am very much focused on raising awareness of the urgent needs of the MTM-CNM community.

I have a very special job and I sincerely appreciate what I get to do. Many of the rare patients and families I have met have faced tremendous obstacles in their lives. The MTM-CNM community is certainly no exception. Still, these brave and dedicated families take the time to share their insights about many important aspects of MTM-CNM including their experiences as patients and caregivers and their hopes for the future. Their courage, honesty and resilience inspire me – and everyone else at Dynacure.  I remember and respect each one of these encounters, and I take seriously my role to represent their voice.

People remember how you make them feel. Take the time to develop your interpersonal skills so you can best communicate, learn, and connect with others. It is important to make sure everyone you engage with feels respected, valued, and heard.

I have learned that it’s important to have mentors in your life who recognize and help you nurture the special skills that you bring to the world. If you truly listen to the advice and feedback from people who believe in you, it can be life changing. And whenever you have the opportunity, do your best to mentor and champion others as well.

I have two smart and funny teenage boys who keep me on my toes, and an overgrown Golden Retriever who brightens my every day. I also enjoy running, the outdoors and gardening. My goal next year is to grow sunflowers to help raise awareness for MTM-CNM. You can learn more about this special initiative here.

Anne Robé

Senior Associate Scientist

I studied life sciences and have a Master’s degree in Pre-clinical Drug Development (BAC+5 in France) in 2006 with a one year internship in the field of breast cancer.  I then worked 9 years in a well-recognized research institute in Strasbourg, East of France, in the field of the central nervous system (retina and brain). I joined Dynacure in March 2017. It was my first biotech experience and it was a chance to join the company at the beginning, when it was a start-up. I have learned a lot watching it grow from the inside.

I’m a Senior Associate Scientist. I work in the research team, so I spend a lot of time at the bench doing preclinical experiments and a lot of computer work for preparation and analysis of experiments. We are a small team, and we need to be multitasking. I’m in charge of the logistical part of the lab, including when we originally set the lab up years ago.

I can’t wait to see the first results of our first clinical trial!

Think outside of the box of the societal stereotypes, live your life as it works for you, follow your instinct. Find something you are really interested in, it will be so much easier to do a good job.

It is important to be humble, everyone has something to learn. Empathy is important also, put yourself in other’s shoes before reacting. When a problem comes, another point of view may be the key, and use it as a good opportunity to adapt yourself to be more effective.

Well, I just had a baby, so, honestly, not much for the moment! But I really enjoy going to the movies, practicing yoga and swimming, and just relaxing reading with my older daughter.

Caroline Dorrepaal, MD,PhD, PharmMed

Medical Director for Dynacure

In 1985 I started studying Medicine in Leiden (Netherlands), to become a paediatrician. During my second academic year, I became interested in research and performed various Paediatric clinical research studies, in parallel to my study of Medicine, resulting in a PhD thesis in the field of Paediatric Neonatal Neurology.

After obtaining both clinical and research experience, I discovered that my main passion was driven by performing clinical research, to directly contribute to the development of new treatments for many patients. In December 2020 I joined Dynacure. I was attracted by this organization in particular because of the highly qualified and motivated clinical research team, as well as the patient-oriented approach. At Dynacure, the patient comes first.

I am responsible for the design and execution of the clinical studies for our lead program DYN-101. My role is to ensure the selection of the most appropriate patient population for the studies and determine the optimal endpoints to show the efficacy and safety of the drug. In addition, I am responsible for the medical safety of the patients in the various studies.

Last year, we began to dose the first patients with a single dose in our Unite-CNM clinical study. I am very much looking forward to these results, as these will provide us with new insights into the potential efficacy of the drug.

Follow your heart and do what truly motivates and interests you. Don’t think about  problems to overcome, but rather about  solutions. There’s always a solution if you are creative. Stay curious, and never stop exploring and learning.

You learn the most from past mistakes. It is important to realize that there will always be many things you do not know. Do not hesitate to ask other for advice. Also, try to ensure that you maintain a clear overview of all the facts, benefits, and risks, allowing you to take well-informed decisions.

I enjoy spending time outside and perform various sportive activities (sailing, cycling on my racing bike, hiking, golf, aerobics and yoga). By being active you are able to “refresh” your brain!.

Dominique Nijkamp

Head of Clinical Development for Dynacure

I have more than 20 years of experience working in biotechnology, with a focus on Clinical Development and Quality, where I have been dedicated to helping to bring important therapies to patients with limited or no treatment options. In my role at Dynacure, I design clinical trials to demonstrate if a potential treatment can safely and effectively address the needs of a patient community, and I work to ensure that such studies are aligned to the feedback and insight we receive from patients, physicians, and regulatory authorities.

I oversee the design and execution of the clinical studies for our lead program DYN-101, an investigational antisense product candidate designed to reduce the expression of dynamin 2 protein (DNM2) for the treatment of Myotubular and Centronuclear Myopathies (CNM).

Last year, we began to dose the first patients through our Unite-CNM clinical study – a multicenter, ascending dose study Phase 1/2 clinical trial to evaluate DYN101. The trial is expected to enroll approximately 18 patients aged 16 years or older with X-linked myotubular myopathy (XLCNM) or autosomal dominant CNM (ADCNM). I am excited that as an organization we have entered this phase of development, with the potential to help patients who are desperately in need of treatment options. I am also excited about the potential of this investigational therapy to make a difference, and to learn from each heroic patient’s experience.

Always seek deeper understanding before searching for solutions.

In my work this means looking for the drivers behind what people say or do, asking why a regulation was created, or what the patients really need. Only with this level of understanding you can be truly creative and find solutions that ensure progress in your clinical program despite the inevitable challenges.  Seeking to understand first, will give you the opportunity to influence with impact in any area of your life.

Never hesitate to share your ideas with others. You may bring that much needed different perspective. And if not, take pride in your courage to be visible and engaged!

I enjoy spending time with my dogs as they light up my day with their joy for the outdoors.