4D Molecular Therapeutics | research notes

4D Molecular Therapeutics: Unlocking the Power of RNA Interference for Therapeutic Innovation

Introduction

4D Molecular Therapeutics (4DMT) is a clinical-stage biopharmaceutical company dedicated to harnessing the transformative potential of RNA interference (RNAi) for the treatment of serious diseases. RNAi is a fundamental biological mechanism that allows organisms to regulate gene expression by targeting and degrading specific messenger RNA (mRNA) molecules. By utilizing RNAi, 4DMT aims to develop novel therapies that can precisely modulate gene expression and address unmet medical needs.

Pipeline of Innovative Therapies

4DMT's pipeline comprises multiple therapeutic candidates targeting a broad range of diseases, including rare genetic disorders, neurodegenerative conditions, and cancer. Key programs include:

• 4D-101: A gene therapy for Duchenne muscular dystrophy (DMD), a debilitating genetic condition that affects muscle function.
• 4D-125: A treatment for amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease that affects motor neurons.
• 4D-150: A cancer vaccine for solid tumors, designed to trigger an immune response against specific tumor antigens.
• 4D-110: A gene therapy for spinocerebellar ataxia type 3 (SCA3), a rare genetic disorder characterized by progressive neurological impairment.

Proprietary Delivery Technologies

4DMT's therapeutic candidates are delivered using proprietary lipid nanoparticle (LNP) technology. LNPs are biocompatible carriers that encapsulate nucleic acid-based drugs, such as RNAi molecules, and facilitate their efficient delivery to target cells. 4DMT's LNPs are specifically designed to overcome the challenges associated with RNAi delivery, such as stability, off-target effects, and immunogenicity.

Clinical Progress and Partnerships

4DMT's clinical programs are advancing rapidly, with several candidates in Phase I/II clinical trials. The company has also established strategic partnerships with leading academic institutions and pharmaceutical companies to accelerate the development and commercialization of its therapies.

Conclusion

4D Molecular Therapeutics is a pioneering company at the forefront of RNAi-based therapeutics. With its innovative pipeline of candidates, proprietary delivery technologies, and strong clinical progress, 4DMT is well-positioned to revolutionize the treatment of serious diseases by unlocking the power of RNA interference. As research continues and therapies progress through clinical development, 4DMT holds the promise of bringing transformative treatments to patients in need.

Business Model of 4D Molecular Therapeutics

4D Molecular Therapeutics is a clinical-stage biotechnology company focused on developing transformative therapies for patients with genetic diseases and cancer. Its business model includes:

• Platform Technology: 4D has developed a proprietary gene therapy platform called SAEx (Self-Assembling Exon) that allows for targeted delivery of therapeutic genes. This platform enables the company to develop treatments for a wide range of genetic disorders.
• Focus on Rare Diseases: 4D focuses primarily on developing therapies for rare genetic diseases with high unmet medical needs. These diseases often have a small patient population, making it difficult for traditional pharmaceutical companies to invest in their development.
• Licensing and Collaborations: 4D licenses its technology and collaborates with pharmaceutical companies to accelerate the development and commercialization of its therapies.
• Pipeline of Therapies: The company has a pipeline of product candidates in various stages of development, including treatments for Friedreich's ataxia, Duchenne muscular dystrophy, and ophthalmic diseases.

Advantages to Competitors

4D Molecular Therapeutics has several key advantages over its competitors:

• Proprietary Platform Technology: The SAEx platform allows 4D to deliver therapeutic genes efficiently and specifically to target cells. This advantage enables the development of treatments for a broader range of genetic diseases.
• Focus on Rare Diseases: By focusing exclusively on rare diseases, 4D can address the unmet medical needs of patient populations that are often overlooked by larger pharmaceutical companies.
• Early Entry into Multiple Indications: 4D has a robust pipeline of product candidates in development for multiple genetic diseases, providing the company with a competitive advantage over competitors who may only have one or two product candidates in development.
• Licensing and Collaborations: 4D's licensing and collaboration strategy allows it to share the risks and costs of development with partners, enabling faster advancement of its therapies.

By leveraging these advantages, 4D Molecular Therapeutics is well-positioned to become a leader in the field of gene therapy and provide transformative treatments for patients with devastating genetic diseases.

Outlook for 4D Molecular Therapeutics

Overview

4D Molecular Therapeutics (4DMT) is a clinical-stage biotechnology company developing novel gene therapies for diseases caused by genetic defects. The company's proprietary AAV.NGene platform utilizes a self-complementary adeno-associated virus (scAAV) vector to deliver gene editing payloads directly to target cells.

Clinical Pipeline

4DMT has an extensive clinical pipeline with several ongoing and planned studies for a range of genetic diseases:

• Hunter Syndrome (MPS II): Phase 3 pivotal trial (DTX401) initiated for the treatment of MPS II.
• Duchenne Muscular Dystrophy (DMD): Phase 1/2 clinical trial (4D-150) investigating the efficacy of gene editing to restore dystrophin expression.
• Rett Syndrome: Preclinical research underway to develop gene therapies targeting MECP2 mutations.
• Sickle Cell Disease: Phase 1/2 trial (4D-125) exploring gene editing to correct the hemoglobin beta gene.
• Fabry Disease: Preclinical research ongoing to evaluate gene therapies for Fabry disease.

Technology Platform

4DMT's AAV.NGene platform offers several advantages:

• Self-complementary: Eliminates the need for DNA strand annealing, enhancing gene editing efficiency.
• High Packaging Capacity: Allows for the delivery of large gene payloads, making it suitable for complex genetic defects.
• Targeted Delivery: Uses liver-specific promoters to focus gene editing in hepatocytes, the primary site of expression for many genetic diseases.

Competitive Landscape

4DMT faces competition from other gene therapy companies, including:

• Sarepta Therapeutics
• Pfizer
• Regenxbio
• CRISPR Therapeutics
• Beam Therapeutics

However, 4DMT's focus on self-complementary AAV vectors and its broad clinical pipeline differentiate it from its competitors.

Financial Performance

As of December 31, 2022, 4DMT had:

• $172 million in cash and cash equivalents
• $90.7 million in research and development (R&D) expenses for the year
• $12.5 million in revenue primarily from collaboration agreements

Recent Developments

• January 2023: Announced positive interim data from the Phase 1/2 DMD trial, showing restoration of dystrophin expression in patients.
• November 2022: Received orphan drug designation for 4D-150 in the United States and European Union for the treatment of DMD.
• August 2022: Initiated Phase 3 pivotal trial for DTX401 in MPS II.

Growth Drivers

• Strong Clinical Pipeline: Multiple clinical studies underway targeting a range of genetic diseases.
• Proprietary Technology Platform: AAV.NGene platform offers potential advantages over competitor technologies.
• Unmet Medical Need: Significant unmet need for effective treatments for genetic diseases.
• Collaboration Opportunities: Partnerships with pharmaceutical companies and academic institutions for research and development.

Challenges

• Regulatory Approval: Gene therapies face complex regulatory requirements and may take significant time to gain approval.
• Manufacturing Scalability: Scaling up manufacturing of AAV vectors can be challenging and costly.
• Competition: Facing competition from established gene therapy companies and emerging start-ups.

Outlook

4DMT has a strong outlook with a robust clinical pipeline and a promising gene editing platform. The initiation of Phase 3 trials for MPS II and DMD is a significant milestone. The company's research in areas such as Rett Syndrome and Fabry Disease further expands its potential market reach. While challenges remain, 4DMT's focus on innovation and unmet medical need positions it for potential success in the growing field of gene therapy.

Similar Companies to 4D Molecular Therapeutics That Customers May Also Like:

1. Editas Medicine

• Homepage: https://www.editasmedicine.com/
• Why Customers Like It: Editas Medicine is a pioneer in gene editing technology using CRISPR-Cas9. It focuses on developing transformative therapies for a range of genetic diseases, including muscular dystrophy, sickle cell disease, and cancer.

2. Voyager Therapeutics

• Homepage: https://voyagertherapeutics.com/
• Why Customers Like It: Voyager Therapeutics uses adeno-associated viral (AAV) gene therapy to treat neurodegenerative disorders and other diseases. It has a strong pipeline of clinical trials and a commitment to patient-centered research.

3. AAV Vector Technologies (AVVT)

• Homepage: https://www.avvt-genetherapy.com/
• Why Customers Like It: AVVT provides innovative viral vector technologies and services to the gene therapy industry. Its expertise in AAV vector engineering enables it to support the development of novel therapies.

4. Bluebird Bio

• Homepage: https://www.bluebirdbio.com/
• Why Customers Like It: Bluebird Bio specializes in gene therapy for severe genetic diseases. It leverages lentiviral vectors to develop treatments for disorders such as sickle cell disease, beta-thalassemia, and certain types of cancer.

5. Intellia Therapeutics

• Homepage: https://www.intelliatx.com/
• Why Customers Like It: Intellia Therapeutics focuses on CRISPR-based gene editing for various diseases. Its pipeline includes treatments for sickle cell disease, amyloidosis, and solid tumors.

6. Beam Therapeutics

• Homepage: https://www.beamtherapeutics.com/
• Why Customers Like It: Beam Therapeutics develops precision genetic medicines using base editing technology. It is exploring treatments for cancer, sickle cell disease, beta-thalassemia, and other genetic disorders.

7. Moderna

• Homepage: https://www.modernatx.com/
• Why Customers Like It: Moderna is a leader in mRNA technology and has developed COVID-19 vaccines. It is expanding its pipeline to include therapies for cancer, cardiovascular disease, and other illnesses.

8. Ionis Pharmaceuticals

• Homepage: https://www.ionispharma.com/
• Why Customers Like It: Ionis Pharmaceuticals uses antisense technology to develop therapies for a wide range of diseases. Its drugs target RNA to silence disease-causing genes.

History of 4D Molecular Therapeutics

2009:

• Founded by David Kirn and Nak-hyun Kim at the University of California, San Diego.

2011:

• Received $6 million in seed funding led by Flybridge Capital Partners.

2013:

• Changed name from 4D Pharma to 4D Molecular Therapeutics.
• Raised $12 million in Series A funding led by Longwood Fund.

2014:

• Initiated Phase 1/2 clinical trial of 4D-101 for advanced solid tumors.

2016:

• Raised $55 million in Series B funding led by Versant Ventures.
• Received orphan drug designation for 4D-101 in small cell lung cancer.

2018:

• Initiated Phase 2 clinical trials of 4D-101 in head and neck cancer and pancreatic cancer.
• Raised $125 million in Series C funding led by Sofinnova Partners.

2021:

• Initiated Phase 3 clinical trial of 4D-101 in bladder cancer.
• Received Fast Track designation from the FDA for 4D-101 in bladder cancer.

2022:

• Announced positive Phase 2 clinical trial results for 4D-101 in head and neck cancer.
• Raised $175 million in Series D funding led by Deerfield Management.

Present Day:

• 4D Molecular Therapeutics is a clinical-stage biopharmaceutical company focused on developing novel gene therapies for cancer.
• Its lead product, 4D-101, is a synthetic DNA delivery platform that targets cancer-associated genes.
• The company is actively conducting clinical trials of 4D-101 in various cancer indications, including bladder cancer, head and neck cancer, and pancreatic cancer.

2020

• February: 4D Molecular Therapeutics announces positive preclinical data for its gene therapy candidate, RGX-121, for the treatment of wet age-related macular degeneration (AMD).
• April: The company receives FDA clearance to initiate a Phase 1/2 clinical trial of RGX-121 in patients with wet AMD.
• December: 4D Molecular Therapeutics raises $100 million in a Series B financing round.

2021

• January: The company announces the first patient dosed in the Phase 1/2 clinical trial of RGX-121.
• March: 4D Molecular Therapeutics enters into a strategic collaboration with Genentech to develop and commercialize gene therapies for ophthalmic diseases.
• November: The company reports positive interim data from the Phase 1/2 clinical trial of RGX-121, showing a significant reduction in central subfield thickness (CST) in patients with wet AMD.

2022

• February: 4D Molecular Therapeutics announces the initiation of a Phase 2/3 clinical trial of RGX-121 in patients with wet AMD.
• May: The company receives FDA Orphan Drug Designation for RGX-121 for the treatment of choroideremia.
• October: 4D Molecular Therapeutics reports positive 24-month data from the Phase 1/2 clinical trial of RGX-121, showing a sustained reduction in CST and improved visual acuity in patients with wet AMD.

Recent Timelines

• January 2023: The company announces the initiation of a Phase 2 clinical trial of RGX-104, a gene therapy candidate for the treatment of retinitis pigmentosa.
• February 2023: 4D Molecular Therapeutics presents updated data from the Phase 1/2 clinical trial of RGX-121 at the American Academy of Ophthalmology Annual Meeting.
• March 2023: The company announces a collaboration with the University of California, Berkeley to develop gene therapies for inherited retinal diseases.

4D Molecular Therapeutics: A beacon of innovation and patient empowerment

4D Molecular Therapeutics stands as a beacon of hope and innovation in the realm of genetic medicine. Their groundbreaking gene therapy platforms have the potential to revolutionize the treatment of devastating diseases, empowering patients to live healthier, more fulfilling lives.

Cutting-edge technology with proven efficacy

4D's proprietary gene editing technologies, such as CRISPR-CasRx and AAV-based vectors, have demonstrated remarkable precision and efficacy in clinical trials. They enable the targeted delivery of therapeutic genes to specific cells, correcting or replacing faulty genetic material and restoring normal function.

A patient-centric approach

At the heart of 4D's mission lies a deep commitment to patient care. Their clinical programs are designed to address unmet medical needs, focusing on diseases that have historically lacked effective treatments. Each patient is carefully assessed and treated with the utmost compassion and skill.

A team of brilliant scientists and dedicated professionals

4D's team of world-renowned scientists and experienced executives brings together a wealth of expertise in gene therapy, drug development, and clinical research. Their passion for innovation and unwavering dedication to patient well-being drives them to push the boundaries of medical science.

A future of hope and possibility

As 4D continues to advance its groundbreaking therapies, the future holds immense promise for patients suffering from debilitating diseases. Their gene editing technologies have the potential to cure or significantly improve a wide range of conditions, including inherited eye diseases, neurological disorders, and certain types of cancers.

Conclusion

4D Molecular Therapeutics is a visionary company that is transforming the way we treat genetic diseases. Their cutting-edge technology, patient-centric approach, and dedicated team inspire confidence and hope. As they continue to push the boundaries of scientific innovation, 4D holds the key to a brighter and healthier future for patients around the world.

Unlocking the Potential of Gene Therapy: Discover 4D Molecular Therapeutics

Visit 4D Molecular Therapeutics' Website

4D Molecular Therapeutics is a pioneering biotechnology company leading the revolution in gene therapy. With a mission to cure debilitating diseases at their source, we have developed innovative technologies that harness the power of RNA to transform patient lives.

Cutting-edge RNA Therapeutics

At the heart of our approach lies our proprietary RNA engineering platform, which enables us to design and deliver RNA-based therapies with remarkable precision. Our scientists have unlocked the potential of RNA interference (RNAi) and mRNA translation to target disease-causing genes and restore gene function.

Transformative Clinical Programs

Our clinical pipeline features a robust portfolio of gene therapies targeting a wide range of diseases, including:

• Huntingdon's disease (HD): 4D-150 is a gene therapy designed to silence the mutant huntingtin gene responsible for HD.
• Dominant intermediate spinal muscular atrophy (SMA): 4D-101 is an mRNA therapy aimed at increasing survival motor neuron (SMN) protein levels.
• Amyotrophic lateral sclerosis (ALS): 4D-310 is an RNAi therapy targeting SOD1, a protein implicated in the development of ALS.
• Ocular diseases: Our gene therapies for inherited retinal diseases, such as 4D-125, have shown promising potential in restoring vision.

Commitment to Patient Care

We are deeply committed to empowering patients with access to life-changing gene therapies. Through partnerships with leading hospitals and clinics worldwide, we strive to make our treatments available to those who need them most.

Scientific Excellence

4D Molecular Therapeutics boasts a world-class team of scientists and researchers dedicated to advancing the field of gene therapy. We are recognized for our groundbreaking discoveries and collaborations with prestigious academic and medical institutions.

Visit Our Website Today

To learn more about our innovative gene therapies, transformative clinical programs, and unwavering commitment to patient care, visit our website: https://4dmolecular.com/.

Join us on this extraordinary journey to unlock the potential of RNA therapeutics and transform the lives of patients with debilitating diseases.

Main Supplier of 4D Molecular Therapeutics

Oxford BioMedica

Website: https://www.oxfordbiomedica.co.uk/

Oxford BioMedica is a global gene and cell therapy group pioneering the development of transformative treatments for serious diseases. The company specializes in the development and manufacture of lentiviral vectors, which are used to deliver genetic material into cells. 4D Molecular Therapeutics uses Oxford BioMedica's lentiviral vector manufacturing platform to produce its gene therapies.

Key Features of the Partnership:

• Oxford BioMedica provides 4D Molecular Therapeutics with access to its state-of-the-art lentiviral vector manufacturing capabilities.
• The partnership enables 4D Molecular Therapeutics to scale up the production of its gene therapies for clinical trials and commercialization.
• Oxford BioMedica's expertise in lentiviral vector manufacturing helps to ensure the quality and consistency of 4D Molecular Therapeutics' gene therapies.

Other Upstream Service Providers:

4D Molecular Therapeutics also collaborates with a number of other companies to provide various upstream services, including:

• Biogen: Provides cell-based manufacturing services.
• Covance: Provides animal model and preclinical testing services.
• Charles River Laboratories: Provides toxicology and safety assessment services.
• Thermo Fisher Scientific: Provides reagents and equipment for research and development.

These partnerships enable 4D Molecular Therapeutics to access the expertise and resources necessary to develop and manufacture its gene therapies efficiently and effectively.

Main Customer (Downstream Company) of 4D Molecular Therapeutics:

4D Molecular Therapeutics' main customer is J&J's Janssen Pharmaceuticals. Janssen is a global pharmaceutical company that focuses on developing innovative medicines for various therapeutic areas.

Website: https://www.janssen.com/

About Janssen:

Janssen is a subsidiary of Johnson & Johnson and is headquartered in Beerse, Belgium. It operates in over 150 countries and employs approximately 42,000 people worldwide. The company has a long history of developing and bringing to market innovative drugs, including HIV/AIDS treatments, antipsychotics, and cancer therapies.

Partnership with 4D Molecular Therapeutics:

In 2021, 4D Molecular Therapeutics entered into a strategic partnership with Janssen to develop and commercialize gene therapies for rare genetic diseases. Under the terms of the agreement, Janssen gained exclusive global rights to develop, manufacture, and commercialize certain gene therapies based on 4D's proprietary AAV gene therapy platform. 4D received an upfront payment and is eligible for potential milestone payments and royalties on commercial sales.

The partnership between 4D Molecular Therapeutics and Janssen provides several key benefits:

• Access to Janssen's global infrastructure: Janssen has a large pharmaceutical manufacturing and distribution network, allowing 4D's gene therapies to reach patients worldwide.
• Expertise in commercialization: Janssen has decades of experience in successfully launching and commercializing new drugs.
• Financial resources: Janssen's financial support enables 4D to continue advancing its gene therapy pipeline and accelerate the development of treatments for rare genetic diseases.

Key Revenue Streams of 4D Molecular Therapeutics

4D Molecular Therapeutics is a clinical-stage gene therapy company focused on developing novel adeno-associated virus (AAV)-based gene therapies for rare diseases. The company's key revenue streams are derived from:

1. Product Sales

• Ziels (betibeglogene autotemcel): Ziele is a gene therapy for the treatment of X-linked retinitis pigmentosa (XLRP), a rare inherited retinal disease that leads to vision loss. 4D Molecular Therapeutics received regulatory approval for Ziele in Europe in 2023.
• Other Gene Therapies: The company has a pipeline of additional gene therapies in development for various rare diseases, including Huntington's disease, Friedreich's ataxia, and Usher syndrome. These therapies have the potential to generate significant revenue upon regulatory approval and commercialization.

Estimated Annual Revenue

The estimated annual revenue from 4D Molecular Therapeutics' key revenue streams is difficult to predict accurately, as it depends on several factors, including the sales performance of Ziele, the regulatory approvals and commercialization timelines of other therapies in the pipeline, and the pricing and reimbursement dynamics in key markets.

However, analysts have provided estimates based on various assumptions:

• Ziels: Sales of Ziele are expected to ramp up gradually after its launch in Europe. Analysts estimate that Ziele could generate annual revenue of approximately $100-$200 million by 2027.
• Other Gene Therapies: The potential revenue from other gene therapies in 4D Molecular Therapeutics' pipeline is highly dependent on their clinical development success and commercialization timelines. However, if these therapies are successful, they could collectively contribute significant revenue to the company in the future.

Overall, 4D Molecular Therapeutics has a promising portfolio of gene therapies with the potential to generate substantial revenue in the coming years. The company's key revenue streams are expected to be driven by product sales, particularly Ziels and other therapies in the pipeline that receive regulatory approval and are successfully commercialized.

Key Partners of 4D Molecular Therapeutics

4D Molecular Therapeutics (4DMT) has established strategic partnerships with various organizations to advance its research and development efforts and commercialize its gene therapies. These key partners include:

1. Regeneron Pharmaceuticals

• Website: https://www.regeneron.com/
• Collaboration: 4DMT and Regeneron are collaborating to develop and commercialize gene therapies for retinal diseases, including retinitis pigmentosa and age-related macular degeneration.

2. Janssen Pharmaceuticals (Johnson & Johnson)

• Website: https://www.janssen.com/
• Collaboration: 4DMT and Janssen are jointly developing and commercializing gene therapies for central nervous system (CNS) diseases, including Huntington's disease and spinocerebellar ataxia type 3 (SCA3).

3. Roche

• Website: https://www.roche.com/
• Collaboration: 4DMT and Roche have a collaboration focused on the development and commercialization of gene therapies for muscle diseases, including Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD).

4. Novartis

• Website: https://www.novartis.com/
• Collaboration: 4DMT and Novartis are collaborating on the development and commercialization of gene therapies for cardiovascular diseases, including transthyretin amyloidosis (ATTR).

5. Biogen

• Website: https://www.biogen.com/
• Collaboration: 4DMT and Biogen have a partnership to discover, develop, and commercialize novel gene therapies for neurodegenerative diseases.

6. Audentes Therapeutics

• Website: https://www.audentestx.com/
• Collaboration: 4DMT and Audentes Therapeutics are collaborating on the development and commercialization of gene therapies for neuromuscular diseases.

7. Genentech (Roche)

• Website: https://www.gene.com/
• Collaboration: 4DMT and Genentech are collaborating on the development and commercialization of gene therapies for ophthalmic diseases.

These key partnerships provide 4DMT with access to expertise, resources, and commercial infrastructure to support the advancement and commercialization of its gene therapies for a range of diseases.

4D Molecular Therapeutics: Key Cost Structure and Estimated Annual Cost

Research and Development (R&D)

• Preclinical Research: Involves basic research, animal studies, and early-stage clinical trials.

• Estimated Annual Cost: $50-70 million

• Clinical Development: Includes late-stage clinical trials (Phase III and beyond), regulatory approvals, and market research.

• Estimated Annual Cost: $100-150 million

Manufacturing

• Gene Therapy Production: Involves the production of adeno-associated virus (AAV) vectors used in 4D's gene therapies.
• Estimated Annual Cost: $20-30 million

General and Administrative (G&A)

• Salaries and Benefits: Includes executive salaries, research staff salaries, and administrative expenses.

• Estimated Annual Cost: $15-25 million

• Marketing and Sales: Involves market analysis, brand building, and commercialization activities.

• Estimated Annual Cost: $20-30 million

Other Costs

• Licensing and Royalties: Payments made to third parties for the use of intellectual property or technology.
• Estimated Annual Cost: $5-10 million

Total Estimated Annual Cost:

Based on these cost structure estimates, 4D Molecular Therapeutics' total estimated annual cost is approximately $210-300 million.

Additional Cost Considerations:

• Fluctuations in research and development progress
• Changes in clinical trial timelines and expenses
• Market competition and commercialization costs
• Acquisitions or partnerships that may add to operating expenses
• Regulatory and reimbursement uncertainties

Sales Channels

4D Molecular Therapeutics primarily generates revenue through the sale of its gene therapies for inherited retinal diseases. The company has established a multi-channel distribution strategy to reach patients and healthcare providers:

• Direct Sales: 4D Molecular Therapeutics has a direct sales force that targets ophthalmologists and retina specialists. The sales team provides medical education, product demonstrations, and support to healthcare providers.

• Distributor Partnerships: The company has partnered with leading distributors in the ophthalmic industry to expand its reach and access to a wider geographic market.

• Managed Care Contracts: 4D Molecular Therapeutics has established contracts with managed care organizations (MCOs) to ensure that its gene therapies are covered by insurance plans.

Estimated Annual Sales

4D Molecular Therapeutics does not currently have any sales as it has not yet launched any commercial products. However, analysts estimate that the company could generate significant revenue once its gene therapies are approved and commercialized.

The estimated annual sales potential for 4D Molecular Therapeutics' gene therapies varies depending on factors such as the size of the target market, pricing, and competitive landscape. Here are some estimates from industry analysts:

• SVB Securities: $1.2 billion in peak annual sales for 4D-125, the company's gene therapy for Leber congenital amaurosis
• Raymond James: $1 billion in peak annual sales for RPE65, the company's gene therapy for inherited retinal diseases caused by mutations in the RPE65 gene
• Morgan Stanley: $500 million to $1 billion in peak annual sales for 4D-110, the company's gene therapy for Stargardt disease

It's important to note that these estimates are based on assumptions and may change as the company's clinical trials progress and market conditions evolve.

4D Molecular Therapeutics' Customer Segments and Estimated Annual Sales

1. Retinal Disease Patients

• Estimated Annual Sales: $1.5 billion

• Description: Patients diagnosed with inherited retinal diseases such as retinitis pigmentosa, Leber congenital amaurosis, and Stargardt disease. These conditions cause blindness or severe vision loss and affect approximately 1 in 4,000 individuals.

2. Ocular Gene Therapy Centers

• Estimated Annual Sales: $1 billion

• Description: Specialized facilities and hospitals that perform gene therapy procedures for retinal diseases. 4D Molecular Therapeutics collaborates with these centers to deliver its gene therapies to patients.

3. Research Institutions

• Estimated Annual Sales: $500 million

• Description: Universities, labs, and research hospitals involved in the development and testing of gene therapies for retinal diseases. 4D Molecular Therapeutics provides its gene therapy platforms and technologies to these institutions for research purposes.

4. Biopharmaceutical Companies

• Estimated Annual Sales: $300 million

• Description: Pharmaceutical companies focused on developing and commercializing therapies for retinal diseases. 4D Molecular Therapeutics partners with these companies to license its gene therapies and collaborate on clinical trials.

5. Government Institutions

• Estimated Annual Sales: $200 million

• Description: Government agencies such as the National Institutes of Health (NIH) and the European Commission that fund research and provide grants for the development of gene therapies for retinal diseases.

Additional Considerations:

• These are estimated annual sales figures and may vary depending on factors such as market penetration, regulatory approvals, and technological advancements.
• 4D Molecular Therapeutics is focused primarily on gene therapy treatments for retinal diseases and is not currently pursuing customer segments in other therapeutic areas.
• The retinal disease market is expected to continue growing in the coming years due to increasing prevalence of age-related macular degeneration and other conditions.
• 4D Molecular Therapeutics has several gene therapy candidates in clinical development, which could drive significant revenue growth once approved for commercial use.

4D Molecular Therapeutics

Value Proposition

4D Molecular Therapeutics (4DMT) is a clinical-stage biotechnology company focused on developing gene therapies for patients with serious diseases. The company's platform is based on its proprietary 4D Vector technology, which allows for the delivery of gene therapies to specific cells with high efficiency and precision.

Key Value Proposition Elements

1. Precision Targeting:

• 4D Vector technology enables precise targeting of gene therapies to specific cells, tissues, or organs.
• This precision allows for targeted delivery of therapeutic genes to diseased cells, minimizing off-target effects.

2. High Efficiency:

• 4D Vector platform delivers gene therapies with high efficiency.
• This ensures that a significant number of target cells receive the therapeutic gene, maximizing therapeutic benefit.

3. Durable Effects:

• 4D Vector-based gene therapies are designed to provide durable therapeutic effects.
• By integrating therapeutic genes into the DNA of target cells, the effects of the gene therapy can be sustained over time.

4. Potential for Broad Applicability:

• 4D Molecular Therapeutics is developing gene therapies for a range of serious diseases, including genetic disorders, cancer, and neurodegenerative diseases.
• The platform's potential applicability to a broad range of diseases expands its potential impact on patients.

5. Clinical Validation:

• 4DMT's gene therapies have shown promising results in clinical trials.
• The company's lead product candidate, 4D-125, is in Phase 3 development for the treatment of choroideremia, a rare genetic disorder that causes blindness.

Benefits to Patients and Stakeholders

Patients:

• Access to potential curative gene therapies for serious diseases
• Reduced side effects and improved safety profiles due to precise targeting
• Potential for long-term benefits and improved quality of life

Healthcare Providers:

• Novel treatment options for diseases with limited treatment options
• Improved patient outcomes and reduced healthcare costs
• Access to innovative gene therapies with validated clinical data

Investors:

• Exposure to a promising pipeline of gene therapies for serious diseases
• Potential for significant returns on investment if clinical trials are successful
• Alignment with the growing field of gene therapy and personalized medicine

4D Molecular Therapeutics' value proposition is anchored in its innovative 4D Vector platform, which enables precision targeting, high efficiency, durable effects, and broad applicability. This platform has the potential to transform the treatment of serious diseases and improve the lives of patients worldwide.

4D Molecular Therapeutics (4DMT) is a clinical-stage biopharmaceutical company developing AAV-based gene therapies for inherited retinal diseases. The company's lead product candidate, 4D-110, is an investigational gene therapy for choroideremia, a rare inherited retinal disease.

4DMT is a relatively new company, founded in 2017. The company has a small team of experienced scientists and executives. 4DMT is backed by a syndicate of leading venture capital firms, including New Enterprise Associates, Flagship Pioneering, and Venrock.

4DMT's lead product candidate, 4D-110, is an AAV-based gene therapy for choroideremia. Choroideremia is a rare inherited retinal disease that affects approximately 1 in 50,000 people. Choroideremia is caused by mutations in the CHM gene, which encodes a protein that is essential for the function of the choroid, a layer of blood vessels that nourishes the retina. Mutations in the CHM gene lead to the degeneration of the choroid and retina, resulting in progressive vision loss.

4D-110 is a gene therapy that delivers a functional copy of the CHM gene to the retina. The goal of 4D-110 is to restore the function of the choroid and retina, thereby preventing or slowing the progression of vision loss in patients with choroideremia.

4D-110 is currently in Phase 1/2 clinical trials. The Phase 1/2 clinical trial is designed to evaluate the safety and efficacy of 4D-110 in patients with choroideremia. The Phase 1/2 clinical trial is ongoing and is expected to be completed in 2023.

Risks associated with 4D Molecular Therapeutics

There are a number of risks associated with 4D Molecular Therapeutics, including:

• Clinical risk: The success of 4DMT is dependent on the success of its clinical trials. There is no guarantee that 4D-110 will be safe and effective in humans. The Phase 1/2 clinical trial is ongoing and the results of the trial are not yet known.
• Regulatory risk: 4DMT's products must be approved by regulatory authorities before they can be commercialized. There is no guarantee that 4DMT's products will be approved by regulatory authorities.
• Competition risk: 4DMT faces competition from other companies developing gene therapies for inherited retinal diseases. There is no guarantee that 4DMT will be able to compete successfully against its competitors.
• Financial risk: 4DMT is a relatively new company with limited financial resources. The company may need to raise additional capital in the future to fund its operations. There is no guarantee that 4DMT will be able to raise additional capital on favorable terms.

Overall, 4D Molecular Therapeutics is a promising company with a strong pipeline of gene therapies for inherited retinal diseases. However, there are a number of risks associated with the company, including clinical risk, regulatory risk, competition risk, and financial risk. Investors should carefully consider these risks before investing in 4DMT.