SAN DIEGO, July 09, 2025 (GLOBE NEWSWIRE) -- Calidi Biotherapeutics, Inc. (“Calidi” or the “Company”) (NYSE American: CLDI), a clinical-stage biotechnology company pioneering the development of targeted therapies with the potential to deliver genetic medicines to distal sites of disease, today announced the entry into a definitive agreement for the immediate exercise of certain outstanding Series A, Series B-1, Series C-1, Series D, Series E and Series F Warrants (the “Existing Warrants”) to purchase up to ...

Neurotrophic Keratitis (NK) Market & KB801 Opportunity - The number of patients in the U S with at least one NK claim has increased by over 115% from 31,000 in 2020 to an estimated 68,000 in 2024[9, 10] - In 2023, U S Medicaid and Medicare spent over $540 million on Oxervate, the only FDA-approved therapy for NK[12] - An estimated over 410,000 days of Oxervate therapy were reimbursed in the U S in 2024[12] - KB801 is designed to address the shortcomings of Oxervate, aiming for superior NGF exposure with significantly reduced dosing frequency[11, 21] KB801 Preclinical Data - KB801 transduces primary human corneal epithelial cells in vitro, leading to dose-dependent expression and secretion of mature NGF[25, 26, 29] - In vitro studies confirmed functionality of secreted NGF using a growth factor starved cell proliferation assay[25, 30] - Topical administration of KB801 to wounded murine corneas was well-tolerated and resulted in localized NGF expression[25] - Head-to-head mouse PK studies demonstrated a clear durability advantage with KB801 compared to recombinant NGF, even against intensive recombinant dosing[43, 47] KB801 Clinical Development - The EMERALD-1 study is a Phase 1/2 double-masked, 2:1 randomized, placebo-controlled study in patients with moderate-to-severe NK, with the first patient dosed in July 2025[51, 53, 1] - Krystal is pursuing Platform Technology Designation with the FDA, potentially leading to expedited development of KB801[56, 57]

Updated Data from 17 Patients Consistent with Previously Presented Data; All Patients Treated with BEAM-101 Achieved Hemoglobin F (HbF) Induction of >60%, Hemoglobin S (HbS) Reduction to <40%, and Resolution of Anemia Patients Required a Median of One Mobilization Cycle and Experienced Rapid Neutrophil and Platelet Engraftment Safety Profile Remained Consistent with Busulfan Conditioning, Autologous Hematopoietic Stem Cell Transplantation and Underlying SCD Enrollment Complete in Both Adult and Adolescent C ...

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 ...

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.

Presentation to Include Updated Data from 17 Sickle Cell Disease Patients in the Ongoing BEACON Phase 1/2 Clinical Trial Evaluating Safety and Efficacy of BEAM-101 Beam to Host Investor Webcast on Friday, June 13, 2025, at 4:00 p.m. ET CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will present new data from the BEACON Phase 1/2 clinical trial of BE ...