-- First ever clinical data supporting safety and efficacy of Prime Editing in humans -- -- Initial data from first patient dosed in Phase 1/2 trial finds single dose of PM359 led to 58% DHR positivity by Day 15 and 66% by Day 30, well above levels believed to be potentially curative -- -- Rapid engraftment observed in both neutrophils and platelets -- -- Encouraging safety profile; no serious adverse events related to PM359 -- -- Initiating efforts to explore continued clinical development oppo

JUV-161 is a first-in-class muscle regenerating biologic that will address myopathies starting with Myotonic Dystrophy Type 1 JUV-161 was discovered using Juvena’s proprietary JuvNET platform, an AI and stem cell secretome-based drug discovery and development platform May 19, 2025 -- Juvena Therapeutics, Inc. (“Juvena”), a clinical-stage biotechnology company pioneering tissue restorative biologics for chronic muscle and metabolic diseases, announced today that it is enrolling participants in

May 15, 2025 -- Pathos AI, a leading AI-driven biotech company applying cutting-edge artificial intelligence to drug development, today announced its $365 million Series D financing, bringing its post-money valuation to approximately $1.6 billion. This financing marks a major milestone for Pathos as it expands its AI-enabled platform to drive greater speed, precision, and success in oncology drug development. The proceeds will support advancement of the company’s clinical-stage pipeline and c

Despite growing recognition of long COVID as a serious condition, diagnosed patients have no approved treatment options, with many suffering from debilitating fatigue and brain fog Evidence suggests sustained inflammation plays a central role in the pathogenesis of long COVID, particularly in the associated cognitive dysfunction and other neurological symptoms1 Bezisterim targets key underlying mechanisms of neuroinflammation, and has demonstrated the potential to reduce chronic symptoms in Alzh

May 15, 2025 -- Axovia Therapeutics Ltd., a biotechnology company developing therapies to address the genetic causes of blindness and obesity, announced that today it will unveil new preclinical proof-of-concept data for novelly designed, AXV-201, to treat individuals with severe obesity and very high BMIs resulting from MC4R mutations, in a poster presentation at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, which is being held from May 13-17, 2025 in New Orleans,

Following the formal safety review of Cohort 1, no safety or tolerability issues were observed, allowing the company to move forward with Cohort 2. The company plans on releasing more detailed results from Phase 1 later in 2025 following completion of additional dose cohorts. May 19, 2025 -- GT Biopharma, Inc. (the “Company”) (NASDAQ: GTBP), a clinical stage immuno-oncology company focused on developing innovative therapeutics based on the Company's proprietary TriKE® natural killer (NK) cell e

- First ASXL1 Pediatric Acute Myeloid Leukemia (AML) Patient Dosed at MD Anderson Cancer Center: Program Supported by Rare Pediatric Disease Designation (RPDD) May 15, 2025 -- SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS’’ or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today announced that the first pediatric AML patient has been dosed in the ongoing Phase 2 trial of SLS009 (ta

–FDA grants Priority Review of application, with PDUFA target action date of September 12, 2025– –ORLADEYO would be the first targeted oral prophylactic therapy for patients with HAE under the age of 12, if approved– May 14, 2025 -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for the use of oral, once-daily ORLADEYO® (berotralstat) in pediatric patients with hereditary angioedema (HA

May 14, 2025 -- BetterLife Pharma Inc. (“BetterLife” or the “Company”) (CSE: BETR / OTCQB : BETRF / FRA: NPAU), an emerging biotech company focused on the development of BETR-001, a non-hallucinogenic neuroplastogen in the treatment of psychiatric and neurological disorders, recently presented a scientific update at Bloom Burton Conference on May 5, 2025 in Toronto, Canada. BetterLife reported that its proprietary compound, BETR-001, is showing very promising preclinical data of its breakthrou

Treatment with EBT-107 led to a rapid reduction in clinical biomarkers and suppression of random HBV DNA integration with minimal risk of treatment-related chromosomal aberrations Excision’s proprietary gene editing technology holds unique, curative potential to address the primary cause of liver cancer Chronic hepatitis B infection is estimated to affect 250 million people globally, including 1.5 million new cases each year Preclinical data supports advancing EBT-107 into IND-enabling studies a