CELL AND GENE THERAPY
Rocket Pharmaceuticals, Inc. | September 21, 2022
Rocket Pharmaceuticals, Inc. a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, and Renovacor, Inc. a biotechnology company focused on delivering innovative precision therapies to improve the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases, today announced a definitive agreement under which Rocket will acquire Renovacor in an all-stock transaction for an implied value of approximately $2.60 per share, based on the volume weighted average trading price of Rocket shares of $15.51 for the 30 trading days through and including Monday, September 19, 2022. The boards of directors of both companies have unanimously approved the transaction, which is currently expected to close by the first quarter of 2023.
“The acquisition of Renovacor aligns with our strategy to expand our leadership position in AAV-based gene therapy for cardiac disease and gives us a perfect opportunity to continue on our mission to transform the lives of heart failure patients through the power of gene therapy. Building on our success in Danon Disease to date, I am particularly excited to expand our cardiology focus and capabilities and address a clear unmet medical need in BAG3-associated dilated cardiomyopathy. By combining Renovacor’s compelling preclinical work with our joint clinical, regulatory and CMC expertise, we believe we will be well-positioned to bring the highest impact gene therapy with the best chance for success to these patients in the most productive and efficient manner possible.”
Gaurav Shah, M.D., Chief Executive Officer of Rocket
Dr. Shah continued, “Given the positive pediatric safety data previously announced from our Phase 1 RP-A501 Danon Disease program, and the upcoming pediatric efficacy data and longer-term adult cohort data we anticipate presenting at the Heart Failure Society of America (HFSA) Scientific Meeting at the end of this month, this strategic acquisition gives us what we believe is the broadest platform in the field to address these devastating rare cardiac diseases. Furthermore, the acquisition will bring to Rocket key personnel, namely a team of leading cardiology drug development experts, critical capabilities, and valuable IP to support continued development of the BAG3 as well as other potential cardiac programs, including a gene therapy research collaboration for arrhythmogenic cardiomyopathy.”
Renovacor’s most advanced program, REN-001, is an AAV-based gene therapy targeting BAG3-associated dilated cardiomyopathy (DCM), a severe form of heart failure. BAG3-DCM represents a significant unmet medical need in a patient population with rapidly progressive cardiac dysfunction in whom no treatments targeting the underlying mechanism of disease exist. Renovacor has deep technical expertise in the development of precision therapies that address genetically driven cardiac diseases. Further, Renovacor is supported by world-class scientific collaborators, a robust intellectual property portfolio and personnel with expertise in BAG3-DCM. These assets and capabilities, all together, represent tremendous value and will enhance Rocket’s leading position in cardiac AAV-based gene therapy.
“Renovacor has made tremendous progress in advancing targeted gene therapies to address the high unmet medical needs of patients living with genetically driven forms of heart disease,” said Magdalene Cook, MD, Chief Executive Officer of Renovacor. “Our experienced team is excited to join Rocket in a shared vision of broadening patient access to precision medicines for cardiovascular disease and addressing common barriers jointly. We look forward to combining the considerable resources and expertise of Renovacor and Rocket in creating a category leader in the precision cardiology field. As a result of this combination, we will be suspending current guidance regarding preclinical and clinical timelines for our programs as we evaluate these items with the Rocket team.”
It is currently anticipated that the transaction will close by the first quarter of 2023, subject to approval by Renovacor and Rocket shareholders, receipt of any required customary regulatory approvals and the satisfaction of other customary closing conditions. RTW Investments, LP, a significant shareholder of both Rocket and Renovacor, has entered into a voting agreement with Renovacor, pursuant to which they have agreed, among other things, and subject to the terms and conditions of the agreement, to vote in favor of the Renovacor acquisition as a Rocket stockholder.
SVB Securities is serving as exclusive financial advisor and Goodwin Procter LLP is serving as legal counsel to Rocket. Wells Fargo Securities is serving as exclusive financial advisor and Troutman Pepper Hamilton Sanders LLP is serving as legal counsel to Renovacor.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition.
Renovacor is a biotechnology company focused on delivering innovative precision therapies to improve the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases. The company’s lead program in BAG3-associated dilated cardiomyopathy (DCM) uses gene transfer technology to address the monogenic cause of this severe form of heart failure. Renovacor’s vision is to bring life-changing therapies to patients living with serious genetic cardiovascular and related diseases, by developing medicines that target the underlying cause of disease and provide a transformative benefit and significant improvement to quality of life.
CELL AND GENE THERAPY
Kriya Therapeutics | November 17, 2022
Kriya Therapeutics, Inc., a fully integrated gene therapy company advancing a broad portfolio of innovative therapeutics announced the acquisition of Redpin Therapeutics, Inc., a privately held biotechnology company developing regulatable gene therapies for intractable diseases of the nervous system. The acquisition serves as the foundation for Kriya’s neurology therapeutic area portfolio, with two lead gene therapy programs focused on epilepsy and trigeminal neuralgia.
Redpin’s proprietary chemogenetics platform can selectively activate or silence disease-causing neurons, while leaving normal functioning cells unaffected. Currently available treatments for neurological disorders typically rely on systemically administered drugs or surgical interventions to address local neuron dysfunction. However, these approaches can have limited efficacy and off-target side effects. In contrast, chemogenetics leverages gene products that are selectively responsive to a chosen small molecule. Redpin’s technology utilizes gene therapy to express engineered ion channels that are responsive to modulation by the FDA-approved anti-smoking drug varenicline to either inhibit specific overactive neurons or stimulate underactive ones.
“Redpin’s innovative chemogenetics platform has the potential to transform the lives of patients suffering from intractable neurological conditions. We are looking forward to integrating Redpin’s platform and pipeline into Kriya’s gene therapy engine as we advance our mission to develop life-changing gene therapies that can address diseases affecting millions of patients around the world.”
Shankar Ramaswamy, M.D., Co-Founder and Chief Executive Officer of Kriya
“Redpin has a built a breakthrough, validated chemogenetics approach that could have significant potential in the targeted treatment of neurological disorders,” said Dr. Elma Hawkins, Redpin Therapeutics’ President, Chief Executive Officer and Co-founder. “Kriya Therapeutics is the perfect company to take this leading ion channel-based platform forward and deliver an effective treatment option for patients. We look forward to working with the Kriya team to progress these unique targeted therapies to patients in need as rapidly as possible.”
Redpin’s investors include 4BIO Capital, Arkin Bio Ventures, Takeda Ventures Inc, New York Ventures and Alexandria Venture Investments.
Epilepsy is a brain disorder that causes recurrent spontaneous seizures. Approximately 3.4 million people in the U.S. suffer from active epilepsy, according to the U.S. Centers for Disease Control and Prevention. Current treatments for epilepsy include medication, neurostimulation and surgery to remove sections of the affected part of the brain. However between 20% to 40% of patients with newly diagnosed epilepsy will become refractory to treatment, and will continue to suffer seizures. Uncontrolled epilepsy can lead to sudden unexpected death in epilepsy (SUDEP).
About Trigeminal Neuralgia
Trigeminal neuralgia also known as Tic Douloureux, is a severe and chronic disorder that affects the trigeminal, or fifth cranial, nerve. TN patients experience progressive intense stabbing facial pain attacks that can become more severe and last longer over time. Approximately 10,000 to 15,000 new cases of TN occur each year in the U.S., according to the National Organization for Rare Disorders. While currently available treatments include anti-seizure medications and surgical procedures, a significant proportion of patients are refractory to these approaches.
About Kriya Therapeutics
Kriya is a fully integrated company pioneering novel technologies and therapeutics in gene therapy. The company aims to revolutionize how gene therapies are designed, developed, and manufactured, improving speed to market and delivering significant reductions in cost. The company is advancing a deep and diversified pipeline of innovative gene therapies in multiple therapeutic area divisions, with current pipeline programs in metabolic disorders, neurology and ophthalmology. Kriya is backed by leading life sciences and technology investors, and has core operations in Silicon Valley, California and Research Triangle Park, North Carolina.
Based in New York City, Redpin Therapeutics is a privately held, preclinical stage gene therapy company developing a proprietary chemogenetics platform for targeted cell therapies to address currently intractable diseases of the central nervous system. Using a powerful and innovative technology that inverts traditional drug development by using gene therapy to target an engineered receptor to any cell type responsible for disease and modulating its function with an already-approved drug. This has the potential to deliver effective treatments for a wide range of currently intractable neurological and psychiatric diseases and disorders. Redpin has a worldwide exclusive license from the Howard Hughes Medical Institute for the therapeutic use of this technology, and the company is working with urgency to apply it to advance important medicines for patients in desperate need of effective therapies.
Ordaōs and NonExomics | September 29, 2022
Ordaōs, a biotechnology company designing novel miniPROTM mini-proteins to help drug hunters deliver life-saving treatments, and NonExomics, a company specializing in the identification of novel targets from non-conventional protein-coding regions, announced a proof-of-concept research collaboration. As part of this endeavor, Ordaōs will use its proprietary design engine to generate and rank novel miniPRO™ candidates that meet specific target product profiles, beginning with three targets. NonExomics will conduct pre-clinical certification of these identified MinPRO™ proteins to demonstrate their promising commercial value.
"This partnership allows us to spotlight the power of our Design Engine to create customized miniPRO™ proteins that are more configurable, penetrable and stable, and easier to manufacture. Our collaboration with NonExomics also allows us the mutual opportunity to underscore the potential of our specialties utilizing machine-driven technology."
David Longo, CEO of Ordaōs
NonExomics identifies whether novel proteins can function as drug targets for therapeutic purposes or can be used for diagnostic processes. The company has developed a technology that effectively combines artificial intelligence, proteomics, transcriptomics, and genomics to identify novel targets for quicker diagnosis and treatment of diseases that are currently considered to be incurable or difficult to manage. NonExomics plans to develop new therapies and diagnostics based on its proprietary nonexomic targets.
"We at NonExomics are thrilled to be embarking on this exciting collaboration between our companies to fuel our shared goal of pursuing and developing new therapies for poorly treated or currently deemed incurable diseases," said Sudhakaran Prabakaran, PhD, CEO and Co-Founder of NonExomics.
To select miniPRO™ candidates, Ordaōs will use its multitask meta-learning Ordaōs Design Engine to generate, appraise, and rank hundreds of thousands of proteins on their structures and drug-like properties for novel miniPRO™ candidates that meet the requirements of the desired specifications of NonExomics' product profile. NonExomics will then conduct a series of pre-clinical demonstrations of the selected Ordaōs miniPRO™ designs to prove they meet the specified product profiles.
More About The Ordaōs Design Engine
Ordaōs uses The Ordaōs Design Engine, to deliver true protein property design - leveraging continuous learning loops and proprietary data sets to translate human-targeted product criteria into machine-designed mini-proteins. Starting with amino acids, the Design Engine generates, appraises, and ranks billions of protein sequences and hundreds of thousands of protein structures and properties to create customized miniPRO™ proteins. These proteins are then rapidly evaluated in vitro to provide intelligent feedback on multiple design objectives including protein structure, binding specificity and affinity, solubility, stability, immunogenicity, and developability. This iterative process delivers optimized mini-proteins to meet the client's specific molecular target product profile (mTPP). They are also less likely to cause adverse side effects and are easier and less expensive to test, develop, and manufacture than traditional proteins. Using this approach, the Ordaōs Design Engine creates more ideal, unseen protein leads than others and can accelerate drug candidate development, increasing the probability of more therapeutically effective candidates. All of this provides clients with a high level of confidence in their investigational new drug (IND) applications.
Ordaōs is a human-enabled, machine-driven drug design company that helps birth novel therapies to reduce patient suffering, improve health, and extend life. Our flagship solution, miniPRO™ mini-proteins, enable drug hunters to deliver safer and more effective treatments in a fraction of the time of traditional discovery methods.
NonExomics science and platform developed over eight years of rigorous academic and industry research, mines novel disease-associated proteins using the dark genome. NonExomics has opened up a new area of science with unlimited possibilities to cure diseases.