Rare Diseases News
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Onapristone Extended Release: Safety Evaluation from Phase I–II Studies with an Emphasis on Hepatotoxicity
Introduction Anliprogestins have demonstrated promising activity against breast and gynecological cancers, but liver-related safety concerns limited the advancement of this therapeutic class. Onapristone is a full progesterone receptor antagonist originally developed as an oral contraceptive and later evaluated in phase II studies for metastatic breast cancer. Because of liver enzyme elevations ...
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Biotech Stock News Bite - Longeveron Inc. (NASDAQ: LGVN) Stock Soars on Clinical Pipeline and 2024 Key Priorities and Goals
Investorideas.com (www.investorideas.com) Biotech Stock News Bites - Longeveron Inc. (NASDAQ: LGVN), a clinical stage biotechnology company developing regenerative medicines is one of NASDAQ's top gainers today and the stock is trending on Yahoo Finance, currently trading at $3.8955, up $1.6955 or 77.0682%. The stock had a morning high of $5.47 on volume of over 36 Million/. The Company's CEO, ...
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Cost-effective Animal Disease Models to Power Up Life Science Research
In a pioneering effort to revolutionize life science research, a wide range of cost-effective animal disease models, from Drosophila to non-human primates, from knockout models to different disease models, are released. These groundbreaking models are set to empower researchers worldwide by enabling them to unravel the mysteries of various diseases more efficiently and affordably than ever ...
By Protheragen
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Comprehensive Gene Therapy Development Solutions to Further Boost Rare Disease Research
As several gene therapy approvals for rare disease treatment gain steam in 2023, a comprehensive set of gene therapy development solutions is released to pave richer avenues for rare disease research. This path-breaking move sees the integration of cutting-edge technology with state-of-art research facilities to amplify the possibilities of devising effective remedies for rare diseases. Rare ...
By Protheragen
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BIOtechNOW featured IVS’s participating BIO Innovation Zone
BIOtechNOW featured IVS’s participating BIO Innovation Zone Later this month in Philadelphia at the BIO International Convention, BIO will be partnering with the National Institutes of Health (NIH) and the National Science Foundation (NSF) to host the 2nd annual, newly-expanded BIO Innovation Zone. The Zone will feature Small Business Innovation Research (SBIR) funded early-stage biotech ...
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BIO Investor Forum Company Snapshot: InvivoSciences
BIO Investor Forum Company Snapshot: InvivoSciences What is your company’s lead product or technology? InvivoSciences, Inc. (IVS) is a leading developer of proprietary human 3D cell/tissue culture models for highly-predictive, multi-parameter phenotypic assays both in pharmacology safety & discovery screening and in personalized and regenerative medicine. IVS’s Functional 3D ...
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Clinical Trial Managers Need to Know These Skills for the Future
The Covid-19 epidemic has changed the world in many ways, and many clinical trials have been done to research more about it. According to the World Health Organization (WHO), 12,969 clinical trials were done in America in 2021. Even in pre-pandemic times, clinical trials have always been an ongoing process. Clinical trial managers work on cutting-edge studies and contribute to ...
By Brio Group
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SeqOne Genomics and the French Thrombotic MicroAngiopathies National reference center (CNR-MAT) pioneer the use of Oxford Nanopore sequencing technology to improve patient outcomes in kidney disease while reducing turnaround times
SeqOne today announced the results of a long-standing research collaboration with Pr. Laurent Mesnard, Co-Director of the French National center for Thrombotic MicroAngiopathies (CNR-MAT), aimed at improving the diagnosis and management of patients suspected of suffering from thrombotic microangiopathies and atypical Hemolytic syndrome (aHUS). The program involved using Oxford Nanopore’s ...
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FDA U.S. Food & Drug Administration – Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis
A five-year action plan developed to meet requirements under Section 4 of the Accelerating Access to Critical Therapies for ALS Act. ...
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Teva and MODAG Announce Licensing Collaboration for Neurodegenerative Disease Drug Candidate
TEL AVIV & WENDELSHEIM, Germany--(BUSINESS WIRE)-- Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA) and MODAG GmbH today announced a strategic collaboration on the exclusive worldwide licensing and development of MODAG's lead compound anle138b and a related compound, sery433. Anle138b targets pathological alpha-synuclein oligomers and is being evaluated in patients with ...
By MODAG GmbH
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Roswell Biotechnologies Forms Scientific Advisory Board
Roswell Biotechnologies, Inc., the molecular electronics company, announced today the formation of its Scientific Advisory Board (SAB). Comprised of industry and academic leaders in science and technology, the new SAB will be instrumental in the final stages of development and commercialization of the company's molecular electronics chip, a fully scalable universal biosensor capable of seeing ...
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Nautilus Biotechnology Partners with the Translational Genomics Research Institute (TGen) to Investigate Applications of Single-Molecule Proteomic Analysis in Diffuse Intrinsic Pontine Glioma (DIPG)
Nautilus Biotechnology, Inc. (NASDAQ: NAUT; or “Nautilus”), a company pioneering a single-molecule protein analysis platform, and the Translational Genomics Research Institute (TGen), part of City of Hope, today announced a partnership to explore the utility of the Nautilus platform by studying specific protein targets in diffuse intrinsic pontine glioma (DIPG), a rare and often fatal ...
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Oligomerix Presents Data on Lead Program at Clinical Trials on Alzheimer’s Disease (CTAD) Conference and Society for Neuroscience Meeting
Oligomerix, Inc., a privately held company pioneering the development of small molecule therapeutics targeting tau for Alzheimer’s and rare neurodegenerative diseases, announced that the Company presented data from a therapeutic study in an animal model of its lead product candidate OLX-07010 at the 14th Annual Clinical Trials on Alzheimer's Disease (CTAD) conference and at the Society for ...
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Eagle Pharmaceuticals Provides Business Update and Guidance for 2023
Eagle Pharmaceuticals, Inc. (Nasdaq: EGRX) (“Eagle” or the “Company”) today provided a business update and guidance for 2023. Highlights: During the 12 months ended September 30, 2022, Eagle recorded net income of $21.3 million or $1.63 per diluted share and adjusted EBITDA of $125.6 million and non-GAAP earnings per diluted share of $7.54, a significant increase from ...
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NextPoint Therapeutics Announces $80 Million Series B Financing co-led by Leaps by Bayer and Sanofi Ventures to Advance Novel Immuno-Oncology Programs
NextPoint Therapeutics, a biotechnology company developing a new world of precision immuno-oncology, announced today that it raised $80 million in Series B financing co-led by Leaps by Bayer, the impact investment arm of Bayer AG, and Sanofi Ventures, the strategic venture capital arm for Sanofi. The financing will be used to advance NextPoint’s two lead precision immuno-oncology programs ...
By Bayer AG
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BRIM Biotechnology receives FDA Orphan Drug Designation for BRM424 to treat neurotrophic keratitis
BRIM Biotechnology, Inc. (“BRIM,” TPEx 6885) is pleased to announce that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for BRM424 in the treatment of neurotrophic keratitis (NK), a rare degenerative eye disease which causes very severe cornea damage and can lead to loss of sight. Dr. Haishan Jang, Chairwoman and CEO of BRIM Biotechnology, ...
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LISCure`s Phase 2 Drug Candidate Granted Orphan Drug Designation (ODD) from US FDA
LB-P8, a novel drug being developed by LISCure Biosciences, Inc. (LISCure) as a treatment for Primary Sclerosing Cholangitis (PSC), has been designated as an orphan drug by the US FDA. On December 1, 2022, the US FDA notified LISCure of the orphan drug designation of LB-P8, LISCure's microbiome novel drug, as treatment of PSC. LB-P8 is, to the best of company's knowledge, the first drug to be ...
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Lumos Pharma to Present at the H.C. Wainwright BIOCONNECT Virtual Conference
Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, announced that the Company will present at the H.C. Wainwright Virtual BIOCONNECT Conference being held January 10-13, 2022. A webcast of the presentation will be available at 7:00AM ET on January 10th through the following link: ...
By Lumos Pharma
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CY6463 administration is linked to improvements in Alzheimer’s disease-relevant biomarkers, as revealed by eXplainable AI-driven analysis of multiple Phase 1 clinical trials ?
Phase 1 studies show improvement in cognitive factors, consistent with therapeutic potential of CY6463 to improve key features in neurological diseases CY6463 favorable tolerability and safety profile was confirmed Ariana Pharma, a leading Artificial Intelligence (AI) drug development company, in collaboration with Cyclerion Therapeutics, presented today safety and pharmacodynamic results ...
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Lumos Pharma to Host KOL Webinar to Discuss Interim Phase 2 Data from OraGrowtH Trials in PGHD
Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical stage biopharmaceutical company focused on rare endocrine disorders, announced that the Company will host two key opinion leaders in the field of pediatric endocrinology for a discussion of interim data from our Phase 2 OraGrowtH210 and PK/PD OraGrowtH212 Trials evaluating oral LUM-201 in Pediatric Growth Hormone Deficiency (PGHD). The KOL webinar will ...
By Lumos Pharma
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