Core Viewpoint - Passage Bio, Inc. is making progress in its clinical trials for PBFT02, a treatment for neurodegenerative diseases, with a focus on frontotemporal dementia (FTD) and plans for future regulatory engagement [2][6]. Recent Highlights - The company treated its first FTD-GRN patient with Dose 2 of PBFT02 and enrolled a second patient, with additional patients being evaluated for trial eligibility [6]. - A high productivity, suspension-based manufacturing process for PBFT02 was presented at the ASGCT 28th Annual Meeting, showing significant improvements in productivity and purity [6]. - The company has a cash runway expected to last into the first quarter of 2027, with cash and cash equivalents totaling $63.4 million as of March 31, 2025 [12]. Anticipated Upcoming Milestones - The company plans to initiate dosing of FTD-C9orf72 patients in the first half of 2025 [4]. - Interim safety and biomarker data from Dose 2 and 12-month data from Dose 1 patients are expected to be reported in the second half of 2025 [6]. Financial Results - For the first quarter of 2025, research and development expenses were $7.7 million, down from $11.5 million in the same quarter of 2024 [12]. - General and administrative expenses decreased to $6.1 million from $6.5 million year-over-year [12]. - The net loss for the quarter was $15.4 million, or $0.25 per share, compared to a net loss of $16.7 million, or $0.30 per share, in the prior year [12][15].

Core Insights - Korro Bio, Inc. is focused on developing genetic medicines through RNA editing for both rare and prevalent diseases, with a significant milestone expected in the second half of 2025 regarding the KRRO-110 clinical trial for Alpha-1 Antitrypsin Deficiency (AATD) [2][5][16] Financial Performance - As of March 31, 2025, Korro reported cash, cash equivalents, and marketable securities totaling $139.0 million, down from $163.1 million at the end of 2024, which is expected to fund operations into 2027 [9][23] - Collaboration revenue for the first quarter of 2025 was $2.6 million, a notable increase from zero in the same period of 2024, attributed to the partnership with Novo Nordisk [10] - Research and Development (R&D) expenses rose to $19.7 million in Q1 2025 from $13.6 million in Q1 2024, primarily due to increased costs associated with KRRO-110 [11] - General and Administrative (G&A) expenses slightly decreased to $7.8 million in Q1 2025 from $7.9 million in Q1 2024 [12] - The net loss for Q1 2025 was $23.4 million, compared to a net loss of $19.6 million in Q1 2024 [13] Operational Updates - The company is streamlining operations and reducing its workforce by approximately 20% to focus on key program milestones, including the completion of the Phase 1/2a REWRITE clinical trial in 2026 [3][5] - Korro is executing a 3-2-1 strategy aimed at establishing three clinical-stage development programs targeting two tissue types by leveraging its OPERA platform [6][16] - The leadership team has been strengthened with new appointments, enhancing the company's capabilities in drug discovery and development [6][7] Pipeline Developments - The interim readout from the Phase 1/2a REWRITE clinical trial of KRRO-110 for AATD is on track for the second half of 2025, with trial completion expected in 2026 [5][6][16] - Korro plans to announce a second development candidate by the end of 2025, focusing on a rare metabolic disorder program targeting the liver [16] - The collaboration with Novo Nordisk continues to progress, with efforts to advance up to two programs targeting cardiometabolic diseases [7][16]

Company Overview - Krystal Biotech, Inc. is a fully integrated, commercial-stage global biotechnology company focused on the discovery, development, and commercialization of genetic medicines to treat diseases with high unmet medical needs [3] - The company's first commercial product, VYJUVEK, is the first-ever redosable gene therapy and the first genetic medicine approved by the FDA and EMA for the treatment of dystrophic epidermolysis bullosa [3] - Krystal Biotech is advancing a robust preclinical and clinical pipeline of investigational genetic medicines in various fields including respiratory, oncology, dermatology, ophthalmology, and aesthetics [3] Upcoming Events - The company will participate in the BofA Securities 2025 Health Care Conference on May 14, 2025, in Las Vegas [1] - Krish S. Krishnan, Chairman and CEO, will take part in a fireside chat scheduled at 11:20 am PT and will host investor meetings throughout the day [1] - A webcast of the presentation will be available starting at 11:20 am PT on May 14, 2025, and will be posted on the Investors section of the company's website [2]