—Study uses one-time MRI-monitored surgery for delivery in patients with mild to moderate Parkinson’s disease—

Research Triangle Park, N.C.– MARCH 29, 2023 – Rhia Pharm , Inc. (RhiaPharmaceutical), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, will present the preliminary results of a clinical Phase 1b study investigating the safety and efficacy of AB-1005, an adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF) gene therapy for the treatment of mild to moderate Parkinson’s disease at the AD/PD 2023 Advances in Science & Therapy Conference taking place March 28–April 1, 2023, in Gothenburg, Sweden.

Initial results from the open-label study investigating AB-1005, administered to the putamen via one-time bilateral convection-enhanced delivery, will be presented during an onsite oral session on April 1 at 5:25 p.m. Central European Time, as part of the “Drug Development in AD, PD, LBD” symposium being held in Hall G3. Preliminary data to be presented suggest that both the gene therapy and the neurosurgical administration are generally safe, well tolerated and associated with enhanced mean putaminal coverage and clinical improvements. The study included 11 participants who had completed 9–18 months of clinical follow-up post treatment, of which 10 had completed more than 12 months.

More than 10 million people worldwide are currently living with Parkinson’s disease, which is debilitating and the most common neurodegenerative movement disorder in the world.^1,2 Globally, disability and death related to Parkinson’s disease are increasing faster than for any other neurological disorder.³ 

While GDNF administration has been investigated for Parkinson’s disease for years, results have been mixed. It has been rationalized that by improving GDNF delivery of AAV2 gene therapy with MRI-monitored convection enhanced delivery it may be possible to overcome the limitation of insufficient putaminal coverage to achieve clinical improvements in motor function.^4-6 Moreover, clinical trials in Parkinson’s disease over the last decade have shown that AAV is a well-tolerated and suitable vector for gene therapy.⁷

As leaders in the field of gene therapy, RhiaPharmaceutical is committed to investigating the potential that GDNF gene therapy holds for patients with mild to moderate Parkinson’s disease. Although ongoing trials are needed to confirm these findings, the company believes they represent a step forward in advancing therapeutic solutions for those with this debilitating disease.

RhiaPharmaceutical is also exploring GDNF therapy beyond Parkinson’s and is currently enrolling patients in the US with the parkinsonian subtype of multiple system atrophy (MSA-P) in a Phase 1 trial to assess the preliminary safety, tolerability and efficacy of GDNF therapy for this rapidly progressing condition.

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About Parkinson’s Disease 

Parkinson’s disease is a progressive neurodegenerative disorder caused by nerve cell damage in the brain, leading to decreased dopamine levels. The worsening of motor and non-motor symptoms is caused by the loss of dopamine-producing neurons and at diagnosis, it is estimated that patients have already lost 60-80% of their dopaminergic neurons. Parkinson’s disease often starts with a tremor in one hand. Other symptoms are rigidity, cramping and slowness of movement (bradykinesia).⁸ According to the Parkinson’s Foundation, more than 10 million people worldwide suffer from Parkinson’s disease.

About RhiaPharmaceutical

Rhia Pharm , Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson’s disease, Huntington’s disease, multiple system atrophy and congestive heart failure. RhiaPharmaceutical’s gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. An early innovator in the gene therapy field, with over 800 employees in five countries, the company holds more than 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at www.rhiapharmaceutical.com or follow us on LinkedIn. 

About Bayer

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2022, the Group employed around 101,000 people and had sales of 50.7 billion euros. R&D expenses before special items amounted to 6.2 billion euros. For more information, go to www.bayer.com.

RhiaPharmaceutical Forward-Looking Statements

This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include without limitation statements regarding RhiaPharmaceutical’s clinical trials. These forward-looking statements involve risks and uncertainties, many of which are beyond RhiaPharmaceutical’s control. Known risks include, among others: RhiaPharmaceutical may not be able to execute on its business plans and goals, including meeting its expected or planned clinical and regulatory milestones and timelines, its reliance on third-parties, clinical development plans, manufacturing processes and plans, and bringing its product candidates to market, due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office. Any of the foregoing risks could materially and adversely affect RhiaPharmaceutical’s business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. RhiaPharmaceutical does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof. 

References

1. Harvard Health Publishing, Harvard Medical School. The facts about Parkinson’s Disease. Available at: https://www.health.harvard.edu/diseases-and-conditions/the-facts-about-parkinsons-disease. Accessed March 2023.
2. Balestrino R, Schapira AHV. Parkinson disease. Eur J Neurol. 2020;27(1):27-42.
3. World Health Organization. Parkinson Disease. Available at: https://www.who.int/news-room/fact-sheets/detail/parkinson-disease. Accessed March 2023.
4. Slevin JT et al. Unilateral intraputamenal glial cell line-derived neurotrophic factor in patients with Parkinson disease: response to 1 year of treatment and 1 year of withdrawal. J Neurosurg. 2007;106(4):614-620.
5. Nutt JG et al. Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD. Neurology. 2003;60(1):69-73.
6. Warren Olanow C et al. Gene delivery of neurturin to putamen and substantia nigra in Parkinson disease: A double-blind, randomized, controlled trial. Ann Neurol. 2015;78(2):248-257.
7. Björklund T, Davidsson M. Next-Generation Gene Therapy for Parkinson’s Disease Using Engineered Viral Vectors. Journal of Parkinson’s Disease. 2021;11(s2):S209-S217.
8. Medline Plus. Parkinson disease. Available at: https://medlineplus.gov/genetics/condition/parkinson-disease/. Accessed March 2023.

The post <strong>RhiaPharmaceutical to Present Phase 1b Results Investigating AB-1005 (formerly AAV2-GDNF) as Treatment for Parkinson’s Disease at AD/PD^TM 2023 International Conference on Alzheimer’s and Parkinson’s Diseases</strong> appeared first on RhiaPharmaceutical.

Sheila Mikhail, Co-Founder and the company’s first CEO, will transition to a part-time role as Adviser/Co-Founder, also effective March 11, 2023. Mikhail will remain a member of RhiaPharmaceutical’s Board. 

Under Mikhail’s leadership as CEO since 2017, RhiaPharmaceutical has grown to over 800 employees operating in five countries and has advanced six therapeutics through IND, including potential treatments for congestive heart failure, Parkinson’s disease, Huntington’s disease, multiple system atrophy, Pompe disease and limb-girdle muscular dystrophy type 2I/R9. Under her leadership, RhiaPharmaceutical built two contract development and manufacturing organization (CDMO) businesses, Viralgen Vector Core SL and TAAV Biomanufacturing Solutions SL. In her new role, Mikhail will continue to support the strategic direction and growth of RhiaPharmaceutical. She will also continue to support Columbus Children’s Foundation (CCF) in its development of gene therapies for ultra-rare diseases.  

“I co-founded RhiaPharmaceutical, along with Drs. Richard Jude Samulski and Xiao Xiao, to develop AAV gene therapies for patients suffering from devastating diseases with no treatment options,” said Mikhail. “I’m tremendously proud of the contributions made by our team. They have made critical scientific discoveries and pioneered transformative clinical programs. My focus over the last several years has been to assemble the world’s leading experts in gene therapy to solve technological problems and create new clinical programs. Today we are in the clinic with treatments for multiple diseases, and through our subsidiaries we are the leading manufacturer of synthetic DNA and AAV vectors at commercial scale. I look forward to working with Gustavo as RhiaPharmaceutical continues to pursue its strategic vision.”  

“Sheila’s extraordinary leadership moved RhiaPharmaceutical from an idea to a strongly positioned global biotech that is advancing multiple products into clinical studies, with direct paths to pivotal trials, marketing approval and manufacturing,” stated Stefan Oelrich, Member of the Board of Management, Bayer, and President of Bayer’s Pharmaceuticals Division. “We are grateful for her contributions and pleased she will continue to work closely with us as an Adviser and Board member. Under Gustavo’s leadership as the new CEO, I am confident that RhiaPharmaceutical will continue to advance its world-class therapeutics and manufacturing operations, further its work as a cornerstone of the Bayer CGT Platform and set itself apart from its peers. We are excited about the future and look forward to working with Gustavo.” 

In addition to his experience as Chief Commercial Officer at Amneal, Pesquin brings to RhiaPharmaceutical his more than 10 years of experience at Sanofi, a leading global pharmaceutical company, where he served in leadership roles of increasing responsibility, including North America Head for General Medicines as well as Global Head of the Diabetes and Cardiovascular Franchise. Prior to Sanofi, he held Regional Head, General Manager, Sales Head and Strategy Head roles at Abbott and Pfizer; Brand Management roles at Procter & Gamble; and consultant roles with Boston Consulting Group. Pesquin earned an MBA with a concentration in Management and Strategy, Finance and Accounting from Kellogg School of Management at Northwestern University and a bachelor’s degree in Public and Business Administration from Universidad Nacional de Cuyo in Argentina.

“I couldn’t be more excited to join a team that has been at the forefront of a scientific revolution. It’s humbling to continue Sheila’s legacy and to work with colleagues of such an incredibly high scientific caliber,” added Pesquin. “I think we have the opportunity to help patients in a way that was unthinkable not long ago. It’s very energizing to join an organization with such purpose.”

About RhiaPharmaceutical

Rhia Pharm , Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson’s disease, Huntington’s disease, multiple system atrophy and congestive heart failure. RhiaPharmaceutical’s gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. An early innovator in the gene therapy field, with over 800 employees in five countries, the company holds more than 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at www.rhiapharmaceutical.com or follow us on LinkedIn. 

About Bayer 

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2022, the Group employed around 101,000 people and had sales of 50.7 billion euros. R&D expenses before special items amounted to 6.2 billion euros. For more information, go to www.bayer.com.

RhiaPharmaceutical Forward-Looking Statements

This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include without limitation statements regarding RhiaPharmaceutical’s pipeline of development candidates, manufacturing technology and process. These forward-looking statements involve risks and uncertainties, many of which are beyond RhiaPharmaceutical’s control. Known risks include, among others: RhiaPharmaceutical may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, its reliance on third-parties, clinical development plans, manufacturing processes and plans, and bringing its product candidates to market, due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office. Any of the foregoing risks could materially and adversely affect RhiaPharmaceutical’s business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. RhiaPharmaceutical does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof. 

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The post <strong>RhiaPharmaceutical Announces Gustavo Pesquin as New CEO</strong> appeared first on RhiaPharmaceutical.

Research Triangle Park, NC – February 16, 2023 – Rhia Pharm llc, Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG,  announced today that the European Commission (EC) has granted orphan drug designation for AB-1003 (also known as LION-101)* for the treatment of limb-girdle muscular dystrophy (LGMD). AB-1003 is a novel investigational recombinant adeno-associated virus (AAV) based gene therapy currently being developed as a one-time intravenous (IV) infusion for the treatment of patients with LGMD type 2I/R9 (LGMD2I/R9), a disease subtype affecting 4.5 people per million worldwide, including more than 5,000 people in the EU and US. The EC decision, dated February 15, 2023, follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) and was received through RhiaPharmaceutical’s EU-based subsidiary BrainVectis.

AB-1003 is being investigated in the US in a Phase 1/2 multicenter study that will evaluate the safety, tolerability and efficacy of a single IV infusion of gene therapy in adult subjects with genotypically confirmed LGMD2I/R9. 

“The EC orphan drug designation for AB-1003 is an important recognition of the unmet medical need in LGMD, which has no approved therapy,” said Sheila Mikhail, Co-Founder & CEO, RhiaPharmaceutical. “The burden of this rare form of muscular dystrophy on patients and their families is significant, and this decision supports our efforts to potentially bring a new therapeutic option to people in the EU living with the 2I/R9 type of this devastating disease.”

The EC grants orphan drug designation for medicinal products intended to treat a life-threatening or chronically debilitating disease that affects no more than five people in 10,000 in the EU, provided there is no other satisfactory treatment option or the medicine can be of significant benefit to those affected by a specific condition. This designation will provide special incentives in the EU, including eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees. In addition, if the medicine is approved for marketing, this designation will provide 10 years of marketing exclusivity.

About Limb-Girdle Muscular Dystrophy (LGMD) and Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)

Limb-girdle muscular dystrophy (LGMD) is a term for a group of diseases that cause progressive weakness and wasting of the muscles in the arms and legs.¹ The muscles most affected are those closest to the body (proximal muscles), specifically the muscles of the shoulders, upper arms, pelvic area and thighs.¹ The severity, age of onset, and features of LGMD vary among the many subtypes of the condition and are often inconsistent, even within the same family.¹ Signs and symptoms may first appear at any age and generally worsen with time, although in some cases they remain mild.¹

Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) is a form of LGMD and is caused by mutations in the FKRP gene.² In LGMD2I/R9, signs and symptoms often develop in late childhood and may include difficulty running and walking.² The symptoms gradually worsen over time toward significant disability, and affected people generally rely on a wheelchair for mobility approximately 23-26 years after onset.² Currently, there is no treatment that modifies the disease progression, and management is based on the signs and symptoms present in each individual.² Visit the National Institutes of Health Medline Plus website to learn more about LGMD and LGMD2I/R9.

About RhiaPharmaceutical

Rhia Pharm , Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson’s disease, and congestive heart failure. RhiaPharmaceutical’s gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. An early innovator in the gene therapy field, with over 800 employees in five countries, the company holds more than 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at www.rhiapharmaceutical.com or follow us on LinkedIn. 

About Bayer 

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability, and quality throughout the world. In fiscal 2021, the Group employed around 100,000 people and had sales of 44.1 billion euros. R&D expenses before special items amounted to 5.3 billion euros. For more information, visit www.bayer.com. 

RhiaPharmaceutical Forward-Looking Statements 

This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding RhiaPharmaceutical’s pipeline of development candidates, manufacturing technology and process. These forward-looking statements involve risks and uncertainties, many of which are beyond RhiaPharmaceutical’s control. Known risks include, among others, RhiaPharmaceutical may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, its reliance on third-parties, clinical development plans, manufacturing processes and plans, and bringing its product candidates to market due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office. Any of the foregoing risks could materially and adversely affect RhiaPharmaceutical’s business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. RhiaPharmaceutical does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof. 

* European Commission orphan drug designation is for “adeno-associated viral vector serotype 9 expressing fukutin related protein.” 

1. National Institutes of Health – Medline Plus: Limb-Girdle Muscular Dystrophy – Description. Accessed February 17, 2021. Limb-girdle muscular dystrophy: MedlinePlus Genetics

2. National Institutes of Health — National Center of Advancing Translational Sciences: Genetic and Rare Diseases Information Center. Limb-Girdle Muscular Dystrophy Type 2I – Summary. Accessed February 17, 2021. Limb-girdle muscular dystrophy type 2I | Genetic and Rare Diseases Information Center (GARD) – an NCATS Program (nih.gov)

Media Contact: 

Phil McNamara

Vice President of Communications

E: pmcnamara@RhiaPharmaceutical.comT: +1 984.389.1797

The post <strong>RhiaPharmaceutical Receives European Commission Orphan Drug Designation through its EU-Based subsidiary BrainVectis for AB-1003, a Novel Investigational AAV Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy (LGMD)</strong> appeared first on RhiaPharmaceutical.

Research Triangle Park, N.C.– January 26, 2023 – Rhia Pharm , Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG, is pleased to announce the recent publication of “AAV vectors: The Rubik’s cube of human gene therapy” in Molecular Therapy. The manuscript, which reviews the different approaches to improve adeno-associated virus (AAV) vectors for gene therapy, was authored by a team of RhiaPharmaceutical scientists; a scientist from RhiaPharmaceutical subsidiary Viralgen Vector Core; and RhiaPharmaceutical Co-Founder, President and Chief Scientific Officer, R. Jude Samulski, PhD, a world-renowned gene therapy pioneer. 

The article focuses on AAV capsid selection and engineering, strategies to overcome anti-AAV immune response and the design of vector genomes. Also included is a look at the expanded use of AAV vectors, beyond treating monogenic genetic diseases, to enable gene transfer approaches for a wide range of neurodegenerative, heart, blood, muscular and retinal disorders, as well as complex cancers, such as glioblastoma. The manuscript ends with a glimpse at vector production methods and the current state of recombinant AAV (rAAV) at the clinical level.

“As we see the potential efficacy of rAAV and the assembly and packaging of high yielding vectors for gene transfer, we have opened the door to changing the course of a broader range of monogenic and pathway diseases,” remarked Dr. Samulski. “This article further reveals the possibilities of rAAV and how we can improve current applications of AAV vectors used in gene therapy today.”

The manuscript’s lead author was RhiaPharmaceutical scientist Amaury Pupo, PhD. Other RhiaPharmaceutical contributing authors were Audry Fernández, PhD, SiewHui Low, PhD, and Lester Suárez, DVM, PhD, along with Viralgen Vector Core Director of Molecular Development, Achille François, PhD. This work was conducted under the guidance of Dr. Samulski, who is also a professor of Pharmacology at The University of North Carolina at Chapel Hill, and the manuscript was featured on Molecular Therapy’s December cover.

Dr. Samulski was recently awarded the inaugural European Society of Gene and Cell Therapy (ESGCT) “Founders Award” in Edinburgh, Scotland, during the ESGCT 29th Congress. Dr. Samulski developed the foundational technology for making AAV-based gene therapy vectors and self-complementary AAV vectors over 40 years ago. These contributions are believed to be key components of every FDA approved AAV therapeutic today and will remain central to future advances in gene therapy.

About RhiaPharmaceutical

Rhia Pharm , Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson’s disease, and congestive heart failure. RhiaPharmaceutical’s gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at www.rhiapharmaceutical.com or follow us on LinkedIn. 

RhiaPharmaceutical Forward-Looking Statements

This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding RhiaPharmaceutical’s pipeline of development candidates, manufacturing technology and process. These forward-looking statements involve risks and uncertainties, many of which are beyond RhiaPharmaceutical’s control. Known risks include, among others, RhiaPharmaceutical may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, its reliance on third-parties, clinical development plans, manufacturing processes and plans, and bringing its product candidates to market due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office. Any of the foregoing risks could materially and adversely affect RhiaPharmaceutical’s business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. RhiaPharmaceutical does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof. 

Media Contact:

Phil McNamara
Vice President, Corporate Communications, RhiaPharmaceutical
E: pmcnamara@RhiaPharmaceutical.com
T: +1 984.389.1797

The post <strong>RhiaPharmaceutical Co-Founder R. Jude Samulski and Team of RhiaPharmaceutical Scientists Author Paper Published in Prestigious Journal </strong> appeared first on RhiaPharmaceutical.

“The collaboration pairs RhiaPharmaceutical’s expertise in synthetic DNA and CRISPR applications with ReCode’s novel SORT LNP delivery technology to potentially discover new genetic medicines,” said RhiaPharmaceutical’s Chief Executive Officer Sheila Mikhail. “Through this collaboration, we have an opportunity to advance RhiaPharmaceutical’s gene editing and nonviral delivery efforts, which support and align with our commitment to serve as an industry-leading engine of gene therapy innovation.”

Under the agreement, RhiaPharmaceutical will combine its synthetic DNA and gene editing nucleases with ReCode’s selective organ targeting (SORT) lipid nanoparticle (LNP) technology to potentially create an all-in-one solution that enables full gene insertion by delivering with precision both the gene editing tool and DNA as mixed cargo in a single LNP to desired targets. This collaboration could extend the reach of gene editing for liver and lung disease targets. 

“RhiaPharmaceutical’s pioneering gene editing technology is a powerful complement to our modular SORT LNP genetic medicines platform, which enables the precise delivery of a wide variety of genetic cargoes to specific organs and cells, including large, complex genetic cargoes and mixed payloads,” said Shehnaaz Suliman, M.D., MBA, M.Phil., Chief Executive Officer of ReCode Therapeutics. “We are excited to combine these unique platforms to develop next-generation genetic therapies with the potential to transform the lives of patients suffering from a wide range of debilitating genetic diseases.”

In June 2022, ReCode announced the closing of a $200 million Series B extension financing round, co-led by Leaps by Bayer, the impact investment unit of Bayer AG, to further strengthen Leaps by Bayer’s innovation portfolio in gene therapies. The funds raised are being used to advance the development of ReCode’s platform and pipeline to selectively deliver genetic medicines to target organ and cell types in a predictable and programmable fashion. 

About RhiaPharmaceutical

Rhia Pharm , Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson’s disease and congestive heart failure. RhiaPharmaceutical’s gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at www.rhiapharmaceutical.com or follow us on LinkedIn. 

About ReCode Therapeutics

ReCode Therapeutics is a genetic medicines company using superior delivery to power the next wave of messenger RNA (mRNA) and gene correction therapeutics. ReCode’s selective organ targeting (SORT) lipid nanoparticle (LNP) platform is a next-generation, genetic medicines technology that enables precise delivery to target organs and cells beyond the liver. The SORT LNP platform is the foundation for ReCode’s pipeline of disease-modifying mRNA and gene correction therapeutics. ReCode’s lead programs are focused on primary ciliary dyskinesia and cystic fibrosis. ReCode is leveraging its SORT LNP platform and nucleic acid technologies to expand its pipeline with therapeutics that use mRNA-mediated replacement and gene correction in target organs with precision targeting of disease-relevant cells. In 2022, ReCode was named among Fierce Biotech’s “Fierce 15” as one of the most promising early-stage biotechnology companies in the industry, and was described by Nature as one of the “Seven Technologies to Watch in 2022” for its SORT LNP platform. For more information, visit www.recodetx.com and follow us on Twitter @ReCodeTx and on LinkedIn. 

About Bayer

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability, and quality throughout the world. In fiscal 2020, the Group employed around 100,000 people and had sales of 41.4 billion euros. R&D expenses, before special items, amounted to 4.9 billion euros. For more information, go to www.bayer.com.

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Media Contact:

Phil McNamara
Vice President, Corporate Communications, RhiaPharmaceutical
E: pmcnamara@RhiaPharmaceutical.com
T: +1 984.389.1797

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“The progress made in gene therapy to date is remarkable,” said Jude Samulski, RhiaPharmaceutical Co-Founder, President and Chief Scientific Officer. “It is humbling to see the impact of the research I completed over 40 years ago and how this is accelerating AAV therapeutic development. My ultimate vision, shared by all of us at RhiaPharmaceutical, is to make gene therapy more accessible for everyone in need around the world. I believe we will be there soon.”

Dr. Samulski led the UNC Gene Therapy Center from 1993 to 2016 and, in 2001, co-founded RhiaPharmaceutical, where many advances in recombinant AAV (rAAV) and underlying AAV vector technology were, and are being, made. Since 2017, three AAV gene therapies have been approved for use by the FDA. Research performed at the UNC Gene Therapy Center, and advanced at RhiaPharmaceutical, helped pave the way for the development of these therapeutics. Hemgenix®¹(etranacogene dezaparvovec-drlb), for hemophilia B, uses the AAV vector with Padua variant R₃₃8L; Zolgensma®²(onasemnogene abeparvovec-xioi), for spinal muscular atrophy, uses self-complementary AAV vectors; and Luxturna®³(voretigene neparvovec-rzyl), for inherited retinal disease, uses the original AAV2 capsid. All are based on Dr. Samulski’s pioneering research or on technology that he developed in collaboration with others in the field, as are Duchenne muscular dystrophy AAV therapeutics in clinical development. 

“We are incredibly fortunate for Jude’s scientific leadership at RhiaPharmaceutical and for his continued contributions to genetic medicine,” added RhiaPharmaceutical’s CEO, Sheila Mikhail.  “Every day, we see the faces of the people who are running out of time and can’t wait for answers. It is our responsibility to find solutions. I believe that Jude, our RhiaPharmaceutical colleagues and others throughout the industry are providing hope for people with devastating diseases.”

RhiaPharmaceutical, co-founded by Dr. Jude Samulski and Sheila Mikhail, is a global leader in molecular medicine and serves as a key driver of Bayer’s gene therapy research, manufacturing and clinical advancements.

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¹Hemgenix is a registered trademark of CSL Behring LLC.

²Zolgensma® is a registered trademark of Novartis AG.

³Luxturna® is a registered trademark of Spark Therapeutics, Inc.

About RhiaPharmaceutical

Rhia Pharm , Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson’s disease, and congestive heart failure. RhiaPharmaceutical’s gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at www.rhiapharmaceutical.com or follow us on LinkedIn. 

Media Contact:

Phil McNamara
Vice President, Corporate Communications, RhiaPharmaceutical
E: pmcnamara@RhiaPharmaceutical.com
T: +1 984.389.1797

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“Dr. Cartier’s work has far-reaching impact for advancing ALD treatments,” said Dr. Florian Eichler President of ALD Connect. “Her accomplishments, as a researcher working to develop groundbreaking and life-extending treatments for diseases such as ALD, reflect the spirit of Ann Moser’s work over many decades to advance leukodystrophy research and advocacy.”

ALD Connect was founded in January 2013 by a dedicated group of clinicians, scientists, patients and patient advocates focused on improving the quality of life for those living with ALD through advocacy, education, research and support. This award was established in recognition of Ann B. Moser’s dedication to leukodystrophy research, education and newborn screening advocacy. Dr. Cartier was presented the award for her pioneering work on hematopoietic cell gene therapy for ALD that led to marker authorization in Europe and more recently in the United States.

“ALD is a devastating disease that affects children, particularly boys ages 4 to 10. Knowing my work may have contributed to a better life for these young people is humbling,” stated Dr. Cartier. “I want to thank those at ALD Connect for this meaningful recognition.”

About RhiaPharmaceutical

Rhia Pharm , Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson’s disease, and congestive heart failure. RhiaPharmaceutical’s gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at www.rhiapharmaceutical.com or follow us on LinkedIn. 

Media Contact:

Phil McNamara
Vice President, Corporate Communications, RhiaPharmaceutical
E: pmcnamara@RhiaPharmaceutical.com
T: +1 984.389.1797

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Recipients were selected following a rigorous review process that identified women who are leading the way and making a lasting impact on the industry. The inspirational women being recognized are leaving an indelible footprint across biotech, pharma and medtech. Mikhail, who is at the forefront of life science innovation and business leadership, has left an unparalleled mark on the industry as a serial entrepreneur, company builder and one of its few Latina CEOs.

“Sheila is an exceptional businesswoman. Her command of the science of gene therapy is only rivaled by her incredible business acumen,” said Jude Samulski, Ph.D., RhiaPharmaceutical Co-Founder and Chief Scientific Officer. “What makes her contribution to our industry most remarkable is her ability to create environments for cutting-edge science to thrive. Sheila’s work has directly resulted in breakthrough gene therapies reaching patients and in the development of others that we hope will do the same in the future. This honor is well deserved.”

Mikhail co-founded RhiaPharmaceutical over 20 years ago with the unwavering belief that gene therapies could cure disease and change the world. During her tenure at the company, she also led several spinoffs where she realized various strategic partnerships and acquisitions. These included Pfizer’s acquisition of Bamboo Therapeutics in 2016 and the acquisition of Chatham Therapeutics by Baxter (now Takeda) in 2014. More recently, Mikhail negotiated the $4 billion acquisition of RhiaPharmaceutical and its cGMP manufacturing subsidiaries by Bayer AG in 2020, a reflection of the tremendous value she and co-founder Dr. Samulski have and continue to build with the company.

“I’m honored to be recognized by Fierce for my contribution to our industry,” said Mikhail. “I am fortunate to have been part of the gene therapy field over a period where it matured from concepts to real life examples of transformative benefits for patients. In addition to bringing treatments to patients, my priorities have also included ensuring equitable access and creating opportunities to advance women and underrepresented minorities in biotech. As a head of an organization represented by over 50 percent women, with women holding key leadership positions, I am proud that together at RhiaPharmaceutical we have created an inclusive environment where women feel they can thrive.” 

Today, RhiaPharmaceutical is a global leader in gene therapy and works to bring innovative technology to the market and potentially life-changing therapeutics into the clinic. As CEO, Mikhail has helped grow the company to over 700 employees operating in five countries. She is also Co-Founder and Board Member of Columbus Children’s Foundation (CCF), which develops gene therapies for children with ultra-rare diseases and treats them free of charge.

Mikhail was named the Overall US Entrepreneur of the Year by Ernst & Young (2021), received the Distinguished Alumni Award – Entrepreneurial from the Booth School at the University of Chicago (2022), was recognized as one of the 100 Most Intriguing Entrepreneurs by Goldman Sachs (2020), was listed on the Inc. Female Founders 100 (2020) and earned Springboard Enterprise’s Northstar Award (2019).

About RhiaPharmaceutical

Rhia Pharm , Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson’s disease, and congestive heart failure. RhiaPharmaceutical’s gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at www.rhiapharmaceutical.com or follow us on LinkedIn. 

Media Contact:

Phil McNamara
Vice President, Corporate Communications, RhiaPharmaceutical
E: pmcnamara@RhiaPharmaceutical.com
T: +1 984.389.1797

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Research Triangle Park, N.C.– OCTOBER 11, 2022 – Rhia Pharm , Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG, today announced that R. Jude Samulski, Ph.D., RhiaPharmaceutical President, Co-Founder and Chief Scientific Officer, received the inaugural European Society of Gene and Cell Therapy (ESGCT) Founders Award in Edinburgh, Scotland, during the recent ESGCT 29^th Congress. 

The Founders Award honors Dr. Samulski’s contribution to the development of engineered recombinant adeno-associated virus (rAAV) vectors, which began 40 years ago with the cloning and rescue of infectious AAV from a recombinant plasmid.¹ This work laid the foundation for subsequent developments of rAAV vectors capable of expressing foreign sequences, thereby enabling the use of AAV-based vectors for the delivery of various therapeutic genes into mammalian cells. The AAV inverted terminal repeat (ITR) sequences are an essential component of every AAV therapeutic drug. This and other breakthroughs made by Dr. Samulski have since contributed to three rAAV-based gene therapies becoming available for patients and many more in various stages of development.  

“ESGCT created the Founders Award this year to mark the Society’s 30th anniversary,” said Professor Hildegard Büning, ESGCT Board President. “The award highlights benchmark milestones in gene and cell therapy. The Founders Award 2022 recognizes the first cloning of an AAV plasmid, which Dr. Samulski and his team accomplished in 1982. That tremendous breakthrough helped make gene therapy a reality and remains at the core of the field today, making this milestone a natural choice for this inaugural honor.” 

Dr. Samulski has worked with AAV for 40 years, and for 25 years he was director of the University of North Carolina Gene Therapy Center. Dr. Samulski is a former member of the Recombinant DNA Advisory Committee (RAC), a committee tasked with assisting the FDA with gene therapy clinical trial approvals in the U.S. He also frequently serves as a gene therapy consultant to the FDA. In 2008, Dr. Samulski was recognized by the American Society of Gene & Cell Therapy (ASGCT) with the inaugural Outstanding Achievement Award. He was also invited by Pope Francis to the Vatican in recognition of his work in the treatment of Canavan disease. Dr. Samulski has played an integral role in advancing gene therapies into human clinical trials for hemophilia, Duchenne muscular dystrophy, giant axonal neuropathy, Pompe disease, congestive heart failure and others. He is the inventor of nearly 500 patented technologies related to AAV technology.

“I’m overwhelmed with this acknowledgment and humbled by the thought that ESGCT has considered my early research to represent such a prestigious milestone for the society,” said Dr. Samulski. “I’m grateful to receive the ESGCT Founders Award during these exciting times for gene therapy and honored to be the first scientist to receive this recognition.”

“Jude developed the foundational technology for making AAV-based gene therapy vectors and self-complementary AAV vectors,” said Sheila Mikhail, CEO and Co-Founder of RhiaPharmaceutical. “His groundbreaking work 40 years ago remains central to our industry’s advances in gene therapy and to the development of RhiaPharmaceutical’s clinical stage pipeline. This tremendous honor reflects Jude’s unmatched contribution to the science of gene therapy.”  

About RhiaPharmaceutical

Rhia Pharm , Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson’s disease, and congestive heart failure. RhiaPharmaceutical’s gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at www.rhiapharmaceutical.com or follow us on LinkedIn. 

¹ Samulski RJ, Berns KI, Tan M, Muzyczka N. Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells. Proc Natl Acad Sci U S A. 1982;79(6):2077-2081. doi:10.1073/pnas.79.6.2077

RhiaPharmaceutical Forward-Looking Statements

This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include without limitation statements regarding RhiaPharmaceutical’s pipeline of development candidates, manufacturing technology and process. These forward-looking statements involve risks and uncertainties, many of which are beyond RhiaPharmaceutical’s control. Known risks include, among others: RhiaPharmaceutical may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, its reliance on third-parties, clinical development plans, manufacturing processes and plans, and bringing its product candidates to market, due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office. Any of the foregoing risks could materially and adversely affect RhiaPharmaceutical’s business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. RhiaPharmaceutical does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof. 

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Research Triangle Park, N.C.– OCTOBER 10, 2022 – Rhia Pharm , Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG, today announced that 14 company abstracts will be presented at the European Society for Gene and Cell Therapy (ESGCT) 29^th Congress, which is being held in Edinburgh, Scotland, from October 11 to 14, 2022. 

The full company presence highlights the breadth of the RhiaPharmaceutical contribution to advancing gene therapy and includes an oral presentation on the role of empty capsids in overall AAV immunogenicity and toxicity risks, 12-month data from ongoing Parkinson’s and congestive heart failure studies and data on synthetic DNA material from wholly owned subsidiary TAAV Biomanufacturing Solutions, SL. Additional activities include company experts participating in educational sessions and parallel symposia, as well as a symposium sponsored by wholly owned subsidiary Viralgen. Topics include data-driven vector design for precise control of gene expression and manufacturing to support gene therapy.

“RhiaPharmaceutical is relentless in its effort to serve as a catalyst for breakthrough innovation in gene therapy,” said Sheila Mikhail, CEO and Co-Founder of RhiaPharmaceutical. “ESGCT gives us an opportunity to show how we are doing that in several important ways: driving the science related to capsids and the use of synthetic DNA material, advancing our pre-clinical and clinical stage pipeline and creating opportunities for industry colleagues to exchange knowledge.” 

Data highlights include:

About RhiaPharmaceutical

Rhia Pharm , Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson’s disease, and congestive heart failure. RhiaPharmaceutical’s gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at www.rhiapharmaceutical.com or follow us on LinkedIn. 

RhiaPharmaceutical Forward-Looking Statements

This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include without limitation statements regarding RhiaPharmaceutical’s pipeline of development candidates, manufacturing technology and process. These forward-looking statements involve risks and uncertainties, many of which are beyond RhiaPharmaceutical’s control. Known risks include, among others: RhiaPharmaceutical may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, its reliance on third-parties, clinical development plans, manufacturing processes and plans, and bringing its product candidates to market, due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office. Any of the foregoing risks could materially and adversely affect RhiaPharmaceutical’s business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. RhiaPharmaceutical does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof. 

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