Core Insights - XORTX Therapeutics Inc. is focused on developing therapies for progressive kidney disease, with recent discoveries related to Autosomal Dominant Polycystic Kidney Disease (ADPKD) and the role of genetic factors in disease progression [1][2] Group 1: Company Developments - XORTX announced a presentation by Dr. Allen Davidoff at the Rare and Genetic Kidney Disease Summit, discussing the influence of genetic factors on xanthine oxidase (XO) expression and its implications for ADPKD progression [1] - The company is advancing its clinical trial for ADPKD, aiming to explore the newly identified genetic factors and their impact on disease progression [2] - XORTX has a patent portfolio that supports its expertise in developing XO inhibitors, which are crucial for treating individuals at risk of ADPKD [2] Group 2: Scientific Findings - Recent research indicates that genetic factors may lead to over-expression of XO, contributing to chronic hyperuricemia and potentially accelerating ADPKD progression [2][3] - Studies suggest a precision-medicine approach, where genetic risk variants guide treatment decisions for conditions associated with XO, including kidney dysfunction and diabetes [3] Group 3: Product Pipeline - XORTX has two clinically advanced products: XRx008 for ADPKD and XRx-101 for acute kidney injury related to COVID-19, along with a pre-clinical program, XRx-225, targeting Type 2 Diabetic Nephropathy [5]

Core Insights - Spruce Biosciences, Inc. announced the discontinuation of its CAHmelia-204 and CAHptain-205 clinical trials for tildacerfont in treating congenital adrenal hyperplasia (CAH) to conserve financial resources and maximize shareholder value [1][3]. Group 1: Clinical Trial Results - The CAHmelia-204 trial was a Phase 2b study involving 100 adults with classic CAH, assessing the safety and efficacy of tildacerfont in reducing glucocorticoid (GC) usage [2]. - The trial did not meet its primary efficacy endpoint, showing a placebo-adjusted reduction in daily GC dose of only 0.7mg HCe at week 24 [3]. - Compliance was high, with approximately 98% of patients adhering to the study drug, and tildacerfont was generally safe and well tolerated, with no serious adverse events reported [3][6]. Group 2: Additional Trial Insights - The CAHptain-205 trial evaluated the safety, pharmacodynamics, and pharmacokinetics of tildacerfont in both pediatric and adult patients, with doses ranging from 50mg QD to 400mg BID [4]. - A trend was observed indicating larger reductions in androstenedione (A4) levels with higher BID doses of tildacerfont [5]. - Similar to the CAHmelia-204 trial, tildacerfont was found to be generally safe and well tolerated across all doses, with no drug-related serious adverse events [6]. Group 3: Market Reaction - Following the announcement, Spruce Biosciences' stock price fell by 23.40%, trading at $0.41 during the premarket session [7].

Core Insights - The ALS Society of Canada has launched Canada's first National Genetic Counsellor to support individuals affected by amyotrophic lateral sclerosis (ALS), funded by a $265,000 donation from TD Bank Group [1][3][4] - The pilot project aims to provide virtual care services to help individuals understand their genetic risk and navigate treatment options [1][5] - Nearly 4,000 Canadians currently live with ALS, with care and support varying by location [4][8] Group 1: Project Overview - The National Genetic Counsellor will be based at the University of Calgary and will collaborate with Dr. Gerald Pfeffer [4] - The role will connect Canadians to local genetics experts and establish an education plan regarding ALS genetics [6] - The project is part of a broader initiative to enhance access to genetic counselling and testing for ALS patients [3][5] Group 2: Importance of Genetic Counselling - Access to genetic counselling is identified as a fundamental right for individuals living with ALS [3] - The role of the National Genetic Counsellor is expected to significantly impact the lives of ALS patients and their families [4][7] - There is a growing understanding of the genetic factors related to ALS, with potential therapies available for specific genetic variants [7] Group 3: Organizational Commitment - TD Bank Group's support is part of their corporate citizenship platform, aiming to provide access to critical health services [5] - ALS Canada is focused on addressing the urgent unmet needs for treatments and advocating for equitable access to therapies [9][10] - The organization has been active since 1977 and relies on donations for funding, as it does not receive core government support [10]

Core Insights - Avidity Biosciences has expanded its RNA delivery technology into precision cardiology with two new wholly-owned development candidates targeting rare genetic cardiomyopathies: AOC 1086 for PLN Cardiomyopathy and AOC 1072 for PRKAG2 Syndrome [1][2] - Preclinical data for AOC 1072 and AOC 1086 showed approximately 80% reduction in cardiac PLN mRNA and PRKAG2 mRNA, demonstrating robust siRNA delivery to the heart [1][2] - The company introduced next-generation technology innovations that enhance siRNA delivery in skeletal muscle and increase durability, allowing for less frequent dosing [3] Company Developments - Avidity is the first company to successfully deliver siRNA directly to skeletal muscle and has now demonstrated similar capabilities for the heart in preclinical studies [2] - AOC 1072 and AOC 1086 were well tolerated in preclinical studies, showing no adverse effects on electrocardiogram parameters [2] - Avidity will present preclinical data for AOC 1072 at the American Heart Association (AHA) Scientific Sessions 2024 on November 16 [2] Technology Innovations - Next-generation technology innovations include siRNA modifications and evolved antibody engineering, resulting in up to a 30-fold increase in siRNA delivery in skeletal muscle and sustained target inhibition for three months [3] - These advancements in siRNA delivery and durability are expected to improve patient convenience through less frequent dosing [3] Background on Target Diseases - PLN Cardiomyopathy is caused by a mutation in the PLN gene, leading to severe cardiac issues with no FDA-approved disease-modifying therapies available [5][6] - PRKAG2 Syndrome results from a mutation in the PRKAG2 gene, causing excessive cardiac glycogen storage and increasing the risk of sudden cardiac arrest or heart failure, with no FDA-approved therapies currently available [7] Company Mission - Avidity's mission is to improve lives by delivering a new class of RNA therapeutics, Antibody Oligonucleotide Conjugates (AOCs), which combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies [8] - The company is broadening its pipeline to include programs in cardiology and immunology through internal discovery efforts and partnerships [8]

Dose-dependent increase in neuroprotective APOE2 expression in all participants with ongoing durability at 12 months Consistent reductions across CSF tau biomarkers and tau PET in majority of participants LX1001 well tolerated across all dose cohorts with no reports of amyloid-related imaging abnormalities (ARIA) Company to host webcast today at 7:00 AM ET NEW YORK, Oct. 30, 2024 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering tr ...

Myriad's MyRisk® test is the first hereditary cancer test integrated into Flatiron's OncoEMR® SALT LAKE CITY, Oct. 15, 2024 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in genetic testing and precision medicine, today announced a collaboration with Flatiron Health, a leading health tech company dedicated to expanding the possibilities of point of care solutions in oncology. This collaboration allows physicians to order Myriad's MyRisk® Hereditary Cancer Test and view the results of the ...

NEW YORK, Oct. 15, 2024 (GLOBE NEWSWIRE) -- Tourmaline Bio, Inc. (Tourmaline) (NASDAQ: TRML), a late-stage clinical biotechnology company developing transformative medicines to dramatically improve the lives of patients with life-altering immune and inflammatory diseases, announced today that it will host a webinar, Understanding the Genetic Validation for IL-6 Inhibition in Cardiovascular Disease, featuring Dr. Dipender Gill, CEO and Founder of Sequoia Genetics. The webinar will be held on Friday, November ...

CALGARY, Alberta, Oct. 09, 2024 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the "Company") (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, is pleased to announce the Company has initiated a precision medicine program. On August 29, 2024, XORTX announced that independent peer-reviewed research reported that genetic factors are linked to the over-expression of xanthine oxidase ...

CAMBRIDGE, Mass., Sept. 30, 2024 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced that Paul Bolno, MD, MBA, President and Chief Executive Officer, is scheduled to participate in an analyst-led fireside chat at the Chardan 8th Annual Genetic Medicines Conference in New York City on Tuesday, October 1, 2024, at 1:00 p.m. ET. A live webcast of this presentation w ...

Commission file number 000-51504 UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 FORM 20-F ☐ REGISTRATION STATEMENT PURSUANT TO SECTION 12(b) OR (g) OF THE SECURITIES EX- CHANGE ACT OF 1934 OR ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended June 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to OR ☐ SHELL COMPANY REPORT PURSUANT TO SEC ...