Cytomegalovirus Retinitis

Pictured: RNA/iStock, Artur Plawgo Based on the significant progress made over the last few decades with RNA therapeutics, RNA editing is widely considered the next generation of promising medicines in this field. RNA therapies have made significant progress over the last few years, with an increasing number of FDA approvals beginning in 1998 with Vitravene for CMV retinitis, followed by Macugen for macular degeneration in 2004 and Spinraza for spinal muscular atrophy in 2016. There have also been multiple siRNA-based drugs, including Onpattro for polyneuropathy of hereditary transthyretin-mediated amyloidosis in 2018. And finally, in 2020, perhaps the most well-known products in the RNA space were introduced: the mRNA-based COVID-19 vaccines. All of these demonstrate the strength of RNA therapies and their potential impact on diseases with high unmet need. RNA therapeutics are indeed elegant approaches to altering RNA and thus protein expression, opening the potential to target a broad array of diseases. The field has seen a renewed and increased interest as reversible changes offer flexibility and RNA approaches introduce therapeutic opportunities that were not accessible before. RNA editing technology was first known and recognized as an interesting approach to treating genetic conditions and reversing disease-causing mutations at the RNA level. RNA editing is a naturally occurring and highly active process that uses the body’s existing capabilities to perform nucleotide changes. Since 2014, when ProQR Therapeutics invented the technique of using oligonucleotides recruiting endogenous adenosine deaminase action on RNA, known as ADAR-mediated editing, the field has rapidly progressed. This growth can be attributed to the rapid progression of knowledge about the technology. Alpha-1 antitrypsin deficiency (AATD) is the first indication that many RNA editing companies have decided to pursue, as it provides the opportunity to address liver and lung symptoms of the disease. Clinical trials for AATD run by both Wave Life Sciences and Korro Bio are planned to begin this year and next. Genetic and Common Disorders There is great excitement in the field about using learnings from decades of oligonucleotide-based drug development, natural RNA editing, and knowledge of biological pathways to make RNA editing technology a compelling approach to target various pathophysiological processes. This offers, for example, the possibility not only of reducing or restoring protein expression but also of modulating protein activity involved in diseases. This application of RNA editing offers the potential to impact both genetic disorders and common conditions, such as metabolic and cardiovascular diseases. ProQR’s approach differentiates RNA editing, as it provides the opportunity to target conditions that have thus far not been treatable with other technologies. Indeed, RNA editing offers the possibility of introducing protective variants, informed by human genetics, that could address or prevent diseases including certain cholestatic or cardiovascular conditions. For example, it has been reported in the literature that an Old Order Amish-enriched variant in a functional B4GALT1 was associated with lower serum LDL-C and lower plasma fibrinogen. This protective variant can be introduced via ADAR RNA editing technology, which has the potential to simultaneously address the two cardiovascular risk factors. Delivery is an important aspect of oligonucleotide base therapeutics. RNA therapies, including RNA editing, have again made great progress and generally use conjugation or lipid nanoparticle approaches. As an example, Alnylam made tremendous progress in its siRNA-based treatments for amyloidosis, with Onpattro in 2018 offering an intravenous treatment once every three weeks. Only four years later, Amvuttra arrived on the market for the same condition but with a subcutaneous 3-month dosing approach. For now, the majority of RNA editing programs are focused on targeting the liver where delivery is relatively de-risked, although progress is also being made in exploring new frontiers such as the central nervous system, as evidenced by the partnerships between Roche and Shape Therapeutics and ProQR and Eli Lilly. In summary, the RNA editing space is making impressive progress. The recent approvals and clinical results demonstrating the potential of RNA therapy to target a broad array of organs are extremely encouraging for RNA editing. Near term, we expect to see further development of the technology, more programs advancing to clinical development, expansion of therapeutic areas addressed, and ultimately, we are hopeful that the next few years will bring considerable progress for patients in need. Gerard Platenburg is a cofounder of ProQR and has served as the company’s chief scientific officer since 2022. Gerard has an extensive background in RNA modulation and orphan drug discovery and development and currently leads ProQR's Innovation unit.

When the drug fomivirsen was approved by the FDA in 1998 for the treatment of cytomegalovirus retinitis in patients with HIV/AIDs, it was hailed as a milestone in drug discovery because it was the first antisense oligonucleotide (ASO)—a class of drugs that works by targeting RNA to alter gene expression and control aberrant proteins in ways that traditional drugs can’t. An additional nine ASOs have since been approved by the FDA. Despite the potential ASOs showed in the early days, this class of drugs has faced several hurdles during its development journey. It has taken years for researchers to optimize the compounds and understand the best gene targets and diseases. After fomivirsen was approved, several ASO clinical trials failed due to insufficient efficacy. I believe conditions are ripe for a resurgence of this important class of drugs. First, technology advances in research and discovery are improving both drug design and target selection. At the same time, the market for ASOs is poised to grow thanks to multiple efforts supporting the development of drugs to treat ultra-rare genetic diseases—an area where ASOs could prove to be ideal first-line treatments. To look into the future of the ASO class, it’s helpful to first understand its history. The method was first described in the late 1970s by two Harvard scientists who used unmodified DNA ASOs to inhibit viral RNA translation in the Rous sarcoma virus. The following decade brought rapid innovations in antisense methods, including improvements in the stability and binding affinity of ASOs, which in turn improved their potency. More recently, scientists working in the ASO field discovered certain advantages when targeting RNA species in the cell nucleus. This led to the discovery of novel targets, including long-noncoding-RNAs, which are responsible for regulating gene transcription and post-translational modifications. During the 2010s, several ASO-class therapeutics were approved by the FDA, including nusinersen for spinal muscular atrophy, and eteplirsen and casimersen for Duchenne muscular dystrophy. Importantly, patents covering some of the key chemical modifications employed to make the most safe and effective ASO compounds expired, encouraging more groups to join the effort to develop antisense therapeutics. Another factor bolstering the development of ASO therapeutics is the push that’s underway to support the development of new therapies to treat rare diseases. Earlier this year, the FDA’s Center for Biologics Evaluation and Research said it was preparing to pilot test a program aimed at rare diseases that’s modeled after Operation Warp Speed, the government’s effort to accelerate the development of Covid-19 vaccines. The hope is to reduce the time and expense of developing genomic therapies to treat rare diseases. Likewise, the Nucleic Acid Therapy Accelerator, a unit of the Medical Research Council, was established with similar goals in the U.K. ASOs could be particularly valuable in targeting rare genetic disorders—a fact that’s not lost on one of the pioneers in the field, Dr. Stanley Crooke, who led the development of ASO therapeutics as Ionis Pharmaceuticals’ CEO. In 2020, he founded the nonprofit n-Lorem Foundation, which is on a mission to develop and deliver ASO therapeutics to patients at no cost. Because more ASOs have been administered to humans than any other class of oligonucleotide drug, there is a greater understanding of the safety profile and risks of these medicines. This could make them ideal as first-line therapies for rare genetic disorders, where extensive testing prior to patient administration is not feasible. The N1C is a collaborative formed from several foundations and institutions with the goal of increasing the pace of development of treatments for rare genetic disorders via a variety of new technologies, including antisense, RNA interference, and CRISPR genome editing. The potential for further growth of ASOs is clear, but hurdles remain. As is the case with all next-generation therapeutics, balancing cellular uptake and potency with minimum off-target effects and toxicity will continue to be a challenge. Researchers working in this field should take advantage of all of the resources available to them—from technology advances to support from regulators and foundations—so they can continue to advance new therapies for patients in need.

NEW YORK, Oct. 6, 2022 /PRNewswire/ -- The cytomegalovirus (CMV) therapeutics market is expected to grow by USD 332.21 million during 2021-2026 according to the latest market research report by Technavio. As per Technavio, the reformulation of drugs will have a positive impact on the market and contribute to its growth significantly over the forecast period. This research report also analyzes other significant trends and market drivers that will influence market growth. Cytomegalovirus (CMV) Therapeutics Market Regional Analysis Continue Reading Technavio has announced its latest market research report titled Global Cytomegalovirus (CMV) Therapeutics Market 2022-2026 North America will account for 37% of market growth. Cytomegalovirus (CMV) treatments are primarily sold in the US and Canada North America. Compared to the rest of the world, this region's market will increase more quickly (ROW). The cytomegalovirus (CMV) therapeutics market will increase in North America over the forecast period due to the high sales of authorized therapies and the considerable prevalence of various kinds of CMV in nations like the US. Request Free Sample Report. Cytomegalovirus (CMV) Therapeutics Market: Segmentation Analysis Route of administration Oral Parenteral Others Geography North America Europe Asia Rest of World (ROW) The oral sector will significantly increase its market share for cytomegalovirus (CMV) therapies during the anticipated period. In the upcoming years, the presence of F. Hoffmann-La Roche and its cutting-edge medication VALCYTE will fuel market expansion in this niche. The US FDA later granted it marketing permission for the prevention of CMV in patients who have undergone kidney, heart, or kidney-pancreas transplantation after it first gained the necessary approval for the treatment of CMV retinitis in patients with acquired AIDS. Buy Sample Report. Market Drivers The expanding risk factors for CMV are one of the major reasons boosting the global cytomegalovirus (CMV) treatments market. CMV is more prevalent in populations with inadequate immunity, such as those living with HIV. Although the incidence rate has decreased, there is still a significant unmet market demand because of the high prevalence, which is thought to affect around 37 million people globally, and the paucity of licensed medicines. The danger of CMV infection is further increased by the rising number of hematopoietic stem cell transplant (HSCT) procedures performed for diverse causes. Another known risk factor for CMV is chemotherapy. During the anticipated period, all of these elements are anticipated to fuel market expansion. Download Free Sample Report. Market Trends Another major market trend for CMV therapies that are anticipated to have a favorable effect on the sector throughout the forecast period is the reformulation of medications. Vendors are creating medications in multiple forms in order to offer treatment options and guarantee the high efficacy of their products. Cytomegalovirus (CMV) Therapeutics Market Vendors Astellas Pharma Inc. AstraZeneca Plc Bausch Health Companies Inc. Chimerix Inc. Clinigen Group Plc Exela Pharma Sciences LLC F. Hoffmann La Roche Ltd. Fortress Biotech Inc. Fresenius SE and Co. KGaA Genentech Inc. Gilead Sciences Inc. Merck and Co. Inc. Related Reports Stem Cell Umbilical Cord Blood (UCB) Market by End-user and Geography - Forecast and Analysis 2022-2026 Lennox-Gastaut Syndrome Treatment Market by Type and Geography - Forecast and Analysis 2022-2026 Table of Contents 1 Executive Summary 1.1 Market Overview Exhibit 01: Executive Summary – Chart on Market Overview Exhibit 02: Executive Summary – Data Table on Market Overview Exhibit 03: Executive Summary – Chart on Global Market Characteristics Exhibit 04: Executive Summary – Chart on Market by Geography Exhibit 05: Executive Summary – Chart on Market Segmentation by Route of Administration Exhibit 06: Executive Summary – Chart on Incremental Growth Exhibit 07: Executive Summary – Data Table on Incremental Growth Exhibit 08: Executive Summary – Chart on Vendor Market Positioning 2 Market Landscape 2.1 Market ecosystem Exhibit 09: Parent market Exhibit 10: Market Characteristics 3 Market Sizing 3.1 Market definition Exhibit 11: Offerings of vendors included in the market definition 3.2 Market segment analysis Exhibit 12: Market segments 3.3 Market size 2021 3.4 Market outlook: Forecast for 2021-2026 Exhibit 13: Chart on Global - Market size and forecast 2021-2026 ($ million) Exhibit 14: Data Table on Global - Market size and forecast 2021-2026 ($ million) Exhibit 15: Chart on Global Market: Year-over-year growth 2021-2026 (%) Exhibit 16: Data Table on Global Market: Year-over-year growth 2021-2026 (%) 4 Five Forces Analysis 4.1 Five forces summary Exhibit 17: Five forces analysis - Comparison between2021 and 2026 4.2 Bargaining power of buyers Exhibit 18: Chart on Bargaining power of buyers – Impact of key factors 2021 and 2026 4.3 Bargaining power of suppliers Exhibit 19: Bargaining power of suppliers – Impact of key factors in 2021 and 2026 4.4 Threat of new entrants Exhibit 20: Threat of new entrants – Impact of key factors in 2021 and 2026 4.5 Threat of substitutes Exhibit 21: Threat of substitutes – Impact of key factors in 2021 and 2026 4.6 Threat of rivalry Exhibit 22: Threat of rivalry – Impact of key factors in 2021 and 2026 4.7 Market condition Exhibit 23: Chart on Market condition - Five forces 2021 and 2026 5 Market Segmentation by Route of Administration 5.1 Market segments Exhibit 24: Chart on Route of Administration - Market share 2021-2026 (%) Exhibit 25: Data Table on Route of Administration - Market share 2021-2026 (%) 5.2 Comparison by Route of Administration Exhibit 26: Chart on Comparison by Route of Administration Exhibit 27: Data Table on Comparison by Route of Administration 5.3 Oral - Market size and forecast 2021-2026 Exhibit 28: Chart on Oral - Market size and forecast 2021-2026 ($ million) Exhibit 29: Data Table on Oral - Market size and forecast 2021-2026 ($ million) Exhibit 30: Chart on Oral - Year-over-year growth 2021-2026 (%) Exhibit 31: Data Table on Oral - Year-over-year growth 2021-2026 (%) 5.4 Parenteral - Market size and forecast 2021-2026 Exhibit 32: Chart on Parenteral - Market size and forecast 2021-2026 ($ million) Exhibit 33: Data Table on Parenteral - Market size and forecast 2021-2026 ($ million) Exhibit 34: Chart on Parenteral - Year-over-year growth 2021-2026 (%) Exhibit 35: Data Table on Parenteral - Year-over-year growth 2021-2026 (%) 5.5 Others - Market size and forecast 2021-2026 Exhibit 36: Chart on Others - Market size and forecast 2021-2026 ($ million) Exhibit 37: Data Table on Others - Market size and forecast 2021-2026 ($ million) Exhibit 38: Chart on Others - Year-over-year growth 2021-2026 (%) Exhibit 39: Data Table on Others - Year-over-year growth 2021-2026 (%) 5.6 Market opportunity by Route of Administration Exhibit 40: Market opportunity by Route of Administration ($ million) 6 Customer Landscape 6.1 Customer landscape overview Exhibit 41: Analysis of price sensitivity, lifecycle, customer purchase basket, adoption rates, and purchase criteria 7 Geographic Landscape 7.1 Geographic segmentation Exhibit 42: Chart on Market share by geography 2021-2026 (%) Exhibit 43: Data Table on Market share by geography 2021-2026 (%) 7.2 Geographic comparison Exhibit 44: Chart on Geographic comparison Exhibit 45: Data Table on Geographic comparison 7.3 North America - Market size and forecast 2021-2026 Exhibit 46: Chart on North America - Market size and forecast 2021-2026 ($ million) Exhibit 47: Data Table on North America - Market size and forecast 2021-2026 ($ million) Exhibit 48: Chart on North America - Year-over-year growth 2021-2026 (%) Exhibit 49: Data Table on North America - Year-over-year growth 2021-2026 (%) 7.4 Europe - Market size and forecast 2021-2026 Exhibit 50: Chart on Europe - Market size and forecast 2021-2026 ($ million) Exhibit 51: Data Table on Europe - Market size and forecast 2021-2026 ($ million) Exhibit 52: Chart on Europe - Year-over-year growth 2021-2026 (%) Exhibit 53: Data Table on Europe - Year-over-year growth 2021-2026 (%) 7.5 Asia - Market size and forecast 2021-2026 Exhibit 54: Chart on Asia - Market size and forecast 2021-2026 ($ million) Exhibit 55: Data Table on Asia - Market size and forecast 2021-2026 ($ million) Exhibit 56: Chart on Asia - Year-over-year growth 2021-2026 (%) Exhibit 57: Data Table on Asia - Year-over-year growth 2021-2026 (%) 7.6 Rest of World (ROW) - Market size and forecast 2021-2026 Exhibit 58: Chart on Rest of World (ROW) - Market size and forecast 2021-2026 ($ million) Exhibit 59: Data Table on Rest of World (ROW) - Market size and forecast 2021-2026 ($ million) Exhibit 60: Chart on Rest of World (ROW) - Year-over-year growth 2021-2026 (%) Exhibit 61: Data Table on Rest of World (ROW) - Year-over-year growth 2021-2026 (%) 7.7 US - Market size and forecast 2021-2026 Exhibit 62: Chart on US - Market size and forecast 2021-2026 ($ million) Exhibit 63: Data Table on US - Market size and forecast 2021-2026 ($ million) Exhibit 64: Chart on US - Year-over-year growth 2021-2026 (%) Exhibit 65: Data Table on US - Year-over-year growth 2021-2026 (%) 7.8 Denmark - Market size and forecast 2021-2026 Exhibit 66: Chart on Denmark - Market size and forecast 2021-2026 ($ million) Exhibit 67: Data Table on Denmark - Market size and forecast 2021-2026 ($ million) Exhibit 68: Chart on Denmark - Year-over-year growth 2021-2026 (%) Exhibit 69: Data Table on Denmark - Year-over-year growth 2021-2026 (%) 7.9 China - Market size and forecast 2021-2026 Exhibit 70: Chart on China - Market size and forecast 2021-2026 ($ million) Exhibit 71: Data Table on China - Market size and forecast 2021-2026 ($ million) Exhibit 72: Chart on China - Year-over-year growth 2021-2026 (%) Exhibit 73: Data Table on China - Year-over-year growth 2021-2026 (%) 7.10 Canada - Market size and forecast 2021-2026 Exhibit 74: Chart on Canada - Market size and forecast 2021-2026 ($ million) Exhibit 75: Data Table on Canada - Market size and forecast 2021-2026 ($ million) Exhibit 76: Chart on Canada - Year-over-year growth 2021-2026 (%) Exhibit 77: Data Table on Canada - Year-over-year growth 2021-2026 (%) 7.11 India - Market size and forecast 2021-2026 Exhibit 78: Chart on India - Market size and forecast 2021-2026 ($ million) Exhibit 79: Data Table on India - Market size and forecast 2021-2026 ($ million) Exhibit 80: Chart on India - Year-over-year growth 2021-2026 (%) Exhibit 81: Data Table on India - Year-over-year growth 2021-2026 (%) 7.12 Market opportunity by geography Exhibit 82: Market opportunity by geography ($ million) 8 Drivers, Challenges, and Trends 8.1 Market drivers 8.2 Market challenges 8.3 Impact of drivers and challenges Exhibit 83: Impact of drivers and challenges in 2021 and 2026 8.4 Market trends 9 Vendor Landscape 9.1 Overview 9.2 Vendor landscape Exhibit 84: Overview on Criticality of inputs and Factors of differentiation 9.3 Landscape disruption Exhibit 85: Overview on factors of disruption 9.4 Industry risks Exhibit 86: Impact of key risks on business 10 Vendor Analysis 10.1 Vendors covered Exhibit 87: Vendors covered 10.2 Market positioning of vendors Exhibit 88: Matrix on vendor position and classification 10.3 AstraZeneca Plc Exhibit 89: AstraZeneca Plc - Overview Exhibit 90: AstraZeneca Plc - Product / Service Exhibit 91: AstraZeneca Plc - Key news Exhibit 92: AstraZeneca Plc - Key offerings 10.4 Bausch Health Companies Inc. Exhibit 93: Bausch Health Companies Inc. - Overview Exhibit 94: Bausch Health Companies Inc. - Business segments Exhibit 95: Bausch Health Companies Inc. - Key news Exhibit 96: Bausch Health Companies Inc. - Key offerings Exhibit 97: Bausch Health Companies Inc. - Segment focus 10.5 Chimerix Inc. Exhibit 98: Chimerix Inc. - Overview Exhibit 99: Chimerix Inc. - Product / Service Exhibit 100: Chimerix Inc. - Key offerings 10.6 Exela Pharma Sciences LLC Exhibit 101: Exela Pharma Sciences LLC - Overview Exhibit 102: Exela Pharma Sciences LLC - Product / Service Exhibit 103: Exela Pharma Sciences LLC - Key offerings 10.7 F. Hoffmann La Roche Ltd. Exhibit 104: F. Hoffmann La Roche Ltd. - Overview Exhibit 105: F. Hoffmann La Roche Ltd. - Business segments Exhibit 106: F. Hoffmann La Roche Ltd. - Key news Exhibit 107: F. Hoffmann La Roche Ltd. - Key offerings Exhibit 108: F. Hoffmann La Roche Ltd. - Segment focus 10.8 Fortress Biotech Inc. Exhibit 109: Fortress Biotech Inc. - Overview Exhibit 110: Fortress Biotech Inc. - Business segments Exhibit 111: Fortress Biotech Inc. - Key offerings Exhibit 112: Fortress Biotech Inc. - Segment focus 10.9 Gilead Sciences Inc. Exhibit 113: Gilead Sciences Inc. - Overview Exhibit 114: Gilead Sciences Inc. - Product / Service Exhibit 115: Gilead Sciences Inc. - Key news Exhibit 116: Gilead Sciences Inc. - Key offerings 10.10 Merck and Co. Inc. Exhibit 117: Merck and Co. Inc. - Overview Exhibit 118: Merck and Co. Inc. - Business segments Exhibit 119: Merck and Co. Inc. - Key news Exhibit 120: Merck and Co. Inc. - Key offerings Exhibit 121: Merck and Co. Inc. - Segment focus 10.11 Takeda Pharmaceutical Co. Ltd. Exhibit 122: Takeda Pharmaceutical Co. Ltd. - Overview Exhibit 123: Takeda Pharmaceutical Co. Ltd. - Product / Service Exhibit 124: Takeda Pharmaceutical Co. Ltd. - Key news Exhibit 125: Takeda Pharmaceutical Co. Ltd. - Key offerings 10.12 Teva Pharmaceutical Industries Ltd. Exhibit 126: Teva Pharmaceutical Industries Ltd. - Overview Exhibit 127: Teva Pharmaceutical Industries Ltd. - Business segments Exhibit 128: Teva Pharmaceutical Industries Ltd. - Key news Exhibit 129: Teva Pharmaceutical Industries Ltd. - Key offerings Exhibit 130: Teva Pharmaceutical Industries Ltd. - Segment focus 11 Appendix 11.1 Scope of the report 11.2 Inclusions and exclusions checklist Exhibit 131: Inclusions checklist Exhibit 132: Exclusions checklist 11.3 Currency conversion rates for US$ Exhibit 133: Currency conversion rates for US$ 11.4 Research methodology Exhibit 134: Research methodology Exhibit 135: Validation techniques employed for market sizing Exhibit 136: Information sources 11.5 List of abbreviations Exhibit 137: List of abbreviations About Technavio Technavio is a leading global technology research and advisory company. Their research and analysis focus on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions. With over 500 specialized analysts, Technavio's report library consists of more than 17,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavio's comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios. Contacts Technavio Research Jesse Maida Media & Marketing Executive US: +1 844 364 1100 UK: +44 203 893 3200 Email: [email protected] Website: SOURCE Technavio