- Nonhuman primate (NHP) study in hemophilia A demonstrated therapeutically relevant factor VIII (FVIII) activity with durable response through approximately 19 months with an encouraging safety profile - Well capitalized with $226.0 million in cash, cash equivalents and available-for-sale marketable securities as of March 31, 2025 with runway anticipated to support operations into 2027 EMERYVILLE, Calif., May 13, 2025 (GLOBE NEWSWIRE) -- Metagenomi, Inc. (Nasdaq: MGX), a precision genetic medicines company ...

Editas Medicine (EDIT) reported an adjusted loss of 43 cents per share in the first quarter of 2025, narrower than the Zacks Consensus Estimate of a loss of 51 cents. The adjusted figure excluded the effect of restructuring and impairment charges in the reported quarter. The company had incurred a loss of 76 cents per share in the year-ago quarter.Collaboration and other research and development (R&D) revenues, which comprise the company’s top line, were $4.7 million in the reported quarter, up significantl ...

Core Insights - Tenaya Therapeutics is advancing its capabilities in genetic medicines for heart disease, presenting five abstracts at the ASGCT 2025 Annual Meeting [1][2] Group 1: Research and Development Highlights - The company is focusing on novel capsid engineering and optimization of cardiomyocyte-targeting genetic medicines, particularly through the use of adeno-associated virus (AAV) gene therapies [2][5] - A prime editing prototype demonstrated successful correction of the mutated RBM20 allele in a murine model of dilated cardiomyopathy (DCM), improving cardiac function [4] - Tenaya has developed a humanized mouse model for RBM20-mutant DCM, validating the effectiveness of cardiac editing using a dual vector prime editing approach [4] Group 2: Presentation Details - Presentations at ASGCT 2025 include advancements in capsid engineering, with high-throughput screening identifying novel capsid candidates that outperform AAV9 in cardiomyocyte targeting [5] - The company is sharing its progress on TN-501, a Cas9 gene editing candidate aimed at treating PLN-R14del-associated DCM, which showed promising preclinical results in murine models [6][11] - Tenaya has established a proprietary HEK293-based manufacturing process for AAV gene therapies, improving yield and reducing costs compared to existing options [6] Group 3: Company Overview - Tenaya Therapeutics is a clinical-stage biotechnology company dedicated to developing curative therapies for heart disease, with a pipeline that includes gene therapies for various cardiomyopathies and heart failure [9]

Core Insights - Cellectis reported financial results for Q1 2025, highlighting progress in clinical studies and partnerships, particularly with AstraZeneca [1][2][9] Financial Results - As of March 31, 2025, Cellectis had a cash position of $246 million, down from $264 million at the end of 2024, providing a runway into H2 2027 [10][11] - Consolidated revenues and other income for Q1 2025 were $12.0 million, an increase from $6.5 million in Q1 2024, primarily due to a $5.9 million increase in revenue recognized under the AstraZeneca joint research collaboration agreement [13][14] - R&D expenses for Q1 2025 were $21.9 million, slightly down from $22.3 million in Q1 2024, while SG&A expenses decreased to $4.7 million from $5.1 million [15][16] - The consolidated net loss attributable to shareholders was $18.1 million for Q1 2025, compared to a net income of $5.6 million in Q1 2024 [19][20] Pipeline Highlights - Cellectis is advancing its clinical programs, including the BALLI-01 study for lasme-cel (UCART22) in relapsed or refractory B-cell acute lymphoblastic leukemia, with results expected in Q3 2025 [3][6][7] - The NATHALI-01 study for eti-cel (UCART20x22) in relapsed or refractory B-cell non-Hodgkin lymphoma is ongoing, with readout expected in late 2025 [4][7] Research and Development - Cellectis presented research data on TALEN®-mediated non-viral transgene insertion and TALE base editing at the ASGCT annual meeting, showcasing advancements in gene editing technologies [5][8] - The company is focusing on the development of its pipeline, including manufacturing and clinical trial expenses for UCART22 and UCART20x22 [12] Partnerships - Ongoing R&D activities under the AstraZeneca partnership include three programs: one allogeneic CAR T for hematological malignancies, one for solid tumors, and one in vivo gene therapy for a genetic disorder [9][6]

Core Viewpoint - Editas Medicine, Inc. announced a partial affirmation and partial vacate of a previous decision by the U.S. Court of Appeals regarding patent interference related to CRISPR/Cas9 editing, with the case remanded back to the Patent Trial and Appeal Board (PTAB) for further review [1] Group 1: Legal and Patent Developments - The U.S. Court of Appeals for the Federal Circuit has affirmed-in-part and vacated-in-part the PTAB's decision regarding patents for CRISPR/Cas9 editing in human cells involving the University of California, University of Vienna, Emmanuelle Charpentier, and the Broad Institute [1] - Editas Medicine's in-licensed patents covering CRISPR/Cas12a are not affected by this decision and are not involved in the ongoing interference proceedings [1] Group 2: Company Strategy and Intellectual Property - The company remains confident in the strength of its intellectual property (IP) portfolio, which is expected to generate significant value now and in the future [2] - Editas holds a large portfolio of foundational U.S. and international patents, including exclusive licenses for Cas9 and Cas12a patent estates, which are crucial for developing human medicines [2] - The foundational IP includes issued patents covering fundamental aspects of CRISPR/Cas12a and CRISPR/Cas9 gene editing in all human cells, essential for CRISPR-based medicines [2] Group 3: Company Mission and Focus - Editas Medicine is focused on translating the potential of CRISPR/Cas12a and CRISPR/Cas9 systems into a pipeline of in vivo medicines for serious diseases [3] - The company aims to discover, develop, manufacture, and commercialize transformative gene editing medicines for a broad class of diseases [3]

Financial Data and Key Metrics Changes - Cash and cash equivalents as of March 31, 2025, were $23.6 million, with net proceeds of $21.4 million from a registered direct offering in January 2025 [22] - Revenue for Q1 2025 was $1 million, an increase of $489,000 compared to $545,000 in the same period last year, attributed to increased activity in partner-funded projects [22] - R&D expenses were $11.8 million for Q1 2025, down from $12 million in the previous year due to cost reduction initiatives [23] - SG&A expenses rose to $9.9 million in Q1 2025 from $7 million in the prior year, primarily due to a $3 million litigation accrual [23] - Net loss for Q1 2025 was $49.4 million, compared to a net loss of $27 million in the same period last year, largely due to a $21 million non-cash goodwill impairment [23][24] Business Line Data and Key Metrics Changes - The rice platform secured agreements with four major rice seed companies, representing approximately 40% of estimated accessible rice acres across North and Latin America [7] - Progress in disease resistance traits for canola included successful edits for four different modes of action, with positive field trial results for the second mode of action [11] - The soybean platform achieved a significant milestone with successful editing of a soybean cell for the HT2 trait, enabling expanded platform development [12] Market Data and Key Metrics Changes - The regulatory environment has improved, with the California Rights Commission approving field research proposals for gene-edited rice, and Ecuador recognizing certain traits as equivalent to conventional breeding [16][17] - The USDA APHIS designated two disease resistance trait products for canola as not regulated, allowing for unrestricted product development in the U.S. [18] Company Strategy and Development Direction - The company is focused on optimizing operations and reducing cash burn in 2025 while pursuing strategic priorities in rice, canola, and soybean platforms [20][25] - The commercialization strategy is progressing, with anticipated trait validation trials in Latin America and continued development of bio-based fermentation products [19][15] - The company aims to maximize shareholder value by prioritizing investments in near-term commercial opportunities with developed traits [20] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the transformative potential of the RTDS technology platform and its ability to attract significant commercial interest [5][25] - The company is positioned at an important inflection point in the agricultural industry, with a focus on gene editing technologies that enhance crop adaptability and productivity [20][25] - Management highlighted the importance of regulatory advancements in supporting the commercialization of gene-edited traits [25] Other Important Information - The company anticipates nominal revenues from its sustainable ingredients program later in 2025, with ongoing partnerships with consumer packaged goods companies [15] - The company is taking a disciplined approach to resource allocation, ensuring financial flexibility to execute strategic priorities [20] Q&A Session Summary Question: Can you walk through the cash burn and the step up in SG&A in Q1? - Management indicated a cash burn of approximately $4.4 million gross in Q1, with the increase in SG&A primarily due to a litigation accrual [30][31] Question: What is the expected timeframe for EU regulatory discussions? - Management believes the trilogue discussions could conclude within six months, with secondary legislation expected within twelve months [33][34] Question: What are the working capital impacts when trait products hit commercial launch? - Management stated that there would not be a significant cash hit as they support customers from a stewardship standpoint [38][42] Question: What is the potential scale and ramp-up speed for biofragrance products? - Management noted strong demand for bio-based products, with a quick ramp-up expected from 2026 to 2028 [40] Question: Are there other Latin American countries with similar regulatory frameworks to Ecuador? - Management confirmed that several Latin American countries view gene-edited products as conventional breeding, following Ecuador's lead [66][68] Question: Is there existing usage data for clethodim by rice farmers in Latin America? - Management clarified that clethodim is an approved herbicide in South America, and the new trait will provide an additional tool for rice farmers [69][71]

Core Insights - Cibus is advancing its gene editing technologies, particularly in rice and canola, with a focus on herbicide tolerance and disease resistance traits, targeting a commercial launch by 2027 [1][2] - Positive regulatory developments in Ecuador and the U.S. validate Cibus' gene editing technologies, enhancing its commercial prospects [1][2][7] - The company reported a significant increase in net loss for Q1 2025, primarily due to a goodwill impairment, while also showing progress in revenue generation [8][9][24] Company Developments - Cibus has achieved positive results in its Sclerotinia resistance program, demonstrating multiple modes of action against the disease in canola [1][4] - The USDA-APHIS has designated Cibus' canola disease resistance traits as not regulated, reinforcing the regulatory status of its RTDS technologies [1][2][7] - The company is expanding its trait development pipeline, with successful field trials and customer agreements in rice, including collaborations with major rice seed companies [5][7] Financial Performance - For Q1 2025, Cibus reported revenue of $1.034 million, up from $0.545 million in the same period last year [23][24] - Research and development expenses were $11.8 million, slightly down from $12.0 million year-over-year, while selling, general, and administrative expenses increased to $9.9 million [8][24] - The net loss for the quarter was $49.4 million, compared to $27.0 million in the previous year, largely due to a $21.0 million goodwill impairment [8][9][24] Regulatory and Market Progress - The California Rice Commission approved the planting of gene-edited rice, marking a significant milestone for Cibus in the U.S. market [7][9] - EU member states have endorsed a negotiating mandate on the regulation of plants obtained by New Genomic Techniques, facilitating discussions for legislative adoption [1][7] - Cibus plans to deliver initial traits to customers in California and Latin America by mid-2025 and the end of 2025, respectively [7][9]

Core Insights - CRISPR Therapeutics AG reported topline results from a Phase 1 trial for CTX310, targeting ANGPTL3 to address atherosclerotic heart disease risk factors [1][4] - The trial involved 10 patients across four cohorts, showing dose-dependent reductions in ANGPTL3, triglycerides (TGs), and low-density lipoprotein (LDL) [2][9] - CTX310 demonstrated a favorable safety profile with no severe adverse events reported [3] Trial Details - The trial included lean body weight-based doses of DL1 (0.1 mg/kg), DL2 (0.3 mg/kg), DL3 (0.6 mg/kg), and DL4 (0.8 mg/kg) with at least 30 days of follow-up for each participant [2] - A DL4 patient experienced an 82% reduction in triglycerides from a baseline of 1073 mg/dL, while a DL3 patient had an 81% reduction in LDL-C from a baseline of 256 mg/dL [9] Financials and Market Response - As of March 31, CRISPR reported cash, cash equivalents, and marketable securities totaling $1.85 billion [4] - Following the announcement, CRSP stock increased by 2.99% to $34.15 [7] Analyst Commentary - Chardan Research lowered the price target for CRISPR from $84 to $82, maintaining a Buy rating, indicating that while the topline results show safety and activity, further data in 2H25 will be crucial for assessing competitiveness [4][5] - The topline readout did not include patient-level data, which may introduce variability, but biomarker results align with benchmarks set by RNAi assets for ANGPTL3 [5]

Core Viewpoint - Cibus, Inc. has received regulatory confirmation from Ecuador's Ministry of Agriculture and Livestock that its herbicide tolerance traits in rice (HT1 and HT3) are equivalent to those developed through conventional breeding, allowing for further product development and commercialization in Ecuador [1][2][3] Company Overview - Cibus is a leading agricultural technology company focused on developing and licensing plant traits to seed companies, utilizing its Rapid Trait Development System™ (RTDS) to enable targeted genetic changes without recombinant DNA [2][5] - The company aims to address critical productivity and sustainability challenges for farmers, with a long-term focus on major global row crops such as canola, rice, and soybean [5] Regulatory Developments - The Ecuadorian Ministry of Agriculture and Livestock has determined that Cibus' HT1 and HT3 rice traits can proceed with registration and commercialization under existing laws, avoiding restrictions that apply to transgenic plants [2][4] - This regulatory decision is expected to facilitate the introduction of Cibus' herbicide tolerance traits in Latin American markets through partnerships with companies like Interoc [3][4] Market Implications - Cibus plans to collaborate with Interoc, which has expertise in rice seed genetics, to integrate HT1 and HT3 traits into elite rice lines, providing innovative weed management solutions to growers in Latin America [3][4] - The company believes that this regulatory approval in Ecuador will pave the way for pursuing similar clearances in other key Latin American markets [4]

CAMBRIDGE, Mass., May 05, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company focused on developing transformative medicines for serious diseases, today announced that it plans to announce Q1 2025 financial results and business updates on May 12 via press release and SEC filings. As previously announced, the Company does not plan to host quarterly financial results conference calls moving forward. Additionally, Editas Medicine management will participate in the f ...