Quark was established in 1994 with the aim of creating a groundbreaking technology platform called BiFAR, which is designed to identify novel functional target genes and proteins. This innovative approach involves high throughput gene inactivation combined with microarray detection of the functional genes responsible for the occurrence of phenotypes of interest. With the recent advancements in siRNA technology, Quark's method has become increasingly popular in the industry for functional studies. The company has applied its BiFAR platform in gene discovery programs for serious diseases with significant unmet medical needs, often working with large pharmaceutical partners.

Tags

Nervous System Diseases

Eye Diseases

Otorhinolaryngologic Diseases

Small interfering RNA

Disease domain score

A glimpse into the focused therapeutic areas

Technology Platform

Most used technologies in drug development

Targets

Most frequently developed targets

Disease Domain	Count

Nervous System Diseases	4
Neoplasms	3
Immune System Diseases	2
Endocrinology and Metabolic Disease	1

Top 5 Drug Type	Count

Small interfering RNA	16

Top 5 Target	Count

caspase 2(Caspase-2)	1
p53(Tumor protein p53)	1
Redd1(DNA damage-inducible transcript 4 protein)	1

Drugs associated with Quark Pharmaceuticals, Inc.

Cosdosiran

Target

caspase 2

Mechanism

caspase 2 inhibitors [+2]

Active Org.

Suzhou Ruibo Biological Technology Co Ltd [+2]

Originator Org.

BioSpring

Active Indication

Optic Neuropathy, Ischemic [+3]

Inactive Indication

Optic Atrophy

Drug Highest PhasePhase 3

First Approval Ctry. / Loc.-

First Approval Date-

PF-4523655

Target

Redd1

Mechanism

Redd1 inhibitors [+1]

Active Org.

Pfizer Inc. [+1]

Originator Org.

Silence Therapeutics Plc

Active Indication

Diabetic macular oedema [+1]

Inactive Indication

Age Related Macular Degeneration [+2]

Drug Highest PhasePhase 2

First Approval Ctry. / Loc.-

First Approval Date-

CTi-1

Target-

Mechanism

RNA interference

Active Org.

Quark Pharmaceuticals, Inc.

Originator Org.

Quark Pharmaceuticals, Inc.

Active Indication

Acute Lung Injury [+1]

Inactive Indication-

Drug Highest PhasePreclinical

First Approval Ctry. / Loc.-

First Approval Date-

Clinical Trials associated with Quark Pharmaceuticals, Inc.

NCT04654221 / TerminatedNot Applicable

Evaluation of eGFR, Determined by Cystatin C and Creatinine, and mGFR, Measured by Iohexol Clearance, in Subjects at High Risk for Acute Kidney Injury (AKI) Following Cardiac Surgery

To evaluate the differences between serum cystatin C based estimated glomerular filtration rate (eGFRcys), serum creatinine based eGFR (eGFRcreat) and measured glomerular filtration rate (mGFR) in subjects at high risk for acute kidney injury (AKI) approximately 90 days following cardiac surgery

Start Date21 Feb 2020

Sponsor / Collaborator

Quark Pharmaceuticals, Inc.

NCT03510897 / TerminatedPhase 3

A Randomized, Double-Blind, Placebo Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of QPI-1002 for the Prevention of Major Adverse Kidney Events (MAKE) in Subjects at High Risk for Acute Kidney Injury (AKI) Following Cardiac Surgery

This trial is designed to evaluate QPI-1002 versus placebo for the prevention of Major Adverse Kidney Events (MAKE) in subjects at high risk for acute kidney injury following cardiac surgery. Half of the participants will receive QPI-1002 while the other half will receive placebo.

Start Date29 Jun 2018

Sponsor / Collaborator

Quark Pharmaceuticals, Inc.

CTR20170011 / Suspended

单剂或多剂玻璃体腔注射QPI-1007 治疗急性非动脉炎性前部缺血性视神经病变的随机、双盲、假注射对照的 II/III 期试验

[Translation] A randomized, double-blind, sham-controlled phase II/III trial of single or multiple intravitreal injections of QPI-1007 in the treatment of acute nonarteritic anterior ischemic optic neuropathy

主要目的： 1. 评定近期发作型 NAION 受试者中 QPI-1007 玻璃体内 (IVT) 注射的安全性和耐受性。 2. 确定近期发作型 NAION 受试者中 QPI-1007 玻璃体内 (IVT) 注射对视觉功能的影响。次要目的：评价近期发作型 NAION 受试者中施用 QPI-1007 后视乳头周围视网膜神经纤维层 (pRNFL) 和神经节细胞层的结构性变化。

[Translation]

Main objectives: 1. To assess the safety and tolerability of intravitreal (IVT) injection of QPI-1007 in subjects with recent-onset NAION. 2. To determine the effect of QPI-1007 intravitreal (IVT) injection on visual function in subjects with recent-onset NAION. Secondary objective: To evaluate structural changes in the peripapillary retinal nerve fiber layer (pRNFL) and ganglion cell layer following administration of QPI-1007 in subjects with recent-onset NAION.

Start Date09 Nov 2017

Sponsor / Collaborator

PYRAMID Laboratories, Inc.

[+2]

100 Clinical Results associated with Quark Pharmaceuticals, Inc.

0 Patents (Medical) associated with Quark Pharmaceuticals, Inc.

Literatures (Medical) associated with Quark Pharmaceuticals, Inc.

01 Aug 2014·Nucleic Acid TherapeuticsQ3 · MEDICINE

Toxicological and Pharmacokinetic Properties of QPI-1007, a Chemically Modified Synthetic siRNA Targeting Caspase 2 mRNA, Following Intravitreal Injection

Q3 · MEDICINE

Article

Author: Solano, Elisabeth C. R. ; Kornbrust, Douglas J. ; Beaudry, Amber ; Foy, Jeffrey W.-D. ; Schneider, David J. ; Thompson, James D.

We report the toxicological and pharmacokinetic properties of the synthetic, small interfering RNA (siRNA), QPI-1007, following intravitreal administration. QPI-1007 is a chemically modified siRNA designed to act via the RNA interference (RNAi) pathway to temporarily inhibit expression of the caspase 2 protein and is being developed as a neuroprotectant for the treatment of nonarteritic anterior ischemic optic neuropathy and other optic neuropathies such as glaucoma that result in the death of retinal ganglion cells. The half-life of QPI-1007 in the vitreous and retina/choroid in the Dutch Belted rabbit was about 2 days, and there was no sign of accumulation after repeated administrations at either 2- or 4-week dosing intervals in the rabbit. QPI-1007 was well tolerated in Dutch Belted rabbits following single or repeated intravitreal administrations of up to 11 doses over 9 months. Test-article-related effects were limited to the eyes, with minimal to mild vitreal cellular infiltration being the major finding, which was reversible. In repeated-dose studies, a modest reduction in B-wave amplitude obtained by electroretinography was observed in animals treated with the highest dose level tested (3 mg, which is equivalent to a 12 mg/eye human dose) that was not considered to be clinically meaningful. Administration in the rat of either a single bolus intravenous (i.v.) injection of 100 mg/kg or daily bolus i.v. injections of 75 mg/kg/day for 28 days failed to elicit any macroscopic or microscopic changes, suggesting a low risk for systemic toxicity. QPI-1007 was negative in three genetic toxicity studies. Overall, the nonclinical studies support the further development of QPI-1007.

01 Aug 2012·Nucleic Acid TherapeuticsQ3 · MEDICINE

Toxicological and Pharmacokinetic Properties of Chemically Modified siRNAs Targeting p53 RNA Following Intravenous Administration

Q3 · MEDICINE

Article

Author: Thompson, James D. ; Kornbrust, Douglas J. ; Foy, Jeffrey W.-D. ; Solano, Elisabeth C. R. ; Schneider, David J. ; Feinstein, Elena ; Molitoris, Bruce A. ; Erlich, Shai

We report the toxicological and pharmacokinetic properties of the synthetic, small interfering RNA I5NP following intravenous administration in rodents and nonhuman primates. I5NP is designed to act via the RNA interference (RNAi) pathway to temporarily inhibit expression of the pro-apoptotic protein p53 and is being developed to protect cells from acute ischemia/reperfusion injuries such as acute kidney injury that can occur during major cardiac surgery and delayed graft function that can occur following renal transplantation. Following intravenous administration, I5NP was very rapidly cleared from plasma was distributed predominantly to the kidney, with very low levels in liver and other tissues. Doses of 800 mg/kg I5NP in rodents, and 1,000 mg/kg I5NP in nonhuman primates, were required to elicit adverse effects, which in the monkey were isolated to direct effects on the blood that included a sub-clinical activation of complement and slightly increased clotting times. In the rat, no additional adverse effects were observed with a rat analogue of I5NP, indicating that the effects likely represent class effects of synthetic RNA duplexes rather than toxicity related to the intended pharmacologic activity of I5NP. Taken together, these data support clinical testing of intravenous administration of I5NP for the preservation of renal function following acute ischemia/reperfusion injury.

01 Jan 2004·Journal of Computational BiologyQ4 · BIOLOGY

A local alignment metric for accelerating biosequence database search

Q4 · BIOLOGY

Article

Author: Spiro, Peter A. ; Macura, Natasa

We introduce a metric for local sequence alignments that has utility for accelerating optimal alignment searches without loss of sensitivity. The metric's triangle inequality property permits identification of redundant database entries guaranteed to have optimal alignments to the query sequence that fall below a specified score threshold, thereby permitting comparisons to these entries to be skipped. We prove the existence of the metric for a variety of scoring systems, including the most commonly used ones, and show that a triangle inequality can be established as well for nucleotide-to-protein sequence comparisons. We discuss a database clustering and search strategy that takes advantage of the triangle inequality. The strategy permits moderate but significant acceleration of searches against the widely used "nr" protein database. It also provides a theoretically based method for database clustering in general and provides a standard against which to compare heuristic clustering strategies.

News (Medical) associated with Quark Pharmaceuticals, Inc.

23 Jan 2024

·www.biospace.com

Alkeus Pharmaceuticals Appoints Seemi Khan, M.D., Chief Medical Officer

CAMBRIDGE, Mass., Jan. 23, 2024 (GLOBE NEWSWIRE) -- Alkeus Pharmaceuticals, Inc. today announced that Seemi Khan, M.D., M.P.H., M.B.A., has joined the Company as Chief Medical Officer. “We are thrilled to welcome Dr. Khan to the Alkeus executive team as we pursue our mission to innovate transformative therapies for degenerative eye diseases,” said Leonide Saad, Ph.D., President and CEO at Alkeus. “An accomplished executive, Dr. Khan has a track record in bringing rare disease treatments to patients in need across multiple therapeutic areas.” Prior to joining Alkeus, Dr. Khan served as Chief Medical Officer at Reata Pharmaceuticals through its acquisition by Biogen, where she oversaw all clinical programs from early stage to commercialization. Dr. Khan was instrumental in the approval and launch of the first treatment for Friedreich’s ataxia. Dr. Khan’s experience also includes senior leadership roles at Mitsubishi Tanabe Pharma America, Quark Pharmaceuticals, Abbott and AbbVie. An internist and nephrologist with expertise in rare disease, immunology and rheumatology, she has served as an Assistant Professor of Medicine at Tufts University School of Medicine. She further has received an M.P.H. from Harvard T.H. Chan School of Public Health and an M.B.A. from Brandeis University. "I am excited to join Alkeus, particularly at a time when gildeuretinol has shown promising advancement toward the treatment of Stargardt disease,” Dr. Khan said. “I’m eager to continue building the team to bring significant impact on the lives of individuals confronted with progressive vision loss. My commitment to medical innovation and patient care aligns seamlessly with Alkeus' mission, and I look forward to driving forward this life-changing treatment." About Alkeus Pharmaceuticals Alkeus Pharmaceuticals is a biopharmaceutical company with headquarters in Cambridge, Mass. Co-founded by Leonide Saad, Ph.D. and Ilyas Washington, Ph.D., Alkeus is focused on developing therapies for serious diseases of the eye with high unmet need. Alkeus’ lead candidate, gildeuretinol (ALK-001), is currently being evaluated in clinical trials for the treatment of Stargardt disease and for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). About Gildeuretinol (ALK-001) Gildeuretinol acetate (ALK-001) is a novel molecule created as a specialized form of deuterated vitamin A designed to reduce the dimerization of vitamin A without disrupting vision. In preclinical studies, gildeuretinol decreased vitamin A dimerization to the normal rate seen in unaffected individuals and prevented retinal degeneration and blindness in animals with Stargardt disease. In addition to the TEASE trials, a Phase 3 study of gildeuretinol in 200 patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) has recently been completed and is expected to read out topline data in 2024. Gildeuretinol has received breakthrough therapy designation and orphan drug designation by the U.S. Food and Drug Administration. For further information, please contact: David Setboun, Chief Operating Officer Email: media01@alkeuspharma.com Website:

Phase 3Executive ChangeBreakthrough TherapyOrphan DrugClinical Result

28 Nov 2023

·www.biospace.com

CellChorus Appoints Andrew Last, PhD, to Board of Directors

Dr. Last brings more than three decades worth of experience leading a variety of life sciences organizations HOUSTON, Texas — November 28, 2023 — CellChorus Inc., the leader in applying artificial intelligence to visually evaluate how cells perform and interact over time, announced the appointment of Andrew Last, PhD, to its board of directors. Dr. Last brings more than three decades of experience leading global life sciences businesses to advise the CellChorus team as it grows the TIMING™ franchise. He currently serves as Executive Vice President and Chief Operating Officer of Bio-Rad Laboratories (NYSE: BIO and BIOb), a global leader of life science research and clinical diagnostic products with nearly $3 billion in revenue. Dr. Last previously served as Chief Commercial Officer at Berkeley Lights. Prior to that he was Chief Operating Officer at both Intrexon Corporation and Affymetrix. Dr. Last has also held senior positions with Becton, Dickinson and Company; Applied Biosystems; Incyte Genomics; and Monsanto Company. He holds a PhD with Agrochemical Specialization and an MS in Bio-Aeronautics from Cranfield University, UK, as well as a BS from the University of Leicester, UK. “Andy has had a front seat at some of the most exciting scientific and commercial advances in life sciences over the past several decades,” said Daniel Meyer, CEO of CellChorus. “We are thrilled to have his guidance as CellChorus advances the next frontier of single-cell analysis to provide scientists with a comprehensive understanding of how their cells interact and perform.” About CellChorus CellChorus® is the leader in applying artificial intelligence (AI) to visually evaluate how thousands of individual immune cells, such as T cells and NK cells, perform over time. The company applies Time lapse Imaging Microscopy in Nanowell Grids (TIMING™) with neural network-based AI to identify cells and evaluate their activity, including how they move, activate, kill and survive. The patent-protected CellChorus platform can link TIMING data and insights with information from other analysis modalities such as single-cell RNA sequencing and flow cytometry to provide a comprehensive understanding of cellular function, state and phenotype for the life sciences industry. Please visit cellchorus.com for more information. Company Contact: Daniel Meyer Chief Executive Officer CellChorus Inc. TIMING@cellchorus.com

Executive Change

07 Mar 2023

·www.prnewswire.com

$2.14 Billion Worldwide Antisense & RNAi Therapeutics Industry to 2032: Rising Prevalence of Coronary Artery Diseases, Neurodegenerative Disorders and Infectious Diseases Drives Growth

DUBLIN, March 7, 2023 /PRNewswire/ -- The "Antisense & RNAi Therapeutics Global Market Report 2023" report has been added to ResearchAndMarkets.com's offering. This report provides strategists, marketers and senior management with the critical information they need to assess the market. The global antisense & RNAI therapeutics market is expected to grow from $1.32 billion in 2021 to $1.42 billion in 2022 at a compound annual growth rate (CAGR) of 8%. The antisense & RNAi therapeutics market is expected to reach $2.14 billion in 2026 at a CAGR of 10.8%. Reasons to Purchase Gain a truly global perspective with the most comprehensive report available on this market covering 50+ geographies. Understand how the market has been affected by the coronavirus and how it is responding as the impact of the virus abates. Assess the Russia - Ukraine war's impact on agriculture, energy and mineral commodity supply and its direct and indirect impact on the market. Measure the impact of high global inflation on market growth. Create regional and country strategies on the basis of local data and analysis. Identify growth segments for investment. Outperform competitors using forecast data and the drivers and trends shaping the market. Understand customers based on the latest market shares. Benchmark performance against key competitors. Suitable for supporting your internal and external presentations with reliable high quality data and analysis Major players in the antisense & RNAi therapeutics market are Alnylam Pharmaceuticals, Benitec Biopharma Ltd., Gene Signal, GlaxoSmithKline Plc, Ionis Pharmaceuticals, Marina Biosciences, Quark Pharmaceuticals, Sanofi S.A, Acuitas Therapeutics and Antisense Therapeutics Ltd. The antisense and RNAi therapeutics market includes revenues earned by entities by regulating gene expression at multiple levels, such as at replication, transcription, and translation. The market value includes the value of related goods sold by the service provider or included within the service offering. Only goods and services traded between entities or sold to end consumers are included. Antisense therapy refers to the selective, sequence-specific suppression of gene expression by single-stranded DNA oligonucleotides. Contrarily, RNA interference (RNAi) is activated by double-stranded RNA (dsRNA) and results in the sequence-specific mRNA degradation of single-stranded target RNAs. The main technologies of antisense & RNAi therapeutics are RNA interference and antisense RNA. RNA interference is a conserved biological response to double-stranded RNA that mediates resistance to both endogenous parasitic and exogenous pathogenic nucleic acids and regulates protein-coding gene expression. The various route of administration includes pulmonary delivery, intravenous injections, intra-dermal injections, intraperitoneal injections, topical delivery, and other delivery methods that are used for the indication of oncology, cardiovascular diseases (CVDs), respiratory disorders, neurological disorders, infectious diseases, other. The rising prevalence of coronary artery diseases, neurodegenerative disorders, and infectious diseases is projected to contribute to the demand for the antisense & RNAi therapeutics market. Gene suppression approaches including RNA interference and antisense oligonucleotides are used for the treatment of various neurodegenerative conditions by repairing mutant genes. According to the National Institute of Environmental Health Sciences, Alzheimer's disease and Parkinson's disease are the most common neurodegenerative diseases affecting millions of people globally. In the USA, around 930,000 people had Parkinson's disease by the end of 2020. Furthermore, according to the World Health Organization (WHO), infectious diseases are a major concern and dominated the WHO's list of health threats ranging from various climate change issues to inadequate healthcare facilities. Hence, the growing prevalence of several neurodegenerative diseases and infectious diseases is expected to drive the growth of the antisense & RNAi therapeutics market. The high cost of RNA interference-based drugs is expected to act as a major restraint on the growth of the antisense & RNAi therapeutics market in the future. For instance, according to Future Medicine, the price of Onpattro is approximately $450,000 per year. Onpattro (patisaran) is the RNA interference-based drug used for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. Stringent regulations, a long product approval process, high development cost, and a comparably small number of patients are a few factors leading to the high cost of RNA interference drugs, which is expected to hinder the antisense & RNAi therapeutics market growth in upcoming years. Major companies operating in the antisense & RNAi therapeutics market are undertaking strategic initiatives such as collaborations and partnerships for product innovation to sustain in the increasingly competitive market, companies are developing innovative products as well as sharing skills and expertise with other companies. While companies have long collaborated as well as with academic and research institutions in this market by way of partnerships, and in or out-licensing deals. This trend has been increasing over recent years. The regions covered in the antisense & RNAi therapeutics market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa. The countries covered in the antisense & RNAi therapeutics market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK and USA. Key Topics Covered: 1. Executive Summary 2. Antisense & RNAi Therapeutics Market Characteristics 3. Antisense & RNAi Therapeutics Market Trends And Strategies 4. Antisense & RNAi Therapeutics Market - Macro Economic Scenario 4.1 COVID-19 Impact On Antisense & RNAi Therapeutics Market 4.2 Ukraine-Russia War Impact On Antisense & RNAi Therapeutics Market 4.3 Impact Of High Inflation On Antisense & RNAi Therapeutics Market 5. Antisense & RNAi Therapeutics Market Size And Growth 5.1. Global Antisense & RNAi Therapeutics Historic Market, 2017-2022, $ Billion 5.1.1. Drivers Of The Market 5.1.2. Restraints On The Market 5.2. Global Antisense & RNAi Therapeutics Forecast Market, 2022-2027F, 2032F, $ Billion 5.2.1. Drivers Of The Market 5.2.2. Restraints On the Market 6. Antisense & RNAi Therapeutics Market Segmentation 6.1. Global Antisense & RNAi Therapeutics Market, Segmentation By Technology, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion RNA Interference Antisense RNA 6.2. Global Antisense & RNAi Therapeutics Market, Segmentation By Route of Administration, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Pulmonary Delivery Intravenous Injections Intra-dermal Injections Intraperitoneal Injections Topical Delivery Other Delivery Methods 6.3. Global Antisense & RNAi Therapeutics Market, Segmentation By Indication, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Oncology Cardiovascular Diseases (CVDs) Respiratory Disorders Neurological Disorders Infectious Diseases Other Indications 7. Antisense & RNAi Therapeutics Market Regional And Country Analysis 7.1. Global Antisense & RNAi Therapeutics Market, Split By Region, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion 7.2. Global Antisense & RNAi Therapeutics Market, Split By Country, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion For more information about this report visit About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. Media Contact: Research and Markets Laura Wood, Senior Manager [email protected] For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716 Logo: SOURCE Research and Markets

OligonucleotidesiRNA

100 Deals associated with Quark Pharmaceuticals, Inc.

100 Translational Medicine associated with Quark Pharmaceuticals, Inc.

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Pipeline

Pipeline Snapshot as of 05 Jul 2024

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

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Phase 2 Clinical

Phase 3 Clinical

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Current Projects

Drug(Targets)	Indications	Global Highest Phase

Cosdosiran ( caspase 2 )	Optic Nerve Injuries More	Phase 3
Teprasiran ( p53 )	Acute Kidney Injury More	Phase 3
PF-4523655 ( Redd1 )	Diabetic macular oedema More	Phase 2
QPI-3000R	Hearing Loss More	Preclinical
AHLi-11	Meniere Disease More	Preclinical

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