Lexeo Therapeutics (LXEO) Conference June 05, 2025 02:00 PM ET Speaker0 Available to clients in this call. If the company is presenting, any recording may also be posted on their website. Usually, the opinion discussed by any experts speaking on this call are those who are speaking and not to check for Morgan. After this conference call, we will also be and participants now. I would now like to turn the call over to Tess Romero to begin. Please go ahead when you're ready. Speaker1 Thank you, operator, and t ...

Solid Biosciences (SLDB) 2025 Conference June 04, 2025 02:00 PM ET Speaker0 Everyone. My name is Maury Raycroft and I'm one of the biotech analysts at Jefferies. It's with great pleasure that I'd like to welcome the SolidBio team. We've got Bo Kumbow and Gabe Brooks joining us today. And we're going to do fireside chat format. So, for those who are new to the story, maybe give a brief intro to Solid, your key programs and platform. Speaker1 Yeah, thank you, Mari. I appreciate the invitation. Solid Bioscienc ...

enGene Holdings (ENGN) 2025 Conference June 04, 2025 11:05 AM ET Speaker0 Well, thank you very much. It's a delight to be here, and I'm grateful for this opportunity to speak about NGIN, which is a non viral genetic medicines company that's at a very important stage in our life cycle. I'm looking forward to sharing more about that with all of you. So again, thank you to the conference organizers for this opportunity. As you will expect, I'm gonna be making some forward looking statements during today's pres ...

Company Participation - Krystal Biotech, Inc. will participate in the Goldman Sachs 46th Annual Global Healthcare Conference on June 10, 2025, in Miami [1] - Krish S. Krishnan, Chairman and CEO, will engage in a fireside chat at 2:00 pm ET and host investor meetings throughout the day [1] Webcast Information - A webcast of the presentation will be available starting at 2:00 pm ET on June 10, 2025, and will be posted on the Investors section of the Company's website [2] Company Overview - Krystal Biotech, Inc. is a fully integrated, commercial-stage global biotechnology company focused on genetic medicines for 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 treating dystrophic epidermolysis bullosa [3] - The Company is advancing a robust pipeline of investigational genetic medicines in various fields including respiratory, oncology, dermatology, ophthalmology, and aesthetics [3]

Company Overview - BridgeBio Pharma, Inc. is a biopharmaceutical company focused on discovering, creating, testing, and delivering transformative medicines for genetic diseases [3] - Founded in 2015, the company has a pipeline that includes programs from early science to advanced clinical trials [3] - The team consists of experienced drug discoverers, developers, and innovators dedicated to applying advances in genetic medicine [3] Upcoming Event - Members of BridgeBio's management team will participate in a fireside chat at the Goldman Sachs 46th Annual Global Healthcare Conference 2025 on June 9 at 8:40 am ET [1] - A live webcast of the presentation will be available on the company's website, with a replay accessible for 30 days post-event [2]

Solid Biosciences Investor Contact: CHARLESTOWN, Mass., May 28, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the "Company" or "Solid"), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, announced that Bo Cumbo, President and CEO, and Gabriel Brooks, M.D., Chief Medical Officer, will participate in a fireside chat at the Jefferies Global Healthcare Conference on Wednesday, June 4, at 2:00 pm ET. A live webcast of the fireside chat will b ...

Core Insights - Verve Therapeutics is a clinical-stage company focused on developing genetic medicines for cardiovascular disease, aiming to shift treatment from chronic therapies to single-course gene editing medicines [2] Group 1: Company Overview - Verve Therapeutics, Inc. (Nasdaq: VERV) is developing a new class of genetic medicines targeting cardiovascular diseases [2] - The company's lead programs include VERVE-102, VERVE-201, and VERVE-301, which target key cholesterol drivers of atherosclerosis: LDL-C, triglycerides, and Lp(a) [2] - VERVE-102 aims to permanently turn off the PCSK9 gene in the liver, initially for heterozygous familial hypercholesterolemia (HeFH) and ultimately for patients with established atherosclerotic cardiovascular disease (ASCVD) [2] - VERVE-201 is designed to permanently turn off the ANGPTL3 gene for refractory hypercholesterolemia and homozygous familial hypercholesterolemia (HoFH) [2] - VERVE-301 targets the LPA gene to reduce Lp(a) levels, which is an independent risk factor for ASCVD and related conditions [2] Group 2: Investor Engagement - Management will participate in fireside chats at upcoming investor conferences, including Jefferies Global Healthcare Conference on June 4, 2025, and Goldman Sachs 46th Annual Global Healthcare Conference on June 9, 2025 [3] - Live webcasts of these events will be available on the company's website and archived for 30 days [1]

Cash runway extended into 2028; capital proceeds to fund operations through potential 2027 efficacy readout for LX2006 in Friedreich ataxia cardiomyopathy Financing led by Frazier Life Sciences and Janus Henderson Investors with participation from new and existing investors NEW YORK, May 27, 2025 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering novel treatments for cardiovascular diseases, today announced it has entered into a se ...

Summary of Cora Bio Conference Call Company Overview - **Company**: Cora Bio - **Industry**: Biotechnology, specifically focusing on genetic medicines for chronic diseases [1][2] Core Points and Arguments - **Mission and Strategy**: Cora Bio aims to develop genetic medicines targeting complex chronic diseases by leveraging pharmacology and genetic insights. The focus is on creating a toolbox for biological pathway activation that is not available with small molecules [3][4] - **RNA Editing Technology**: The company utilizes RNA editing through oligonucleotides to modify specific genes, aiming for high levels of editing (above 50%) to restore normal protein function in patients [4][5] - **Pipeline Development**: Cora Bio has a three:two:one strategy, planning to advance three candidates into clinical trials across two tissue types. The third indication is expected to target the central nervous system (CNS) for ALS [6][7] - **Regulatory Environment**: The company has engaged with the FDA, receiving orphan drug designation and conducting pre-IND meetings. There is concern about the increasing regulatory bar but confidence in the data driving approval processes [10][12][13] - **Preclinical Data**: Cora Bio presented promising preclinical data showing significant editing efficiency (60-65%) in animal models, which translates well to protein levels in circulation [18][20] - **Lipid Nanoparticle Delivery**: The company uses lipid nanoparticles for drug delivery, which have shown safety in patients with liver conditions. The focus is on optimizing dosing to achieve therapeutic protein levels [21][23][25] Important but Overlooked Content - **Patient Population Insights**: The company discussed the baseline liver function levels in patients with ZZ mutations, indicating that many have normal liver function, which may mitigate concerns about toxicity from lipid nanoparticles [22][23] - **Dosing Strategy**: Cora Bio aims for a dosing frequency of every three to six weeks, with the goal of achieving therapeutic protein levels that are comparable to normal individuals [26] - **Regulatory Pathway Confidence**: The company believes that demonstrating protein levels close to normal and achieving significant editing levels could support discussions for accelerated approval with regulators [39][40] - **Financial Position**: Cora Bio has a cash runway into 2027, with $139 million on the balance sheet and potential milestones from collaborations, indicating a solid financial foundation for ongoing development [47]

SOUTH SAN FRANCISCO, Calif., May 15, 2025 (GLOBE NEWSWIRE) -- Tenaya Therapeutics Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced that it granted stock options to purchase an aggregate of 461,000 shares of Tenaya common stock to three new non-executive employees in connection with the commencement of their employment. The stock options have an exerci ...