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 ...

PITTSBURGH, June 03, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) today announced that the Company will participate in the Goldman Sachs 46th Annual Global Healthcare Conference on June 10, 2025, in Miami. Krish S. Krishnan, Chairman and Chief Executive Officer, will take part in a fireside chat scheduled at 2:00 pm ET and host investor meetings throughout the day. A webcast of the presentation will be available here beginning at 2:00 pm ET on Tuesday, June 10, 2025 and will ...

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 ...

Investor Contact Jen Robinson Verve Therapeutics, Inc. jrobinson@vervetx.com Media Contact Ashlea Kosikowski 1AB ashlea@1abmedia.com Jefferies Global Healthcare Conference on Wednesday, June 4, 2025, at 9:20 a.m. ET in New York, NY Goldman Sachs 46 Annual Global Healthcare Conference on Monday, June 9, 2025, at 11:20 a.m. ET in Miami, FL Live webcasts will be available in the investor section of the company's website at www.vervetx.com. The webcasts will be archived for 30 days following the fireside chats. ...

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 ...

Summary of Krystal Biotech (KRYS) Conference Call Company Overview - **Company Name**: Krystal Biotech - **Industry**: Biotechnology - **Focus**: Development and commercialization of genetic medicines for monogenic diseases - **Location**: Pittsburgh, with all manufacturing and intellectual property based in the US [4][5] Core Points and Arguments Commercial Stage and Pipeline - Krystal Biotech launched its drug, **BIJUVAK**, for dystrophic epidermolysis bullosa (DEB) in the US about 18 months ago and received approval in Europe, with plans to launch in Europe and Japan later in 2025 [4][5] - The company has been cash flow positive for the last seven quarters and does not plan to seek financing in the next few years [5][6] - The focus for 2025 includes expanding into lung and eye tissues, with early entry into oncology [5] Market Trends and Patient Dynamics - In Q1, patient healing led to temporary pauses in treatment, which is expected to result in a return of patients over the next 60-120 days [8][9] - The company has 17 sales representatives covering 52 states, facing challenges in prescription pull-through due to patient dispersion [10][11] - Insurance changes in Q1 impacted revenue but are expected to normalize in Q2 [12][13] European and Japanese Market Launch - The EU approval allows broader patient self-administration compared to the US, with launches expected in Germany and France in Q3 2025 [19][20] - Pricing in the EU is anticipated to be around 50-75% of the US price, with a conservative accrual strategy [27][28] Pipeline Developments - Upcoming focus on cystic fibrosis (CF) and alpha-one antitrypsin deficiency treatments, with strong value propositions for patients with null mutations [34][35] - The company is preparing for a lung cancer announcement at ASCO, reinforcing confidence in its lung delivery capabilities [37][38] Ophthalmology Programs - The company is initiating a registrational study for KBM-803 targeting ocular complications of DEB, with a market opportunity for about 10-15% of the RDEB population having eye lesions [48][49] - Another program, KB801, targets neurotropic keratitis, expected to be a significant market opportunity but not anticipated for launch until 2026 [50][51] Financial Outlook - The company is confident in its financial position, with plans to manage the launch of BIJUVAK and advance other pipeline assets without needing additional funding [53][55] Other Important Insights - The company emphasizes the importance of patient comfort and convenience in drug administration, with a high percentage of patients preferring home administration [30][31] - The management is focused on strategic partnerships for larger indications to enhance market reach and financial stability [55] This summary encapsulates the key points discussed during the conference call, highlighting the company's strategic direction, market dynamics, and pipeline developments.