A Phase 1 Open-Label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered VP-001 in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy

A Phase 1 Open-Label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered VP-001 in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy

开始日期2024-06-01

申办/合作机构

PYC Therapeutics Ltd.

NCT05902962 / Recruiting临床1期

A Phase 1 Open-Label, Single Arm Dose Escalation Study to Evaluate the Safety and Tolerability of Intravitreally Administered VP-001 in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy

A Phase 1 Open-Label, Single Arm Dose Escalation Study to Evaluate the Safety and Tolerability of Intravitreally Administered VP-001 in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy

开始日期2023-04-20

申办/合作机构

PYC Therapeutics Ltd.

100 项与 VP-001 (PYC Therapeutics) 相关的临床结果

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100 项与 VP-001 (PYC Therapeutics) 相关的转化医学

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100 项与 VP-001 (PYC Therapeutics) 相关的专利（医药）

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项与 VP-001 (PYC Therapeutics) 相关的文献（医药）

2020-07-01·Acta tropica3区 · 医学

Diagnosis of Taeniosis in rural Venezuelan communities: Preliminary characterization of a Taenia solium specific monoclonal (VP-1) Coproantigen ELISA

3区 · 医学

Article

作者: Rojas R, Glenda ; Aguilar, Cruz Manuel ; Parkhouse, R Michael E ; Cortez Alcovedes, Maria M ; Ferrer, Elizabeth ; Medina, Claudio

The objective of this study was to identify and treat carriers of adult Taenia solium present in two rural Venezuelan communities through examination of faecal samples by coproscopical analysis, and by the application of a polyclonal and a monoclonal (VP-1) coproantigen ELISA. Both the polyclonal and monoclonal ELISA's were negative when tested with soluble extracts of adults of Ascaris lumbricoides, Hymenolepis nana and Trichuris trichura. The polyclonal ELISA was positive for soluble extracts adults of T. solium and T. saginata, whereas the monoclonal ELISA, which recognizes a glycoprotein, was restricted to T. solium, and was also negative with faecal samples from five cases of T. saginata adult infections. In the first community studied, Potrero Largo (Total population: 300), of 248 faecal samples examined, 2 individuals were positive for Taenia spp eggs by coproscopical analysis and the VP-1 ELISA, and yielded T. solium adults upon purging. In contrast, when the polyclonal coproAg ELISA was applied to the same 248 faecal samples, there were a considerable number of positives. Indeed, seven patients highly positive in the polyclonal ELISA did not yield a Taenia spp upon purging and were negative in the VP-1 ELISA. In the second community studied La Yuca (Total population 560), none of the 333 individuals who donated faeces was positive for Taenia spp eggs. Many, however, were infected with a range of intestinal helminth and protozoan parasites. A total of 76 faecal samples with representative intestinal parasite were then tested in the polyclonal and VP-1 assays. Of these, many gave an unacceptable number of significant optical densities in the polyclonal coproAg ELISA. In contrast, all were negative in the VP-1 ELISA, thus providing evidence for the species specificity of the VP-1 ELISA in faecal samples. These results with the VP-1 coproAg ELISA, although preliminary, justify further validation through the testing of more faecal samples from T. solium and T. saginata adult infected individuals.

2015-03-01·Meat science1区 · 农林科学

Effects of vacuum packaging conditions on the quality, biochemical changes and the durability of ostrich meat

1区 · 农林科学

Article

作者: Brenesselová, Martina ; Mačanga, Ján ; Pipová, Monika ; Jevinová, Pavlína ; Turek, Peter ; Marcinčák, Slavomír ; Koréneková, Beáta

In this study, the effects of vacuum packaging conditions on the concentrations of lactic acid, malondialdehyde, pH, microbial and sensory analysis were determined during chilled storage of ostrich meat. Meat was packed as follows: vacuum packed from 1st day (VP-1), vacuum packed from 3rd day (VP-3) and non-vacuum packed (NVP). Analysis were performed at 1st, 7th, 14th, 21st day after slaughter. Meat consisted of 74.69% water, 2.29% fats, 20.95% proteins. Package conditions had significant effect on the pH (NVP: 6.54 on the 14th day, VP-1: 6.05 and VP-3: 6.07 on the 21th day p<0.001), amount of malondialdehyde (NVP: 8.62mg/kg on the 14th day; VP-1: 1.95 and VP-3: 2.55 on the 21th day; p<0.001) and total microbial count (NVP: 7.4 log CFU/g on the 14th day; VP-1: 6.7 and VP-3: 6.8 on the 21th day p<0.01). Based on these results we can assess that vacuum packed from 1st day is necessary for the 21 days storage of ostrich meat.

2015-01-01·The Journal of thoracic and cardiovascular surgery

A new spin on acquired von Willebrand factor deficiency during continuous-flow left ventricular assist device support

Communications

作者: Toole, John M

33. Tsai HM, Sussman II, Ginsburg D, Lankhof H, Sixma JJ, Nagel RL. Proteolytic cleavage of recombinant type 2A von Willebrand factor mutants R834W and R834Q: inhibition by doxycycline and by monoclonal antibody VP-1. Blood. 1997;89:1954-62. 34. Kircik LH. Doxycycline and minocycline for the management of acne: a review of efficacy and safety with emphasis on clinical implications. J Drugs Dermatol. 2010;9:1407-11.

项与 VP-001 (PYC Therapeutics) 相关的新闻（医药）

2023-08-11

·PRNewswire

US FDA DESIGNATES PYC'S LEAD AS A FAST TRACK DEVELOPMENT PROGRAM

PYC is a clinical-stage biotechnology company developing a pipeline of first-in-class precision medicines for patients who have genetic diseases and no treatment options available The Company is currently evaluating the safety/tolerability and efficacy of an investigational drug candidate with disease-modifying potential in patients with a blinding eye disease of childhood called Retinitis Pigmentosa type 11 (RP11) for which there are no existing treatments PYC's drug candidate for the treatment of RP11 has today been designated a Fast Track development program by the US Food and Drug Administration (FDA) The purpose of the Fast Track designation is described by the FDA as being 'to get important new drugs to the patient earlier'[1] This drug candidate is now eligible for Accelerated Approval and Priority Review along with a range of other benefits[2] PERTH, Australia and SAN FRANCISCO, Aug. 11, 2023 /PRNewswire/ -- PYC Therapeutics Limited (ASX:PYC) today announces that the VP-001 program, the first investigational drug candidate designed to address Retinitis Pigmentosa type 11 ( RP11) to progress to human trials, has received Fast Track designation from the US Food and Drug Administration ( FDA). The Fast Track process is "designed to facilitate the development, and the review of drugs to treat serious conditions and fill an unmet medical need" with Fast Track status "often leading to earlier drug approval and access by patients"[3]. Benefits of the Fast Track designation include: Increased frequency of meetings with the FDA to discuss the drug's development plan; Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met; and The potential for a Rolling Review in support of a New Drug Application[4]. PYC will utilise the Fast Track designation to work with the FDA on advancing this important drug program towards patients on an accelerated timeline. PYC's CEO Dr Rohan Hockings commented: "We welcome the opportunity to work with the FDA to progress this important drug candidate towards becoming the first treatment option available for patients with RP11 with disease-modifying potential" About VP-001 – the first potential treatment for Retinitis Pigmentosa Type 11 RP11 is a blinding eye disease that begins in childhood and ultimately leads to legal blindness in middle age. The disease affects ~1 in every 100,000 people and is caused by insufficient expression of the PRPF31 gene in the retina. There are currently no treatment options available for patients with RP11 nor are there any in clinical development. VP-001 is a precision therapy that aims to restore the expression of the PRPF31 gene back to levels required for the normal function of the retina. VP-001 utilises PYC's proprietary drug delivery technology to overcome the major challenge for RNA drugs by ensuring that sufficient drug reaches its target inside the cells affected by RP11. About PYC Therapeutics PYC Therapeutics (ASX: PYC) is a clinical-stage biotechnology company creating a new generation of RNA therapies to change the lives of patients with genetic diseases. The Company utilises its proprietary drug delivery platform to enhance the potency of precision medicines within the rapidly growing and commercially proven RNA therapeutic class. PYC's drug development programs target monogenic diseases – the indications with the highest likelihood of success in clinical development[5]. The Company was the first to progress a drug candidate for a blinding eye disease of childhood into human trials and is now progressing multiple 'fast-follower' programs into the clinic. For more information, visit pyctx.com, or follow us on LinkedIn and Twitter. SOURCE PYC Therapeutics

快速通道

2021-06-15

·BioSpace

PYC Therapeutics Establishing New U.S. Headquarters in San Diego, Announces Residency at Johnson & Johnson Innovation, JLABS

Presence in Biopharmaceutical Hub to Enable Further Strengthening of Key Functions as Lead Drug Candidates VP-001 and VP-002 Head to the Clinic PYC Continues to Grow U.S. Team With Top-Tier Talent, Including Preclinical and Clinical Development, Manufacturing, Regulatory and Business Development SAN DIEGO and PERTH, Australia, June 15, 2021 /PRNewswire/ -- PYC Therapeutics (ASX: PYC), a biotechnology company developing a new generation of precision RNA therapeutics to change the lives of patients with inherited diseases, today announced residency at Johnson & Johnson Innovation, JLABS at San Diego (JLABS @ SAN DIEGO) after a successful application and selection process. This new U.S. location will serve as the Company's U.S. headquarters which will house preclinical and clinical development, regulatory, manufacturing, business development and general corporate operations. The Company's drug discovery and laboratory operations will remain in Perth, Australia. "The establishment of a U.S. headquarters is critical for the sustained execution of PYC's preclinical, clinical development and business development strategic priorities. We are pleased to join and contribute to the thriving biotech community in San Diego as we continue to drive momentum for our evolution into a clinical-stage company," said Sahm Nasseri, U.S. Chief Executive Officer of PYC Therapeutics. "We look forward to further growing our cross-functional team with experienced, top-tier talent in the U.S. who will collaborate closely with our foundational scientific and discovery team members in Perth as we develop next-generation RNA therapeutics that hold the potential to change the lives of patients with inherited diseases." PYC has appointed several key team members based at the new U.S. headquarters who will build out capabilities related to preclinical and clinical development, such as chemistry, manufacturing and controls and toxicology, and other corporate activities. The U.S. based team will remain focused on these activities to support the advancement into clinical development of PYC's two lead drug candidates, VP-001 for the treatment of retinitis pigmentosa type 11 and VP-002 for the treatment of autosomal dominant optic atrophy caused by mutations in the OPA1 gene together with engagement with key stakeholders, such as institutional investors and potential BD partners. JLABS @ SAN DIEGO is a 30,000 square-foot life science innovation center, located in San Diego. The labs provide a flexible environment for start-up companies pursuing new technologies and research platforms to advance medical care. Through a "no strings attached" model, JJI does not take an equity stake in the companies occupying JLABS and the companies are free to develop products - either on their own, or by initiating a separate external partnership with JJI or any other company. About PYC Therapeutics PYC Therapeutics (ASX: PYC) is a development-stage biotechnology company pioneering a new generation of RNA therapeutics that utilize PYC's proprietary library of naturally derived cell penetrating peptides to overcome the major challenges of current genetic medicines. PYC believes its PPMO (Peptide conjugated Phosphorodiamidate Morpholino Oligomer) technology enables a safer and more effective RNA therapeutic to address the underlying drivers of a range of genetic diseases for which no treatment solutions exist today. The Company is leveraging its leading-edge science to develop a pipeline of novel therapies including three preclinical stage programs focused on inherited eye diseases and a preclinical discovery program focused on neurodegenerative diseases. PYC's discovery and laboratory operations are located in Australia, and the Company recently launched an expansion into the U.S. for its preclinical, clinical, regulatory and business development operations. For more information, visit pyctx.com, or follow us on LinkedIn and Twitter. Forward looking statements Any forward-looking statements in this ASX announcement have been prepared on the basis of a number of assumptions which may prove incorrect and the current intentions, plans, expectations and beliefs about future events are subject to risks, uncertainties and other factors, many of which are outside the Company's control. Important factors that could cause actual results to differ materially from assumptions or expectations expressed or implied in this ASX announcement include known and unknown risks. Because actual results could differ materially to assumptions made and the Company's current intentions, plans, expectations and beliefs about the future, you are urged to view all forward-looking statements contained in this ASX announcement with caution. The Company undertakes no obligation to publicly update any forward-looking statement whether as a result of new information, future events or otherwise. CONTACTS: INVESTORS Deborah Elson/Matthew DeYoung Argot Partners deborah@argotpartners.com matthew@argotpartners.com MEDIA Leo Vartorella Argot Partners leo@argotpartners.com View original content to download multimedia: SOURCE PYC Therapeutics Company Codes: Australia:PYC

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
双等位基因RPE65突变相关的视网膜变性疾病	临床1期	美国	PYC Therapeutics Ltd.	2023-04-20
视网膜色素变性11型	临床1期	美国	PYC Therapeutics Ltd.	2023-04-20