Latest news with #Epidermolysisbullosa
Business Wire
27-05-2025
- Business
- Business Wire
RHEACELL and AOP Health enter into strategic partnership to commercialize novel somatic cell therapies in Europe
HEIDELBERG, Germany--(BUSINESS WIRE)--RHEACELL announced today that it has entered into an exclusive partnership agreement with the AOP Health Group, who will commercialize RHEACELL's two stem cell products, EBESANAR ® and AMESANAR ®, in Europe, Turkey, Israel and the MENA region (Middle East and Northern Africa). RHEACELL retains full commercialization rights to EBESANAR ® and AMESANAR ® in other major markets, including North America, Asia-Pacific and Latin America, and will seek strategic distribution partners in those markets. Innovative portfolio in late-stage development RHEACELL's somatic cell therapeutics are at an advanced stage of clinical development and are seen as a beacon of hope for patients with non-healing venous wounds (chronic venous ulcers) and the rare pediatric disease - Epidermolysis bullosa ("butterfly disease"). RHEACELL will continue to be responsible for the clinical phase 3 trials study program for EBESANAR ® and AMESANAR ®. Initial topline data from the EBESANAR ® Phase 3 trial is expected by the end of 2025. The AMESANAR ® Phase 3 clinical trial in chronic venous ulcer disease is also progressing according to plan and is expected to be completed in 2026. Following expected marketing approval in Europe, AOP Health will take the lead in all commercialization and sales activities in the agreed regions, in addition to overseeing medical and regulatory responsibilities. This reflects AOP Health's strong track record of successfully bringing integrated therapies to market across Europe. RHEACELL will retain manufacturing rights and produce both therapies at its state-of-the-art facility in Heidelberg. United by a shared commitment, both companies are working toward a clear goal: making advanced therapeutic solutions available to patients with high unmet medical needs as swiftly as possible. The financial terms of the agreement remain confidential. "This partnership marks a significant milestone for RHEACELL and the further development of our cell therapy programs. Working with such an established partner as AOP Health to commercialize our cell therapeutics will enable us to bring our innovative therapies faster and more efficiently to the markets across Europe," said Dr. Christoph Ganss, MD, CEO of RHEACELL. 'Our mission is to provide patients with rare diseases access to urgently needed therapeutic innovation. This partnership with stem cell pioneer RHEACELL enables us to jointly bring new treatment options to patients living with Epidermolysis bullosa or chronic venous ulcers, who currently have limited or no treatment options. Our teams are fully committed to making these groundbreaking treatments available to all patients affected,' said Dr. Martin Steinhart, CEO of AOP Health. About RHEACELL RHEACELL is a leading integrative biopharmaceutical stem cell company based in Heidelberg, Germany. We focus on the development of innovative stem cell therapies for patients suffering from severe immune- and inflammation-related diseases, who have a very high level of suffering and for whom there are currently no adequate treatment options. Our products are based on ABCB5+ mesenchymal stromal cells as a pure active ingredient with a unique mechanism of action - applied topically or systemically, depending on the clinical picture - which enables the targeted control of inflammation and the restoration of normal physiological wound healing. About AOP Health AOP Health is a globally active group of companies with roots in Austria, where the headquarters of AOP Orphan Pharmaceuticals GmbH ("AOP Health") is located. Since 1996, the AOP Health Group has been dedicated to developing innovative solutions for unmet medical needs, particularly in the areas of rare diseases and critical care. The Group has established itself internationally as a pioneer for integrated therapy solutions and operates worldwide with subsidiaries, agencies and a strong partner network. Under the claim "Needs. Science. Trust.", the AOP Health Group emphasizes its commitment to research and development and the importance of building relationships with physicians and patient advocacy groups to ensure that the needs of these stakeholders are reflected in all aspects of the company's activities About EBESANAR ® EBESANAR ® is a systemic therapy for patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Junctional Epidermolysis Bullosa (JEB), rare genetic diseases that affect the skin and connective tissues. EBESANAR ® is administered via bi-monthly intravenous infusions and is intended to treat skin lesions, reduce internal and external disease burden and prevent accumulation of damage to tissues. EBESANAR ® is currently being studied in a phase 3 trial in the US and Europe. Top line data from this trial is expected to be available in later 2025. About AMESANAR ® AMESANAR ® is a topical therapy for the treatment of non-healing, treatment resistant chronic venous ulcers. AMESANAR ® is administered topically to patients whose ulcers have not responded to other standard therapies and is intended to support wound healing by activating macrophages which express anti-inflammatory & pro-angiogenic cytokines. AMESANAR ® is currently being studied in a Phase 3 clinical trial in the US and Europe with top line data expected in 2026.
Yahoo
27-05-2025
- Business
- Yahoo
RHEACELL and AOP Health: Strategic Partnership to Deliver Breakthrough Therapies for the "Butterfly Children's Disease" and Chronic Venous Wounds
Partnership aims to provide patients with high unmet medical needs access to innovative cell therapies HEIDELBERG, Germany & VIENNA, May 27, 2025--(BUSINESS WIRE)--RHEACELL, a German biotech company specialized in the development of stem cell therapies, and AOP Health, a Europe-based leader in integrated therapies for rare diseases and in intensive care, are forming a strategic alliance. This partnership marks a crucial step toward making breakthrough therapies available to patients with high unmet medical needs – particularly those affected by Epidermolysis bullosa (EB or "butterfly children's disease") and chronic venous wounds. Both conditions currently have limited treatment options, severely impacting the lives of patients and their families. Two novel therapies from RHEACELL's pipeline are currently in Phase 3 clinical development. Combining RHEACELL's cutting-edge R&D capabilities with AOP Health's proven expertise in bringing rare disease therapies to patients in Europe, this partnership underscores both companies' strong commitment to addressing high unmet medical needs. With 30 years of experience and a growing presence in more than 50 countries, AOP Health complements this ambition by providing the expertise needed to make RHEACELL's cell therapies available not only in Europe, but also in the Middle East, North Africa, Turkey, and Israel. Promising cell therapy Cell-based regenerative therapies are gaining increasing importance, particularly for diseases with currently limited therapeutic options. For over 20 years, RHEACELL has focused on the research and development of so-called stromal cells - stabilizing cells obtained from donor skin. RHEACELL's cell therapy is only the second stromal cell product to receive national marketing authorization from the Paul Ehrlich Institute in Germany for the treatment of therapy-resistant, chronic venous wounds ("leg ulcer") on the basis of positive phase 2 clinical trial data.1; 2 An innovative mechanism of action RHEACELL's cell therapy is based on a specific type of cells, so-called ABCB5+ mesenchymal stromal cells, with anti-inflammatory and immunomodulatory properties. These cells represent a promising new therapeutic approach for EB and chronic venous wounds. In patients with EB, this therapy is administered systemically and can thus stimulate both internal and external wound healing by restoring the normal physiological function of affected tissues – an innovative mechanism of action. This can lead to a reduction in the number of existing wounds and help prevent the formation of new ones.3 Building on positive Phase 2 results and positive feedback from regulatory authorities, two Phase 3 clinical trials are currently underway, and a first EMA submission is already expected in 2026. Background: Voices on the Partnership "This partnership marks a significant milestone for RHEACELL and the further development of our stem cell medication programs. Collaborating with an established partner like AOP Health to commercialize our stromal cell medications will allow us to bring our innovative therapies to the European patients faster and more efficiently."Dr. med. Christoph Ganss, CEO RHEACELL "Our mission is to provide patients with rare diseases access to urgently needed therapeutic innovation. This partnership with stem cell pioneer RHEACELL enables us to jointly bring new treatment options to patients living with chronic venous wounds or Epidermolysis bullosa, who currently have limited or no treatment options. Our teams are fully committed to making these groundbreaking treatments available to all patients affected."Dr. Martin Steinhart, CEO AOP Health "In treating Epidermolysis bullosa, we have had few therapeutic options to date. This lack of treatment options adds an additional burden on those affected. From a medical perspective, the concept of regenerative, anti-inflammatory cell therapy is a promising approach. That's why our study center is participating in the next milestone—Phase 3 trials."Univ.-Prof. Dr. Johann Bauer, Head of EB Research at the EB-Haus Austria, the world's first specialized clinic for 'butterfly children'. "RHEACELL's approach of administering stromal cells systemically to stimulate internal and external wound healing distinguishes it from other approaches that focus primarily on local applications on the skin. We are thrilled to follow the ongoing clinical development."Dr. Rainer Riedl, founder and chairman of DEBRA Austria About Epidermolysis bullosa (EB) EB is a congenital and currently incurable condition that can affect not only the skin but also internal organs. With approximately 500,000 people affected worldwide4, EB is a rare disease that severely impacts patients' quality of life. In this genetic disorder, the skin is as fragile as a butterfly's wing. Even minor mechanical stress or friction can cause blistering and painful chronic wounds. The patient organization DEBRA describes EB as "the worst disease you've never heard of." DEBRA was founded in 1995 as a patient organization by patients, families, and physicians with the goal of facilitating exchange and providing support to people living with EB. One of DEBRA Austria's key initiatives is the EB-Haus Austria, located at the Salzburg University Hospital campus. It serves as a center of excellence for Epidermolysis bullosa and is also the world's first specialized clinic for "butterfly children." About Chronic Venous Wounds (leg ulcer) Venous ulcers are chronic wounds that result from prolonged venous insufficiency, primarily affecting older adults. These ulcers develop due to poor blood flow in the veins, leading to tissue breakdown, especially in the lower extremities. They are the most common type of leg ulcers, accounting for 60–80% of cases. About AOP Health AOP Health is a global enterprise group with roots in Austria, where the headquarters of AOP Orphan Pharmaceuticals GmbH ("AOP Health") is located. Since 1996, the AOP Health Group has been dedicated to developing innovative solutions to address unmet medical needs, particularly in the fields of rare diseases and intensive care medicine. The group has established itself internationally as a pioneer in integrated therapy solutions and operates worldwide through subsidiaries, representations, and a strong network of partners. With the claim "Needs. Science. Trust." the AOP Health Group emphasizes its commitment to research and development, as well as the importance of building relationships with physicians and patient advocacy groups to ensure that the needs of these stakeholders are reflected in all aspects of the company's actions. ( About RHEACELL With more than 20 years of experience, we are a leading, integrated biopharmaceutical stem cell company based in Heidelberg, Germany, currently conducting two approval trials (EU, US). We focus on innovative stem cell therapies for patients suffering from severe immune- and inflammation-related diseases who experience high levels of distress and for whom no adequate treatments currently exist. Our goal is to offer these patients a new and innovative treatment option. Our ABCB5+ mesenchymal stromal cells, as a purified active ingredient, can improve the lives of these patients—for example, in cases of Epidermolysis bullosa — and have the potential to transform treatment paradigms for these diseases. Our targeted approach to fighting inflammation through our proprietary stem cell therapy enables the restoration of normal physiological function in affected individuals. 1 2 3 Kiritsi et al. Clinical trial of ABCB5-positive mesenchymal stem cells for recessive dystrophic epidermolysis bullosa. JCI Insight 2021;6:e151922 4 Rashidghamat, E. et al (2017). Persistent and rare diseases research. 2017; 6(1):6-20. DOI: 10.5582/irdr.2017.01005 View source version on Contacts AOP HEALTH: Mag. Nina Roth, MASDirector of Corporate CommunicationT +43 676 3131509E Press Contacts RHEACELL: Daniela WeisE media@ Error while retrieving data Sign in to access your portfolio Error while retrieving data
Yahoo
27-05-2025
- Business
- Yahoo
RHEACELL and AOP Health: Strategic Partnership to Deliver Breakthrough Therapies for the "Butterfly Children's Disease" and Chronic Venous Wounds
Partnership aims to provide patients with high unmet medical needs access to innovative cell therapies HEIDELBERG, Germany & VIENNA, May 27, 2025--(BUSINESS WIRE)--RHEACELL, a German biotech company specialized in the development of stem cell therapies, and AOP Health, a Europe-based leader in integrated therapies for rare diseases and in intensive care, are forming a strategic alliance. This partnership marks a crucial step toward making breakthrough therapies available to patients with high unmet medical needs – particularly those affected by Epidermolysis bullosa (EB or "butterfly children's disease") and chronic venous wounds. Both conditions currently have limited treatment options, severely impacting the lives of patients and their families. Two novel therapies from RHEACELL's pipeline are currently in Phase 3 clinical development. Combining RHEACELL's cutting-edge R&D capabilities with AOP Health's proven expertise in bringing rare disease therapies to patients in Europe, this partnership underscores both companies' strong commitment to addressing high unmet medical needs. With 30 years of experience and a growing presence in more than 50 countries, AOP Health complements this ambition by providing the expertise needed to make RHEACELL's cell therapies available not only in Europe, but also in the Middle East, North Africa, Turkey, and Israel. Promising cell therapy Cell-based regenerative therapies are gaining increasing importance, particularly for diseases with currently limited therapeutic options. For over 20 years, RHEACELL has focused on the research and development of so-called stromal cells - stabilizing cells obtained from donor skin. RHEACELL's cell therapy is only the second stromal cell product to receive national marketing authorization from the Paul Ehrlich Institute in Germany for the treatment of therapy-resistant, chronic venous wounds ("leg ulcer") on the basis of positive phase 2 clinical trial data.1; 2 An innovative mechanism of action RHEACELL's cell therapy is based on a specific type of cells, so-called ABCB5+ mesenchymal stromal cells, with anti-inflammatory and immunomodulatory properties. These cells represent a promising new therapeutic approach for EB and chronic venous wounds. In patients with EB, this therapy is administered systemically and can thus stimulate both internal and external wound healing by restoring the normal physiological function of affected tissues – an innovative mechanism of action. This can lead to a reduction in the number of existing wounds and help prevent the formation of new ones.3 Building on positive Phase 2 results and positive feedback from regulatory authorities, two Phase 3 clinical trials are currently underway, and a first EMA submission is already expected in 2026. Background: Voices on the Partnership "This partnership marks a significant milestone for RHEACELL and the further development of our stem cell medication programs. Collaborating with an established partner like AOP Health to commercialize our stromal cell medications will allow us to bring our innovative therapies to the European patients faster and more efficiently."Dr. med. Christoph Ganss, CEO RHEACELL "Our mission is to provide patients with rare diseases access to urgently needed therapeutic innovation. This partnership with stem cell pioneer RHEACELL enables us to jointly bring new treatment options to patients living with chronic venous wounds or Epidermolysis bullosa, who currently have limited or no treatment options. Our teams are fully committed to making these groundbreaking treatments available to all patients affected."Dr. Martin Steinhart, CEO AOP Health "In treating Epidermolysis bullosa, we have had few therapeutic options to date. This lack of treatment options adds an additional burden on those affected. From a medical perspective, the concept of regenerative, anti-inflammatory cell therapy is a promising approach. That's why our study center is participating in the next milestone—Phase 3 trials."Univ.-Prof. Dr. Johann Bauer, Head of EB Research at the EB-Haus Austria, the world's first specialized clinic for 'butterfly children'. "RHEACELL's approach of administering stromal cells systemically to stimulate internal and external wound healing distinguishes it from other approaches that focus primarily on local applications on the skin. We are thrilled to follow the ongoing clinical development."Dr. Rainer Riedl, founder and chairman of DEBRA Austria About Epidermolysis bullosa (EB) EB is a congenital and currently incurable condition that can affect not only the skin but also internal organs. With approximately 500,000 people affected worldwide4, EB is a rare disease that severely impacts patients' quality of life. In this genetic disorder, the skin is as fragile as a butterfly's wing. Even minor mechanical stress or friction can cause blistering and painful chronic wounds. The patient organization DEBRA describes EB as "the worst disease you've never heard of." DEBRA was founded in 1995 as a patient organization by patients, families, and physicians with the goal of facilitating exchange and providing support to people living with EB. One of DEBRA Austria's key initiatives is the EB-Haus Austria, located at the Salzburg University Hospital campus. It serves as a center of excellence for Epidermolysis bullosa and is also the world's first specialized clinic for "butterfly children." About Chronic Venous Wounds (leg ulcer) Venous ulcers are chronic wounds that result from prolonged venous insufficiency, primarily affecting older adults. These ulcers develop due to poor blood flow in the veins, leading to tissue breakdown, especially in the lower extremities. They are the most common type of leg ulcers, accounting for 60–80% of cases. About AOP Health AOP Health is a global enterprise group with roots in Austria, where the headquarters of AOP Orphan Pharmaceuticals GmbH ("AOP Health") is located. Since 1996, the AOP Health Group has been dedicated to developing innovative solutions to address unmet medical needs, particularly in the fields of rare diseases and intensive care medicine. The group has established itself internationally as a pioneer in integrated therapy solutions and operates worldwide through subsidiaries, representations, and a strong network of partners. With the claim "Needs. Science. Trust." the AOP Health Group emphasizes its commitment to research and development, as well as the importance of building relationships with physicians and patient advocacy groups to ensure that the needs of these stakeholders are reflected in all aspects of the company's actions. ( About RHEACELL With more than 20 years of experience, we are a leading, integrated biopharmaceutical stem cell company based in Heidelberg, Germany, currently conducting two approval trials (EU, US). We focus on innovative stem cell therapies for patients suffering from severe immune- and inflammation-related diseases who experience high levels of distress and for whom no adequate treatments currently exist. Our goal is to offer these patients a new and innovative treatment option. Our ABCB5+ mesenchymal stromal cells, as a purified active ingredient, can improve the lives of these patients—for example, in cases of Epidermolysis bullosa — and have the potential to transform treatment paradigms for these diseases. Our targeted approach to fighting inflammation through our proprietary stem cell therapy enables the restoration of normal physiological function in affected individuals. 1 2 3 Kiritsi et al. Clinical trial of ABCB5-positive mesenchymal stem cells for recessive dystrophic epidermolysis bullosa. JCI Insight 2021;6:e151922 4 Rashidghamat, E. et al (2017). Persistent and rare diseases research. 2017; 6(1):6-20. DOI: 10.5582/irdr.2017.01005 View source version on Contacts AOP HEALTH: Mag. Nina Roth, MASDirector of Corporate CommunicationT +43 676 3131509E Press Contacts RHEACELL: Daniela WeisE media@ Sign in to access your portfolio
Business Wire
27-05-2025
- Health
- Business Wire
RHEACELL and AOP Health: Strategic Partnership to Deliver Breakthrough Therapies for the 'Butterfly Children's Disease' and Chronic Venous Wounds
HEIDELBERG, Germany & VIENNA--(BUSINESS WIRE)-- RHEACELL, a German biotech company specialized in the development of stem cell therapies, and AOP Health, a Europe-based leader in integrated therapies for rare diseases and in intensive care, are forming a strategic alliance. This partnership marks a crucial step toward making breakthrough therapies available to patients with high unmet medical needs – particularly those affected by Epidermolysis bullosa (EB or 'butterfly children's disease') and chronic venous wounds. Both conditions currently have limited treatment options, severely impacting the lives of patients and their families. Two novel therapies from RHEACELL's pipeline are currently in Phase 3 clinical development. Combining RHEACELL's cutting-edge R&D capabilities with AOP Health's proven expertise in bringing rare disease therapies to patients in Europe, this partnership underscores both companies' strong commitment to addressing high unmet medical needs. RHEACELL, a German biotech company specialized in the development of stem cell therapies, and AOP Health, a Europe-based leader in integrated therapies for rare diseases and in intensive care, are forming a strategic alliance. Share With 30 years of experience and a growing presence in more than 50 countries, AOP Health complements this ambition by providing the expertise needed to make RHEACELL's cell therapies available not only in Europe, but also in the Middle East, North Africa, Turkey, and Israel. Promising cell therapy Cell-based regenerative therapies are gaining increasing importance, particularly for diseases with currently limited therapeutic options. For over 20 years, RHEACELL has focused on the research and development of so-called stromal cells - stabilizing cells obtained from donor skin. RHEACELL's cell therapy is only the second stromal cell product to receive national marketing authorization from the Paul Ehrlich Institute in Germany for the treatment of therapy-resistant, chronic venous wounds ("leg ulcer") on the basis of positive phase 2 clinical trial data. 1; 2 An innovative mechanism of action RHEACELL's cell therapy is based on a specific type of cells, so-called ABCB5+ mesenchymal stromal cells, with anti-inflammatory and immunomodulatory properties. These cells represent a promising new therapeutic approach for EB and chronic venous wounds. In patients with EB, this therapy is administered systemically and can thus stimulate both internal and external wound healing by restoring the normal physiological function of affected tissues – an innovative mechanism of action. This can lead to a reduction in the number of existing wounds and help prevent the formation of new ones. 3 Building on positive Phase 2 results and positive feedback from regulatory authorities, two Phase 3 clinical trials are currently underway, and a first EMA submission is already expected in 2026. Background: Voices on the Partnership "This partnership marks a significant milestone for RHEACELL and the further development of our stem cell medication programs. Collaborating with an established partner like AOP Health to commercialize our stromal cell medications will allow us to bring our innovative therapies to the European patients faster and more efficiently." Dr. med. Christoph Ganss, CEO RHEACELL "Our mission is to provide patients with rare diseases access to urgently needed therapeutic innovation. This partnership with stem cell pioneer RHEACELL enables us to jointly bring new treatment options to patients living with chronic venous wounds or Epidermolysis bullosa, who currently have limited or no treatment options. Our teams are fully committed to making these groundbreaking treatments available to all patients affected." Dr. Martin Steinhart, CEO AOP Health "In treating Epidermolysis bullosa, we have had few therapeutic options to date. This lack of treatment options adds an additional burden on those affected. From a medical perspective, the concept of regenerative, anti-inflammatory cell therapy is a promising approach. That's why our study center is participating in the next milestone—Phase 3 trials." Univ.-Prof. Dr. Johann Bauer, Head of EB Research at the EB-Haus Austria, the world's first specialized clinic for 'butterfly children'. "RHEACELL's approach of administering stromal cells systemically to stimulate internal and external wound healing distinguishes it from other approaches that focus primarily on local applications on the skin. We are thrilled to follow the ongoing clinical development." Dr. Rainer Riedl, founder and chairman of DEBRA Austria About Epidermolysis bullosa (EB) EB is a congenital and currently incurable condition that can affect not only the skin but also internal organs. With approximately 500,000 people affected worldwide 4, EB is a rare disease that severely impacts patients' quality of life. In this genetic disorder, the skin is as fragile as a butterfly's wing. Even minor mechanical stress or friction can cause blistering and painful chronic wounds. The patient organization DEBRA describes EB as 'the worst disease you've never heard of.' DEBRA was founded in 1995 as a patient organization by patients, families, and physicians with the goal of facilitating exchange and providing support to people living with EB. One of DEBRA Austria's key initiatives is the EB-Haus Austria, located at the Salzburg University Hospital campus. It serves as a center of excellence for Epidermolysis bullosa and is also the world's first specialized clinic for 'butterfly children.' About Chronic Venous Wounds (leg ulcer) Venous ulcers are chronic wounds that result from prolonged venous insufficiency, primarily affecting older adults. These ulcers develop due to poor blood flow in the veins, leading to tissue breakdown, especially in the lower extremities. They are the most common type of leg ulcers, accounting for 60–80% of cases. About AOP Health AOP Health is a global enterprise group with roots in Austria, where the headquarters of AOP Orphan Pharmaceuticals GmbH ("AOP Health") is located. Since 1996, the AOP Health Group has been dedicated to developing innovative solutions to address unmet medical needs, particularly in the fields of rare diseases and intensive care medicine. The group has established itself internationally as a pioneer in integrated therapy solutions and operates worldwide through subsidiaries, representations, and a strong network of partners. With the claim "Needs. Science. Trust." the AOP Health Group emphasizes its commitment to research and development, as well as the importance of building relationships with physicians and patient advocacy groups to ensure that the needs of these stakeholders are reflected in all aspects of the company's actions. ( About RHEACELL With more than 20 years of experience, we are a leading, integrated biopharmaceutical stem cell company based in Heidelberg, Germany, currently conducting two approval trials (EU, US). We focus on innovative stem cell therapies for patients suffering from severe immune- and inflammation-related diseases who experience high levels of distress and for whom no adequate treatments currently exist. Our goal is to offer these patients a new and innovative treatment option. Our ABCB5+ mesenchymal stromal cells, as a purified active ingredient, can improve the lives of these patients—for example, in cases of Epidermolysis bullosa — and have the potential to transform treatment paradigms for these diseases. Our targeted approach to fighting inflammation through our proprietary stem cell therapy enables the restoration of normal physiological function in affected individuals.
Associated Press
29-04-2025
- Business
- Associated Press
DEBRA Research Announces Impact Investment into Nova Anchora to Advance Protein Therapy for Epidermolysis Bullosa
MUNICH, Germany and SAN FRANCISCO, April 29, 2025 (GLOBE NEWSWIRE) -- DEBRA Research gGmbH, a German non-profit organization dedicated to advancing research and drug development for those affected by Epidermolysis bullosa (EB), is investing alongside Viking Global Investors, CureEB, EBMRF, and Stanford University, in Nova Anchora LLC, an early-stage, preclinical biotech company focused on developing novel protein therapies for EB. The investment will support the development of Nova Anchora's lead candidate, NvA-011, through preclinical development and to IND readiness. Nova Anchora, based in San Francisco, was co-founded in 2021 by Prof. Peter Marinkovich, Associate Professor of Dermatology at Stanford Medicine and a leading expert in the EB field. EB is a group of rare and painful genetic skin disorders that cause the skin to become very fragile and tear or blister at the slightest touch. Nova Anchora is focusing on dystrophic EB (DEB), one of the more severe forms of EB, in which blisters form in the dermis layer of the skin due to defective type VII collagen. By leveraging proprietary technology licensed from Stanford University, Nova Anchora is advancing NvA-011, a recombinant human type VII pro-collagen designed for topical application in DEB. In 2024, NvA-011 was granted FDA orphan drug designation. The new funds will be used to further advance the preclinical development of NvA-011 and support IND-enabling studies, including finalizing manufacturing processes and initiating toxicology studies ahead of clinical trials. As the program progresses, Nova Anchora aims to prepare for a larger Series A financing round to support first-in-human clinical trials within the next two years. 'We're thrilled to partner with DEBRA Research and other mission-aligned investors to accelerate the development of our corrective protein therapy for people living with DEB,' said Jason Bhardwaj, CEO of Nova Anchora. 'We are especially excited to benefit from our partners' deep connections across the DEB community and broader strategic ecosystem to help bring our technology to patients.' 'At DEBRA Research, we believe strategic partnerships and targeted investments are key to driving meaningful change for people living with EB,' said Martin Steiner, PhD, Managing Director at DEBRA Research. 'Nova Anchora's innovative protein therapy, designed to restore defective type VII collagen through a targeted, topical approach, represents a promising step forward for DEB. Backed by a team with deep expertise in EB research and translational medicine, the company is well positioned to push this much-needed treatment toward the clinic. This investment unites philanthropic organizations and a professional venture capital investor in a unique syndicate, and we are thrilled to support the company and collaborate in accelerating the development of NvA-011.' CureEB, DEBRA Research, EBMRF, and Viking Global Investors have come together to form an exceptional syndicate for this financing round. Together with EB Research Partnership (EBRP), an investor in previous rounds, the partners are united by a shared commitment to improving the lives of those affected by EB and working towards a cure for this debilitating disease. CureEB, EBMRF and EBRP are dedicated to raising funds for EB research. DEBRA Research focuses on advancing research and clinical development through strategic partnerships and investments. This collaboration reflects the partners' unified effort to support the advancement of EB research and treatment options. About Nova Anchora LLC Nova Anchora is a biopharmaceutical company advancing a corrective protein therapy for DEB, a severe rare skin disease. Built on foundational technology developed by Dr. Peter Marinkovich and licensed from Stanford, our mission is to improve the lives of those affected by DEB. About DEBRA Research DEBRA Research is a non-profit organization dedicated to advancing treatments and cures for EB, a rare and life-limiting genetic condition. As the research arm of DEBRA Austria – a patient organization established in 1995 to support individuals living with EB – DEBRA Research collaborates globally with academia, biotech, pharma, patient organizations, and regulatory bodies to drive innovation. DEBRA Research focuses on translational research and clinical development to address the unmet needs of people living with EB. Its goal is to accelerate the discovery and development of treatments improving quality of life, while striving for a future where EB is curable – a 'world without EB'. For more information, please visit and follow us on LinkedIn. About Viking Global Investors Founded in 1999, Viking Global Investors ('Viking') is a global investment firm with a long-term, fundamental, research-intensive approach to investing. Viking manages over $51 billion of capital across public and private investments. Viking has offices in Stamford, New York, Hong Kong, London, and San Francisco, and is registered as an investment adviser with the U.S. Securities and Exchange Commission. Other EB-Focused Investors CureEB: Cure EB is a charity registered in England and Wales (no 1158672) that is dedicated to finding effective treatments and a cure for all forms of EB with an emphasis on advancing gene modifying technologies. EB Medical Research Foundation (EBMRF): The EBMRF was founded in 1991 with the mission of funding critical research and raising awareness for EB. They are unique in that much of the administrative work is done on a voluntary basis and no salaries are paid, ensuring that funds are donated directly to research projects. EB Research Partnership (EBRP): The largest global funder of research to accelerate treatments and cures for EB. Contacts DEBRA Research Martin Steiner, PhD [email protected] Nova Anchora [email protected] Media Inquiries MC Services Dr. Cora Kaiser, Dr. Johanna Kobler +49 89 210 2280 [email protected]
