Market Analysis

Marketing Strategies

At the core of our company, we believe that trust between us and the patient is crucial to providing the best quality of care possible. Our company believes that trust begins with a conversation between us and the patient. FSHD is a rare and life-altering disease; for such diseases, trust is necessary for novel therapeutics to be accepted and utilized. Therefore, we will not waste money buying billboards and making advertisements with paid actors; but instead, we will convene with thousands of patients and physicians and hundreds of organizations across the United States. Our plan is to demonstrate the effectiveness of our therapeutic to those that we convene with and have them advocate for its use on our behalf. We are already working with the FSHD Society. We’ve already given talks about our treatment to the FSHD Society and have built a history of trust with them. We will extend this trust to hospitals and care systems.

We have experience teaching synthetic biology to hundreds of people at a time. This will directly apply when we teach patients about our treatment, how it works, and how it would affect them. Because of this, we will be good at educating patients and therefore selling our therapeutic.

Separately, we plan on partnering with celebrities with FSHD to educate patients on our therapeutic. One of these celebrities is Chip Wilson, founder of Lululemon. Chip Wilson is already actively involved in funding FSHD research and advocating for the patients with the disease. Our team intends to treat celebrities such as Chip Wilson so that we can gain their trust and use their voice. Influential figures such as these will give us the ability to reach many patients that don’t see health care professionals often.

Our less formal methods of advertisement will include a social media campaign. As an iGEM team, we have had experience running a team Instagram account and have almost doubled the account following in a few months after adopting a stale account without much engagement. We are confident that we can replicate our social media success, and if given more than a few months, we will gain engagement from those who will benefit from our treatment.

Through the aforementioned methods, we plan on encapsulating the entirety of the FSHD market, both those who are actively seeking treatment and those who are separated from FSHD society as a whole.

Patenting

We hope to patent our final construct within the US. We are considering filing a preliminary patent. This is much easier than a full patient as it would require minimal data to back up our findings and does not require a lawyer. However, because our construct includes motifs from other researchers and they have patented their parts, pursuing this therapeutic will require us to obtain licenses for these constructs. Specifically, DUX-s and KRAB both have been patented for the use in FSHD therapy. Thus, we are still in discussion with patenting lawyers and Stanford’s Office of Technology Licensing about how we should proceed with this process.

Logistics

The costs associated with packaging and labeling for clinical trials typically range between $40,000 and $100,000, depending on the specifics of the trial. For a detailed breakdown of these costs, refer to sources such as Sofpromed and STTark.

Storage

Stem Cell Storage

Annual Storage Fees

Long-Term Storage Plans: Providers may offer discounted rates for long-term storage, ranging from $1,000 to $3,000 for 20 years. Prices can vary based on provider and location. It’s advisable to consult with stem cell storage companies, such as STEMS Health and Cells4Life, for more detailed and specific pricing information.

Sources:

• Parents Guide Cord Blood
• STEMS Health Regenerative Medicine
• Cells4Life
• Fin Models Lab

AAV (Adeno-Associated Virus) Gene Therapy

Manufacturing Costs: Manufacturing AAV gene therapy can vary significantly based on production scale. For instance:

• A 50-liter production batch may cost approximately $1.5 million.
• A 200-liter batch may cost about $3 million.
• A 2,000-liter batch can reach up to $7 million.

Cost per Dose: The cost per dose of AAV therapy can also fluctuate, with larger-scale production bringing costs below $10,000 per dose, while smaller batches or high-dose applications will incur higher costs.

Additional Costs: Additional costs include the storage of raw materials, process development, quality control, and regulatory compliance—all necessary to ensure the safety, efficacy, and consistency of the therapy.

Sources:

• Pharmaceutical Technology
• DeSci Applied Research
• Cell and Gene
• Genetic Engineering & Biotechnology News
• Thermo Fisher Scientific

Stakeholder Analysis

Facioscapulohumeral muscular dystrophy (FSHD) affects about 1 in 8,000 people. This makes about 870,000 individuals with FSHD worldwide. These individuals do not have access to effective FSHD treatment and mitigate symptoms of the disease by doing physical therapy and resting. Physicians, hospitals, and healthcare conglomerates such as Pfizer and Moderna lose out on business because they lack the ability to treat such a disease. Both the patients and the healthcare providers are our stakeholders. Both of these groups greatly benefit from the creation of our therapy.

Unfortunately, there are no effective treatments that are available for FSHD patients. This means that there is no competition in this field; convincing our stakeholders to use our therapy will not pose many challenges because our therapy may very well be the only option.

We are very open to collaboration, and would collaborate with our larger stakeholders such as Pfizer and Moderna. Our collaboration with these companies would result in mass production of our therapy. This collaboration alongside potential collaboration with hospitals and healthcare providers would aid us in getting our mass produced therapy into the bodies of FSHD patients. We would like to work with these companies to ensure that our therapy can be of use to as many people as possible.

TAM (Total Addressable Market): Our therapeutic solution, Dux, will deliver the first effective treatment for patients with Facioscapulohumeral Muscular Dystrophy (FSHD). By strategically selecting targets, our therapy is designed to be effective for both FSHD1 and FSHD2, thus addressing the entire FSHD patient population. With an incidence rate of 1 in 8,000 individuals, this equates to a global market of approximately one million people.

SAM (Serviceable Available Market): The SAM represents patients in developed regions who can afford the therapy. Our SAM is estimated to be focused primarily on patients in North America, Europe, and select parts of Asia. Given the prevalence of FSHD in these regions and the economic factors, we estimate that this market could encompass around 300,000 patients globally.

SOM (Serviceable Obtainable Market): The serviceable obtainable market accounts for the patients we can realistically reach and who are most likely to adopt our therapy. This includes patients within our target regions who are not only able to access the treatment but are also likely to undergo the therapy based on factors such as physician recommendations, awareness, and affordability. We project this market to comprise approximately 100,000 patients in the early stages of commercialization, with the potential for expansion as awareness and distribution efforts grow.