Core Viewpoint - The FDA issued a Complete Response Letter (CRL) for Ultragenyx's Biologics License Application (BLA) for UX111, a gene therapy for Sanfilippo syndrome type A, citing specific chemistry, manufacturing, and controls (CMC) observations that are resolvable [1][2] Group 1: Company Overview - Ultragenyx Pharmaceutical Inc. is focused on developing therapies for rare and ultra-rare genetic diseases, with a diverse portfolio aimed at addressing high unmet medical needs [6] - The company is led by a management team experienced in rare disease therapeutics, emphasizing time- and cost-efficient drug development [7] Group 2: Product Information - UX111 is an in vivo gene therapy in Phase 1/2/3 development for Sanfilippo syndrome type A (MPS IIIA), a rare fatal lysosomal storage disease with no approved treatment [4] - The therapy targets the SGSH enzyme deficiency, which leads to the accumulation of heparan sulfate in the brain, causing neurodegeneration [4] - UX111 is administered via a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene [4] Group 3: Regulatory Status - The FDA's CRL did not raise any issues regarding the clinical data package or clinical inspections, acknowledging the robustness of the neurodevelopmental outcome data and supportive biomarker data [3] - The company plans to address the CMC observations and resubmit the BLA, anticipating a review period of up to 6 months post-resubmission [2][3] Group 4: Disease Context - Sanfilippo syndrome type A (MPS IIIA) affects approximately 3,000 to 5,000 patients in commercially accessible areas, characterized by rapid neurodegeneration and a median life expectancy of 15 years [5] - The disease is caused by biallelic pathogenic variants in the SGSH gene, leading to a deficiency in the sulfamidase enzyme [5]

JENA, Germany, July 11, 2025 (GLOBE NEWSWIRE) -- InflaRx N.V. (Nasdaq: IFRX), a biopharmaceutical company pioneering anti-inflammatory therapeutics by targeting the complement system, today announced that it has received a written notice (the “Notice”), dated July 11, 2025, from the Listing Qualifications Department of The Nasdaq Stock Market LLC (“Nasdaq”) indicating that, for the last thirty (30) consecutive business days, the bid price for the Company’s common shares had closed below the minimum $1.00 pe ...

MiNK Therapeutics, Inc. INKT stock is trading higher on Friday, with a session volume of 20.2 million compared to the average volume of 7.5 million as per data from Benzinga Pro.The clinical-stage biopharmaceutical company developing allogeneic, off-the-shelf invariant natural killer T (iNKT) cell therapies announced the publication of yet another landmark case in Nature’s Oncogene describing a complete and durable remission in a patient with metastatic, treatment-refractory testicular cancer, following tre ...

EDINBURGH, United Kingdom, July 11, 2025 (GLOBE NEWSWIRE) -- NuCana plc (NASDAQ: NCNA) (“NuCana” or the “Company”) announced plans to change the ratio of its American Depository Shares (“ADSs”) to its ordinary shares from one (1) ADS, representing twenty-five (25) ordinary shares, to one (1) ADS representing five thousand (5,000) ordinary shares (the "ADS Ratio"). The change in the ADS Ratio is expected to become effective on or about August 8, 2025 (the "Effective Date"). For the Company's ADS holders, the ...

Core Insights - Rakovina Therapeutics Inc. has partnered with VariationalAI, which has been awarded the 2025 Emerging Company of the Year – Biotech by LifeSciencesBC, highlighting the significant progress and innovation in the biotechnology sector [1][2]. Company Overview - Rakovina Therapeutics is focused on developing innovative cancer treatments utilizing AI technologies, specifically through its proprietary Deep-Docking™ platform [6][7]. - The company aims to advance its pipeline of DNA-damage response inhibitors into human clinical trials in collaboration with pharmaceutical partners [7]. Partnership and Technology - Since the partnership began in September 2024, VariationalAI has utilized its Enki™ generative AI platform to expedite the discovery of novel DNA damage response (DDR) kinase inhibitors, particularly the KT-5000 series [3]. - The collaboration has resulted in multiple structurally novel compounds with favorable drug-like properties, showing promise as brain-penetrant agents for challenging tumors [3][4]. Recognition and Future Outlook - The recognition of VariationalAI as Emerging Biotech Company of the Year underscores the transformative impact of AI in drug discovery and the strength of the collaboration with Rakovina [4]. - The company anticipates further advancements in its AI-powered discovery pipeline, aiming to identify novel drug candidates with increased speed and precision [4][6].

Core Insights - Rakovina Therapeutics Inc. is advancing cancer therapies through AI-powered drug discovery and has partnered with VariationalAI, which has been recognized as the 2025 Emerging Company of the Year – Biotech by LifeSciencesBC [1][2]. Group 1: Partnership and Innovation - VariationalAI has utilized its Enki™ generative AI platform to accelerate the discovery of novel DNA damage response (DDR) kinase inhibitors since partnering with Rakovina in September 2024 [3]. - The collaboration has led to the development of the KT-5000 series, a new generation of synthetic small-molecule candidates targeting DDR pathways, resulting in multiple structurally novel compounds with favorable drug-like properties [3]. - Early data indicates that these candidates may serve as effective brain-penetrant agents for challenging tumors, marking a significant advancement in Rakovina's AI-powered discovery pipeline [3]. Group 2: Company Overview - Rakovina Therapeutics focuses on innovative cancer treatments, leveraging unique technologies for targeting DNA-damage response powered by AI through its proprietary Deep-Docking™ platform [6]. - The company aims to advance one or more drug candidates into human clinical trials in collaboration with pharmaceutical partners, establishing a pipeline of distinctive DNA-damage response inhibitors [7].

– Absence of neurotoxicity of any grade in low-volume disease to-date – – On track for first Biologics License Application (BLA) approved cell therapy in orphan indication relapsed/refractory AL Amyloidosis – – Potential future indication expansion planned – LOS ANGELES, July 11, 2025 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us” or ”IMMX”), a clinical-stage biopharmaceutical company developing cell therapies for AL Amyloidosis and other serious diseases, today announced a ...

MONTREAL and CHARLOTTE, N.C., July 11, 2025 (GLOBE NEWSWIRE) -- Milestone® Pharmaceuticals Inc. (“Milestone”) (Nasdaq: MIST), a biopharmaceutical company focused on the development and commercialization of innovative cardiovascular medicines, today announced the pricing of its previously announced underwritten public offering (the “Offering”) of (i) 31,500,000 of its common shares (the “Shares”), accompanying Series A common warrants (the “Series A Common Warrants”) to purchase an aggregate of 31,500,000 co ...

Exploring Strategic Partnerships to Expand Platform Beyond Reproductive Health The result is a programmable, wirelessly controlled device capable of delivering up to hundreds of individualized doses over months or years, without recharging or surgical replacement. Key Platform Features: Broad Market Potential While the initial focus is DARE-LARC1 for contraception, the flexible DARE-IDDS platform supports integration with GLP-1 analogs, anti-obesity medications, hormone therapies for areas such as diabetes, ...

BOSTON, July 11, 2025 (GLOBE NEWSWIRE) -- Cerevance, a clinical-stage biopharmaceutical company advancing multiple cell type-specific therapies for the treatment of neurodegenerative diseases and obesity, today announced upcoming presentations at the Alzheimer's Association International Conference (AAIC) 2025 being held in Toronto from July 27-31, 2025. Details of the poster presentations at AAIC 2025 are as follows: Title: CVN293, an Investigational Inhibitor of KCNK13 Targeting NLRP3-Mediated Neuroinflam ...