Core Viewpoint - REGENXBIO Inc. has secured a non-dilutive, limited recourse royalty bond agreement of up to $250 million with Healthcare Royalty, which will enhance its cash runway and support upcoming milestones in its gene therapy pipeline [1][2][3] Financing Details - The agreement allows REGENXBIO to receive $150 million at closing, with an extension of cash runway into early 2027 [1][8] - An additional $50 million will be funded upon achieving sales milestones for ZOLGENSMA, and another $50 million will be available upon mutual agreement between the parties [5][6] Strategic Implications - This financing is aimed at advancing late-stage activities, including potential FDA approvals and data readouts for key products such as RGX-121 and RGX-202 [2][3] - The capital infusion is expected to accelerate commercial preparations and maintain REGENXBIO's leadership in rare and retinal gene therapies [2][8] Agreement Terms - HCRx will receive rights to anticipated royalty payments from ZOLGENSMA and other products, with quarterly interest payments based on royalty and milestone revenue [3][4] - The agreement does not include other potential non-dilutive funding sources, such as the sale of a Priority Review Voucher for RGX-121 [6][8] Company Background - REGENXBIO is focused on gene therapy, with a late-stage pipeline targeting rare and retinal diseases, including partnerships with Nippon Shinyaku and AbbVie [8][9] - The company has pioneered AAV gene therapy and aims to improve healthcare delivery through its investigational therapies [9]

High dose and low dose of TSHA-102 continue to be generally well tolerated with no treatment-related SAEs or DLTs in the pediatric, adolescent and adult patients treated (high dose, n=6; low dose, n=4) across both REVEAL trials as of April 10, 2025, data cutoff Program update on pivotal trial design, Rett syndrome natural history data analysis and clinical data from cohort two (high dose) and cohort one (low dose) of both REVEAL trials expected in Q2 2025 in conjunction with IRSF Rett Syndrome Scientific Me ...

Integrated solution aims to reduce time and cost to lead candidates, advancing safer and more effective gene therapies to patients faster.BURLINGTON, Mass., May 13, 2025 /PRNewswire/ -- Azenta, Inc. (Nasdaq: AZTA) today announced a strategic partnership between GENEWIZ from Azenta Life Sciences, a global provider of multiomics and synthetic solutions services, and Form Bio, a leading artificial intelligence (AI) life sciences technology company, to enhance adeno-associated virus (AAV) gene therapy developme ...

Core Insights - The company EG 427 is at the forefront of developing non-replicating HSV-1 (nrHSV-1) vector technology for neurological applications, with data to be presented at the ASGCT meeting [1][4] - The innovative dual-payload nrHSV-1 vector allows for the delivery of multiple transgenes with distinct expression dynamics, providing a significant advantage over existing vector technologies [3][6] - The company has initiated a phase 1/2 clinical study of its lead candidate, EG110A, targeting neurogenic bladder-related incontinence, marking a significant milestone in its development pipeline [4][7] Vector Technology - The nrHSV-1 vector technology enables the expression of two different transgenes controlled by independent regulatory elements, allowing for varied expression durations [2][6] - In vivo studies demonstrated that the vector can achieve high transgene expression in specific neuronal populations, with stable expression levels observed for at least six weeks post-administration [2][6] - The HERMES platform utilized by the company allows for targeted biodistribution of therapeutic proteins within the brain, addressing a range of neurodegenerative diseases [8] Clinical Development - The first patient has been treated in a clinical study of EG110A, which aims to address multiple severe bladder diseases, potentially improving patient care and reducing healthcare costs [4][7] - The ongoing research supports the company's vision of developing genetic medicines that are safe, targeted, and cost-effective for chronic neurological diseases [4][6] Future Prospects - The advancements in nrHSV-1 vector technology are expected to facilitate pipeline growth by addressing challenges in genetic medicine for prevalent pathologies [6][8] - The ability to deliver gene editing proteins and corrected genes through a single vector could revolutionize treatment approaches for autosomal dominant diseases [3][6]

RESEARCH TRIANGLE PARK, N.C., May 12, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage biotechnology company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and other treatments for ophthalmic disorders, today announced presentations related to its IRD gene therapy programs at these upcoming scientific conferences in May. Presentation Details American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting Title:Evaluation of MERTK gene therapy ...

OXB to Present at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) Oxford, UK – 9 May 2025: OXB (LSE: OXB), a global quality and innovation-led cell and gene therapy CDMO, today announces its participation in the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), taking place from 13–17 May 2025 at the Ernest N. Morial Convention Center in New Orleans, Louisiana. The Company will exhibit at Booth #1611, where delegates can meet OXB’s team of viral v ...

DALLAS, May 08, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced that it will report its financial results for the first quarter ended March 31, 2025, and host a corporate update conference call and webcast on Thursday, May 15, 2025, at 8:30 AM Eastern Time. Conference Call DetailsThursday, May 15, ...

Financial Data and Key Metrics Changes - Cryoport reported $41 million in revenue from continuing operations for Q1 2025, representing a 10% year-over-year growth and contributing to significant adjusted EBITDA improvement [10] - The company confirmed its revenue guidance for fiscal year 2025 in the range of $165 million to $172 million, indicating a 7.5% growth compared to fiscal year 2024 [13] Business Line Data and Key Metrics Changes - Life sciences services revenue increased by 17% year-over-year, driven by a 33% growth in support for commercial cell and gene therapies [10] - Life sciences services now account for 56% of total revenue, reflecting the increasing development and commercialization of cell and gene therapies [10] - The life sciences products business showed a 2% year-over-year growth, indicating demand stabilization [12] Market Data and Key Metrics Changes - Cryoport supported 19 commercial therapies and 711 clinical trials as of March 31, representing approximately 70% of cell and gene therapy trials [11] - The company anticipates up to 17 additional application filings and four therapy approvals for the remainder of 2025, indicating a robust pipeline [11] Company Strategy and Development Direction - The strategic partnership with DHL, involving the sale of Cryo PDP for an enterprise value of $195 million, is expected to enhance Cryoport's positioning in Asia Pacific and EMEA [12][13] - The partnership aims to leverage DHL's global scale and capabilities, allowing Cryoport to sharpen its focus on core life sciences service offerings in the regenerative medicine space [13] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth forecast despite potential tariff impacts, stating that they have taken steps to diversify the supply chain [14][15] - The company does not expect tariffs to significantly impact core support for clinical trials or commercial therapies [15] - Management remains optimistic about the commercial revenue ramp-up and the maturation of commercial therapies [44] Other Important Information - The company reported a significant increase in service gross margins year-over-year, with expectations for continued margin improvements [34] - The onboarding process for EntegraCell is progressing smoothly, with multiple commercial contracts already in place [65] Q&A Session Summary Question: Update on the launch of Entegrisel and client adoption - Management sees the market positively, with commercial revenue continuing to ramp and growth in clinical trials [20] Question: Contingent consideration impact on adjusted EBITDA - Management clarified that contingent consideration was backed out, and adjusted EBITDA improved significantly compared to Q1 of the previous year [29] Question: Response to potential tariffs and macro environment - Management indicated no significant tariff impact on cell and gene therapy, with continued commercial revenue growth [41][42] Question: Supply chain initiatives and margin improvements - Management stated that while they cannot quantify the impact of supply chain initiatives on margins, they expect strong gross margins to continue [54] Question: Impact of the new FDA director on therapy modalities - Management expressed optimism about the new FDA director's qualifications and the continued interest in cell and gene therapy [56] Question: Demand for new product launches - Management reported positive reception and ongoing adoption for new products, with expectations for substantial contributions in the future [62]

Cryoport (CYRX) Q1 2025 Earnings Call May 07, 2025 05:00 PM ET Speaker0 Good afternoon, and welcome to Cryoport First Quarter twenty twenty five Earnings Call. All participants will start in a listen only mode. After the speakers' presentation, there will be a question and answer session and instructions will be provided at that time. As a reminder, this call is being recorded. I will now turn the call over to your host, Todd Frommer from KCSA Strategic Communications. Please go ahead, sir. Speaker1 Thank y ...

Financial Data and Key Metrics Changes - Total revenue for the first quarter of 2025 was $10.4 million, down 8% from $11.3 million in the first quarter of 2024 [20][21] - Core revenue remained stable at $8.2 million, reflecting a slight increase of 1% year-over-year [21] - Gross margin decreased to 86% from 88% year-over-year, while non-GAAP adjusted gross margin remained stable at 83% [23] Business Line Data and Key Metrics Changes - Instrument revenue was $1.4 million, down from $1.9 million in the first quarter of 2024, impacted by cautious capital spending from customers [21][22] - License revenue was stable at $2.5 million, while processing assembly (PA) revenue increased to $3.9 million from $3.4 million year-over-year [21] - SPL program-related revenue was $2.1 million, down from $3.2 million in the first quarter of 2024 [22] Market Data and Key Metrics Changes - 57% of core revenue was derived from SPL customers, up from 53% in the first quarter of 2024, indicating a healthy balance of early-stage to clinical-stage customers [22] - The company noted a cautious environment for capital equipment purchasing decisions among customers, leading to some hesitance in capital expenditures [10][42] Company Strategy and Development Direction - The company is focused on executing its growth plan for 2025 and beyond, despite macro uncertainties [8][10] - MaxSite is committed to enhancing operational efficiency and making targeted investments to drive long-term returns [23][24] - The integration of SecureDx is progressing smoothly, allowing the company to expand its offerings in the cell and gene therapy space [12][51] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term health of the business, despite recent regulatory changes at the FDA [31][34] - The company anticipates continued strong demand in the cell and gene therapy market, with a focus on safety assessments [12][34] - Management reiterated guidance for core revenue growth of 8% to 15% for 2025, including at least $2 million from SecureDx [24][25] Other Important Information - The company is seeking shareholder approval to delist from AIM and maintain a single listing on NASDAQ [19] - MaxSite ended the first quarter with $174.7 million in cash equivalents and investments, with no debt [24] Q&A Session Summary Question: Regulatory changes and their impact - Management indicated that it is too early to assess the impact of recent FDA leadership changes, but they do not foresee any regulatory risks affecting their business [30][34] Question: Cost savings from operational review - The company expects to save approximately $100,000 annually from delisting, and has begun realizing savings from personnel reductions [35][36] Question: Changes in customer funding strategies - Management noted some constraints on capital expenditures but remains confident in their guidance for the year [42][43] Question: SecureDx acquisition integration - The integration is going well, with opportunities to expand into in vivo customers and complementary delivery systems [51][52] Question: SPL revenue performance - SPL revenue of $2.1 million exceeded expectations, with confidence in achieving the full-year guidance of $5 million [55][56] Question: Future product launches - The company has a robust product pipeline and plans to launch meaningful products this year [64][65] Question: Pipeline and portfolio management - Management noted a status quo in program rationalization among customers, with no significant expansion observed [71][73] Question: Revenue cadence for the year - Management is comfortable modeling incremental growth, particularly in the latter half of the year, based on identified opportunities [76][77]