Vertex obtains an exclusive license to TreeFrog's C-StemTM manufacturing technology in type 1 diabetes TreeFrog and Vertex to collaborate on scale-up of fully differentiated, insulin-producing pancreatic islet cells BOSTON and BORDEAUX, France, April 23, 2024 /CNW/ -- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and TreeFrog Therapeutics today announced that Vertex has obtained an exclusive license to...Read more
Comprehensive development program, including three Phase 3 clinical trials, demonstrated benefit of tislelizumab for patients with treatment-naïve and relapsed NSCLC Decision represents tislelizumab’s second approval in the region BASEL, Switzerland & BEIJING & CAMBRIDGE, Mass. / Apr 23, 2024 / Business Wire / BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global oncology company, today announced that the European...Read more
SAVITRI™ Study Met Primary Endpoint with Statistically Significant Reduction in Montgomery Åsberg Depression Rating Scale (MADRS) Total Score at Day 28 Met Key Secondary Endpoints, Including Statistically Significant Reduction in MADRS Score at Day 56 NBI-1065845 Was Generally Well-Tolerated SAN DIEGO, April 23, 2024 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX), today announced positive topline data for its Phase 2...Read more
EP262, a first-in-class oral Mas-related G protein-coupled receptor X2 (MRGPRX2) antagonist with the potential to treat a broad range of inflammatory disorders, has demonstrated proof-of-mechanism in chronic inducible urticaria and is in a Phase 2 study for chronic spontaneous urticaria EP547 is a first-in-class oral MRGPRX4 antagonist with the potential to treat cholestatic pruritus and other conditions with severe pruritus Acquisition...Read more
BOULDER, Colo. / Apr 23, 2024 / Business Wire / Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designations for sevasemten for the treatment of Becker muscular dystrophy (Becker) and for the treatment of Duchenne muscular dystrophy (Duchenne). Sevasemten is an investigational orally administered small molecule...Read more
First and only FDA-approved type II RAF inhibitor for patients with relapsed or refractory pLGG harboring a BRAF fusion or rearrangement, or BRAF V600 mutation RAPNO LGG overall response rate (ORR) of 51% Day One receives rare pediatric disease priority review voucher Conference call and webcast to be April 24, 8:30 a.m. Eastern Time BRISBANE, Calif., April 23, 2024 (GLOBE NEWSWIRE) -- Day One Biopharmaceuticals, Inc. (Nasdaq:...Read more
BOSTON and LONDON, April 23, 2024 (GLOBE NEWSWIRE) -- Centessa Pharmaceuticals plc (Nasdaq: CNTA), a clinical-stage pharmaceutical company that aims to discover and develop medicines that are transformational for patients, today announced that it has commenced an underwritten public offering of $100 million of American Depositary Shares (“ADSs”), each representing one ordinary share. All of the ADSs are being offered...Read more
Presentations include new preclinical data for PM359, further demonstrating ability of Prime Editors to efficiently, reproducibly and durably correct causative mutation of CGD; on track to enter clinical development in 2024, with initial data expected in 2025 Prime Medicine Co-Founder, David Liu, Ph.D., Awarded ASGCT Outstanding Achievement Award CAMBRIDGE, Mass., April 23, 2024 (GLOBE NEWSWIRE) -- Prime Medicine, Inc....Read more
First Data Demonstrating Application of PREDATOR™ Platform in Immune-Mediated Diseases WATERTOWN, Mass., April 23, 2024 (GLOBE NEWSWIRE) -- Werewolf Therapeutics, Inc. (the “Company” or “Werewolf”) (Nasdaq: HOWL) today announced a poster presenting preclinical data characterizing conditionally-activated IL-10 INDUKINE molecules for the treatment of colitis in animal models, a novel approach leveraging its PREDATOR™ protein...Read more
Tru Niagen® expands to health-conscious Sprouts consumers in more than 400 locations across 23 states LOS ANGELES / Apr 23, 2024 / Business Wire / ChromaDex Corp. (NASDAQ:CDXC), the global authority on nicotinamide adenine dinucleotide (NAD+) and healthy aging research, announces that its flagship product, Tru Niagen®, the number one healthy-aging NAD+ supplement in the United States†, is now available at Sprouts Farmers Market,...Read more
PHILADELPHIA, April 23, 2024 (GLOBE NEWSWIRE) -- Century Therapeutics (NASDAQ: IPSC), an innovative biotechnology company developing induced pluripotent stem cell (iPSC)-derived cell therapies in immuno-oncology and autoimmune and inflammatory disease, today announced that members of the management team will participate in a virtual fireside chat and panel at Chardan’s 8th Annual Genetic Medicines and Cell Therapy Manufacturing Summit,...Read more
Boca Raton, Florida, April 23, 2024 (GLOBE NEWSWIRE) -- INmune Bio, Inc. (NASDAQ: INMB) (the “Company”), a clinical-stage immunology company focused on developing treatments that harness the patient’s innate immune system to fight disease, is delighted to announce the successful completion of the extended stability validation for XProTM continuous storage in solution at 2-8C. The 24 and 30-month stability test samples passed...Read more
LEIDEN, Netherlands & CAMBRIDGE, Mass., April 23, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced the Annual General Meeting (AGM) of Shareholders will take place on Wednesday, May 22, 2024 at 1630 CET (10:30am EDT) at the offices of Allen & Overy LLP,...Read more
CAMBRIDGE, Mass., April 23, 2024 (GLOBE NEWSWIRE) -- Omega Therapeutics, Inc. (Nasdaq: OMGA) (“Omega”), a clinical-stage biotechnology company pioneering the development of a new class of programmable epigenomic mRNA medicines, today announced it will present a poster at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place in Baltimore, MD, May 7 – 11, 2024. The poster will feature new preclinical data...Read more
Company to Host Key Opinion Leader Event on May 8 at 1:30pm ET to Discuss Updated Results of VERSATILE-002, Unmet Treatment Needs in Advanced Head and Neck Cancer and planned Triple Combination Trial PRINCETON, N.J., April 23, 2024 (GLOBE NEWSWIRE) -- PDS Biotechnology Corporation (Nasdaq: PDSB) (“PDS Biotech” or the “Company”), a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers...Read more
REDWOOD CITY, Calif., April 23, 2024 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT) (“Rezolute” or the “Company”), a clinical-stage biopharmaceutical company committed to developing novel, transformative therapies for serious metabolic and rare diseases, today announced that a poster titled “An Analysis of Overnight Hypoglycemia in Patients with Congenital Hyperinsulinism: Results from the RZ358-606 (RIZE) Study” will be...Read more
CAMBRIDGE, Mass., April 23, 2024 /PRNewswire/ -- Flare Therapeutics Inc., a clinical-stage biotechnology company targeting transcription factors to discover precision medicines for cancer and other diseases, today announced the appointment of Douglas Manion, M.D., FRCP (C), as Chief Executive Officer, effective immediately. Dr. Manion brings over two decades of experience in pharmaceutical research and development, having held leadership...Read more
VIENNA, Va. / Apr 23, 2024 / Business Wire / CEL-SCI Corporation (NYSE American: CVM) today announced the appointment of Mario Gobbo to its Board of Directors. Mr. Gobbo has nearly 40 years of banking and corporate finance experience in healthcare and energy. His expertise encompasses venture capital and private equity as well as investment banking and strategic advisory services. He has served as an officer or director for a number of...Read more
NEW YORK, April 23, 2024 (GLOBE NEWSWIRE) -- Tiziana Life Sciences, Ltd. (Nasdaq: TLSA) (“Tiziana” or the “Company”), a biotechnology company developing breakthrough immunomodulation therapies via novel routes of drug delivery, today announced that the U.S. Food and Drug Administration (FDA) has allowed its intranasal foralumab non-active Secondary Progressive Multiple Sclerosis (na-SPMS) Expanded Access (EA) Program to expand from 10...Read more
Vancouver, British Columbia--(Newsfile Corp. - April 23, 2024) - Defence Therapeutics Inc. (CSE: DTC) (OTCQB: DTCFF) (FSE: DTC) ("Defence" or the "Company"), a Canadian biopharmaceutical company developing novel immune-oncology therapeutics and drug delivery technologies, is pleased to announce that its second-generation ARMTM anti-cancer vaccine using AccuTOX®, called ARM-002TM, is therapeutically effective against pre-established...Read more
CHICAGO / Apr 23, 2024 / Business Wire / MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA”, the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today announced that it has entered into definitive agreements for the purchase and sale of an aggregate of 494,096 shares of common stock at a purchase price of $2.034 per share, in a private placement to accredited investors and certain...Read more
SOUTH SAN FRANCISCO, Calif., April 23, 2024 (GLOBE NEWSWIRE) -- UNITY Biotechnology, Inc. (“UNITY”) [NASDAQ: UBX], a biotechnology company developing therapeutics to slow, halt or reverse diseases of aging, today announced that the ongoing Phase 2b ASPIRE study of UBX1325 has been extended from 24 to 36 weeks to assess potentially greater durability compared to aflibercept. In addition, the study is being upsized from 40 to 50 patients to...Read more
BASKING RIDGE, N.J. and JINAN, China, April 23, 2024 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, and Qilu Pharmaceutical Co., Ltd. (“Qilu”), one of the leading vertically integrated pharmaceutical companies in China that develops, manufactures,...Read more
TORONTO, April 23, 2024 (GLOBE NEWSWIRE) -- Revive Therapeutics Ltd. (“Revive” or the “Company”) (OTCQB: RVVTF) (CSE: RVV) (FRANKFURT:31R), a specialty life sciences company focused on the research and development of therapeutics and diagnostics for infectious diseases, medical countermeasures, and rare disorders, announced today that the U.S. Food & Drug Administration (“FDA”) has granted the Company’s Type C meeting request for the...Read more
MindBio scientist's sleep research published in prestigious peer reviewed scientific journal MindBio's proprietary treatment protocol using MB22001, results in longer sleep and better quality of sleep the day after microdosing VANCOUVER, BC / ACCESSWIRE / April 23, 2024 / MindBio Therapeutics Corp. (CSE:MBIO)(Frankfurt:WF6), (the "Company" or "MindBio"), a clinical stage biopharma company uniquely pioneering novel treatments for...Read more
Phase 1 trial in collaboration with Sant Joan de Déu-Barcelona Children’s Hospital (SJD) determined to have a positive outcome by the study Monitoring Committee Safety and clinical outcomes support the therapeutic potential of VCN-01 in retinoblastoma and emphasize VCN-01’s potential for use in diverse cancer indications ROCKVILLE, Md., April 23, 2024 (GLOBE NEWSWIRE) -- Theriva™ Biologics (NYSE American: TOVX), a diversified...Read more
TARRYTOWN, New York, April 23, 2024 (GLOBE NEWSWIRE) -- via IBN – PaxMedica Inc. (Nasdaq: PXMD) (the “Company”), a leading biopharmaceutical company dedicated to advancing treatments for neurological disorders, expects to take immediate action in response to an urgent request for IV suramin from the Ministry of Health (MOH) of Malawi, a country in east Africa. The request seeks emergency access to the Company’s recently completed...Read more
Intranasal Delivery of SPC-15 shows rapid and effective drug exposure for PTSD Therapy SARASOTA, FL, April 23, 2024 (GLOBE NEWSWIRE) -- Silo Pharma, Inc. (Nasdaq: SILO) (“Silo” or the “Company”), a developmental stage biopharmaceutical company focused on developing novel formulations and drug delivery systems for traditional therapeutics and psychedelic treatments, today announced positive data in the final validation report from the...Read more
Collaboration grants Palisade Bio access to cutting-edge expertise and tools in bioinformatics Partnership marks a significant value-driving milestone in Palisade Bio's mission to redefine UC treatment through targeted interventions based on PDE4-related biomarkers Carlsbad, CA, April 23, 2024 (GLOBE NEWSWIRE) -- Palisade Bio, Inc. (Nasdaq: PALI) (“Palisade”, “Palisade Bio” or the “Company”), a biopharmaceutical company...Read more
Obtained rights from Washington University to develop and incorporate human HER2 directed antibodies Complements early pipeline of novel tunable "Knob domain" antibodies for HER2 & HER3 BRIDGEWATER, NJ / ACCESSWIRE / April 23, 2024 / Tharimmune, Inc. (NASDAQ:THAR) ("Tharimmune" or the "Company"), a clinical-stage biotechnology company developing a portfolio of therapeutic candidates in inflammation & immunology, announced...Read more
SOLANA BEACH, Calif., April 23, 2024 (GLOBE NEWSWIRE) -- Artelo Biosciences, Inc. (Nasdaq: ARTL), a clinical-stage pharmaceutical company focused on modulating lipid-signaling pathways to develop treatments for people living with cancer, pain, dermatological, and neurological conditions, today announced the publication of an article, “The emerging role of fatty acid binding protein 7 (FABP7) in cancers,” in Drug Discovery Today,...Read more
Presentation on Monday, April 29th at 4:15 PM (GMT+1) HOUSTON, TX / ACCESSWIRE / April 23, 2024 / CNS Pharmaceuticals, Inc. (NASDAQ:CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers in the brain and central nervous system, today announced that John Climaco, Chief Executive Officer of CNS Pharmaceuticals will present at the LSX World...Read more
ALTO-101 demonstrated a favorable pharmacokinetic and tolerability profile; novel transdermal formulation delivered significantly greater drug exposure and fewer adverse events typically associated with PDE4 inhibitors First-in-human achievement of target exposure with proprietary transdermal patch formulation developed in partnership with MEDRx Proof-of-concept study in cognitive impairment associated with schizophrenia...Read more
SAN DIEGO, April 23, 2024 (GLOBE NEWSWIRE) -- Skye Bioscience, Inc. (Nasdaq: SKYE) (“Skye”), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel classes of therapeutic drugs that modulate the endocannabinoid system, today announced that Chief Executive Officer & Chair Punit Dhillon and other members of Skye’s executive team will present the company and its programs in obesity and...Read more
Oral presentations include updated results from clinical study of otoferlin gene therapy DB-OTO demonstrating restoration in children with profound genetic hearing loss Additional presentations cover progress in novel genetic medicine delivery systems and immune response modulation TARRYTOWN, N.Y., April 22, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that new and updated data across its...Read more
CAMBRIDGE, MA / ACCESSWIRE / April 22, 2024 / Moderna, Inc. (NASDAQ:MRNA) today announced a contract with the Ministry of Health in Brazil (Ministério da Saúde) to supply its mRNA COVID-19 vaccine as an integral part of Brazil's 2024 national vaccination campaign against COVID-19. This contract follows the Brazilian Health Regulatory Agency's (ANVISA) approval of Moderna's COVID-19 vaccine in March 2024, which encodes the spike protein of...Read more
New indication for this one-time infusion may provide patients with a treatment-free respite as early as first relapse SOMERSET, N.J. / Apr 22, 2024 / Business Wire / Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global leader in cell therapy, announced today that the European Commission (EC) has granted approval of CARVYKTI® (ciltacabtagene autoleucel, cilta-cel) for the treatment of adult patients with relapsed and...Read more
ZUG, Switzerland and BOSTON, April 22, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced an oral presentation highlighting the Company's lipid nanoparticle (LNP) approach for ocular editing will be presented at the American Society of Gene & Cell Therapy (ASGCT) 2024 Annual Meeting, taking place May...Read more
Received Fast Track Designations for both inhaled and intratumoral KB707 PITTSBURGH, April 22, 2024 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, announced today that the first patient was dosed in its Phase 1 clinical trial (KYANITE-1) evaluating inhaled KB707, a modified HSV-1 vector designed to deliver genes encoding both human interleukin-12 (IL-12) and...Read more
Designated an FDA Breakthrough Therapy, the novel immunotherapy ANKTIVA activates the body’s natural killer (NK) and killer T-cell immune system to attack tumor cells Therapy stimulates memory T cells, leading to long duration of complete response exceeding 47 months and ongoing to date, with a median duration of response yet to be determined The percentage of patients with durable responses at 12 and 24 months exceeded the benchmark...Read more
NEW HAVEN, Conn., April 22, 2024 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN), a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology, today announced the closing of its underwritten public offering of 6,451,220 of its common shares, which includes the full exercise of the underwriters' option to purchase...Read more
Selected a move-forward Phase 2 expansion dose for IDE397 monotherapy in MTAP-deletion squamous NSCLC, based on AE profile and preliminary clinical efficacy observed, including multiple partial responses by RECIST 1.1 Evaluating over 40 MTAP PDX preclinical models, squamous NSCLC was identified as the most sensitive tumor type to IDE397 monotherapy where ~50% of the models observed tumor regressions at 30mg/kg QD Over 100,000...Read more
SAN DIEGO / Apr 22, 2024 / Business Wire / Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that Health Canada has accepted its New Drug Submission (NDS) for trofinetide for the treatment of Rett syndrome, a rare neurodevelopmental disorder. Health Canada has granted Priority Review for Acadia’s submission. “Rett syndrome is a profoundly debilitating and complex neurodevelopmental disorder that presents differently across...Read more
MENLO PARK, Calif., April 22, 2024 (GLOBE NEWSWIRE) -- Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic, and neurologic disorders by modulating the effects of the hormone cortisol, today announced positive results from the open-label portion of the pivotal Phase 3 GRACE trial of its proprietary selective...Read more
SOUTH SAN FRANCISCO, Calif. / Apr 22, 2024 / Business Wire / Veracyte, Inc. (Nasdaq: VCYT), a leading cancer diagnostics company, today announced that a new study published in JCO Precision Oncology shows that, among patients undergoing Active Surveillance (AS) for prostate cancer, the Decipher Prostate Genomic Classifier is prognostic for identifying those whose disease is likely to progress. The findings make the Decipher test the only...Read more
Ziftomenib is the first investigational treatment to be granted Breakthrough Therapy Designation for NPM1-mutant AML Registration-directed trial of ziftomenib in NPM1-mutant AML on track to complete enrollment by mid-2024 SAN DIEGO, April 22, 2024 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment...Read more
BOSTON and LONDON, April 22, 2024 (GLOBE NEWSWIRE) -- Centessa Pharmaceuticals plc (Nasdaq: CNTA), a clinical-stage pharmaceutical company that aims to discover and develop medicines that are transformational for patients, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug application (IND) to initiate a Phase 1 first-in-human, clinical trial of ORX750 for the treatment of narcolepsy....Read more
Key Therapeutic Mechanisms of Clinical Activity for Autoimmune Diseases, including B cell Depletion, Tissue Infiltration and Immune Reconstitution Data, from Phase 1 Study of FT819 in Relapsed / Refractory B-cell Malignancies to be Highlighted Patient Enrollment Initiated in Phase 1 Study of FT819 Off-the-shelf, CD19-targeted, iPSC-derived CAR T-cell Therapy for Systemic Lupus Erythematosus SAN DIEGO, April 22, 2024 (GLOBE NEWSWIRE)...Read more
CAMBRIDGE, Mass. & SALISBURY, England / Apr 22, 2024 / Business Wire / KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of oral, small molecule protease inhibitors, today announced that William C. Fairey has been appointed to the Company’s Board of Directors, effective immediately. Mr. Fairey was most recently the Chief Commercial Officer...Read more
CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage gene editing company, today announced that three abstracts have been accepted for presentation, including one oral presentation, at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) being held May 7 – 11, 2024, in Baltimore, MD, and virtually. The Company is presenting pre-clinical data to support its...Read more
Raised gross proceeds of $59 million through the issuance and sale of 5,000,000 of its ordinary shares to Icelandic institutional and existing investors Becomes second company dual-listed on NASDAQ U.S. and NASDAQ Iceland Main Market, trading under the symbol “OCS” beginning April 23, 2024 Oculis will ring the Nasdaq Iceland opening bell on April 23, 2024, at 9:30 GMT Phase 2b RELIEF trial of OCS-02 (Licaminlimab) in Dry Eye Disease...Read more
LEXINGTON, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced that it will present a broad set of data related to its TRACER™ capsid discovery platform and TRACER-driven gene therapy programs across 12 oral and poster presentations at the upcoming American Society of Gene & Cell Therapy’s (ASGCT) 27th annual meeting...Read more
NGN-401 has been generally well-tolerated by three patients with multiple months of follow-up NEW YORK / Apr 22, 2024 / Business Wire / Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that initial safety and tolerability data from its ongoing Phase 1/2 gene therapy clinical trial for Rett syndrome...Read more
Lexeo Therapeutics gains intellectual property rights including current and future clinical data from ongoing Weill Cornell Medicine investigator-initiated trial of gene therapy candidate AAVrh.10hFXN (LX2006) to support regulatory discussions Together with Lexeo Therapeutics’ ongoing study of LX2006, a total of 11 participants have been treated to date; includes patients at treatment durations out to 18-months with no...Read more
GAITHERSBURG, Md. / Apr 22, 2024 / Business Wire / Cartesian Therapeutics, Inc. (NASDAQ:RNAC) (the “Company”), a clinical-stage biotechnology company pioneering mRNA cell therapy for autoimmune diseases, today announced that recently reported twelve-month follow-up data from its Phase 2a trial of Descartes-08 in patients with generalized myasthenia gravis will be featured during an oral presentation at the upcoming American Society of Gene...Read more
WALTHAM, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell therapies (TCR-T) for the treatment of patients with cancer, today announced the acceptance of one abstract for oral presentation and four abstracts for poster presentation at the upcoming American Society of Gene and Cell Therapy...Read more
CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq:GBIO) a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, today announced that six abstracts highlighting preclinical data from its cell-targeted lipid nanoparticle (ctLNP) and immune-quiet DNA (iqDNA) platforms have been accepted for presentation, including one oral presentation, at the American Society of...Read more
This novel editing approach might unlock new strategies for the treatment of metabolic and neurological diseases Non-viral circular ssDNA delivery associated to TALEN® gene editing allows high levels of gene insertion in long-term repopulating HSPCs NEW YORK, April 22, 2024 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering...Read more
Boca Raton, Florida, April 22, 2024 (GLOBE NEWSWIRE) -- INmune Bio, Inc. (NASDAQ: INMB) (the “Company”), a clinical-stage immunology company focused on developing treatments that harness the patient’s innate immune system to fight disease, today announced today that it has entered into definitive agreements for the sale and purchase of 571,592 shares of its common stock with above market warrant exercise price that...Read more
Preclinical findings showed robust anti-tumor activity of ADI-270 in multiple CD70-positive solid and hematological cancer models ADI-270 demonstrated enhanced functional potency and resilience in tumor microenvironment On track to file an investigational new drug (IND) application for ADI-270 in 2Q 2024 Preclinical data will be featured in oral presentation on May 10, 2024 REDWOOD CITY, Calif. & BOSTON / Apr 22, 2024 / Business...Read more
LEIDEN, Netherlands & CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it will present new preclinical data for its proprietary Axiomer™ RNA editing technology platform, including the first preclinical data for its AX-0810 pipeline program for cholestatic diseases targeting NTCP at the American...Read more
SAN MATEO, Calif., April 22, 2024 (GLOBE NEWSWIRE) -- Sagimet Biosciences Inc. (Sagimet, Nasdaq: SGMT), a clinical-stage biopharmaceutical company developing novel fatty acid synthase (FASN) inhibitors designed to target dysfunctional metabolic and fibrotic pathways, today announced it will host a virtual investor and analyst day on Friday, May 3, 2024 at 12:30 PM ET. To register, click here. The event will feature Stephen Harrison, MD...Read more
RICHMOND, Calif. / Apr 22, 2024 / Business Wire / Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the American Society of Gene & Cell Therapy (ASGCT) has accepted 20 Sangamo abstracts for presentation at the 27th ASGCT Annual Meeting being held May 7-11, 2024, in-person in Baltimore, MD and in a virtual format. Presentations will focus on the progression of Sangamo’s neurology-focused...Read more
Receives FDA Complete Response Letter (CRL) based on need for additional CMC information CRL did not identify deficiencies related to clinical efficacy or clinical safety data in BLA, and no new clinical studies requested by FDA to support approval Anticipates completing and submitting requested CMC information in 3Q 2024 Conference call and webcast on Tuesday, April 23, 2024 at 8:30 a.m. ET to provide details on the requested CMC...Read more
Company plans to initiate SERENITY At-Home pivotal Phase 3 safety trial with 120 mcg dose following recent meeting with FDA No FDA-approved therapies for acute treatment of agitation associated with bipolar disorders or schizophrenia in the home setting NEW HAVEN, Conn., April 22, 2024 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop...Read more
70% of Patients in the Expanded Access Program (EAP) Have Seen Measurable Clinical Improvement in Their Fatigue After Six Months of Follow-up All Patients Have Either Stabilized or Improved on Nasal Foralumab Treatment and No Patients Declined in Key Clinical Measures NEW YORK, April 22, 2024 (GLOBE NEWSWIRE) -- Tiziana Life Sciences, Ltd. (Nasdaq: TLSA) (“Tiziana” or the “Company”), a biotechnology company developing breakthrough...Read more
PHILADELPHIA, April 22, 2024 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a clinical-stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases, today announced that William Chou, M.D., president and chief executive officer, and Eden Fucci, senior vice president of technical operations, will participate in a virtual fireside chat at Chardan’s 8th Annual Genetic Medicines and Cell...Read more
Preclinical data highlights the potential of engineered macrophage cell therapy as a treatment for liver and lung fibrosis Novel Redirected Soluble Modulator technology has demonstrated the potential to enhance immune receptor signaling PHILADELPHIA, April 22, 2024 /PRNewswire/ -- Carisma Therapeutics Inc. (Nasdaq: CARM) ("Carisma" or the "Company"), a clinical-stage biopharmaceutical company focused on discovering and...Read more
Approximately 33% of all non-small cell lung cancer patients in East Asia are never smokers – a growing and unaddressed patient population. Dr. Yashushi Goto a leading lung cancer clinician-scientist at the renowned National Cancer Center of Japan will be a lead investigator. DALLAS / Apr 22, 2024 / Business Wire / Lantern Pharma Inc. (NASDAQ: LTRN), an artificial intelligence (“AI”) company developing targeted and transformative...Read more
Jaguar planning to begin commercial launch of FDA-approved Gelclair® prescription gel for oral mucositis (also called “chemo mouth”) in Q3 2024 Company to feature “Make Cancer Less Shitty” patient advocacy program at ONS Congress SAN FRANCISCO, CA / ACCESSWIRE / April 22, 2024 / Jaguar Health, Inc. (NASDAQ:JAGX) ("Jaguar") today announced that Jaguar family company Napo Pharmaceuticals ("Napo") is exhibiting at the Oncology Nursing...Read more
SAN DIEGO, April 22, 2024 (GLOBE NEWSWIRE) -- Cidara Therapeutics, Inc. (Nasdaq: CDTX) (the Company), a biotechnology company using its proprietary Cloudbreak® platform to develop drug-Fc conjugate (DFC) immunotherapies designed to save lives and improve the standard of care for patients facing serious diseases, today reported financial results for the fourth quarter and full year ended December 31, 2023 and provided an update on its...Read more
FARMINGTON HILLS, Mich., April 22, 2024 (GLOBE NEWSWIRE) -- Ocuphire Pharma, Inc. (Nasdaq: OCUP) (“Ocuphire”), a clinical-stage biopharmaceutical company focused on developing novel therapies for the treatment of retinal and refractive eye disorders, today announced that Daniel Su, M.D. will deliver a paper presentation on oral APX3330 at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting to be held May 5-9,...Read more
Presentation to highlight baseline characteristics of Spruce’s CAHmelia program evaluating tildacerfont in adult congenital adrenal hyperplasia (CAH), as an illustration of outcomes of current pediatric CAH disease management SOUTH SAN FRANCISCO, Calif. / Apr 22, 2024 / Business Wire / Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare...Read more
Nes-Ziona, Israel, April 22, 2024 (GLOBE NEWSWIRE) -- Enlivex Therapeutics Ltd. (Nasdaq: ENLV, the “Company”), a clinical-stage macrophage reprogramming immunotherapy company, today announced that the first two patients have been dosed in the Company’s multi-country, randomized, controlled Phase I/II trial evaluating AllocetraTM in up to 160 patients with moderate to severe knee osteoarthritis. The Phase I/II multi-center trial is...Read more
CAMBRIDGE, Mass., April 22, 2024 /PRNewswire/ -- NeuroSense Therapeutics Ltd. (NASDAQ: NRSN) ("NeuroSense"), a company developing novel treatments for severe neurodegenerative diseases announced today a collaboration in Alzheimer's Disease (AD) drug development with Genetika+, a leader in precision medicine for psychiatry and neurology. The multi-phase collaboration, which will commence in NeuroSense's currently ongoing Phase 2 AD...Read more
Interim readout of 22 patients shows unprecedented efficacy data compared to prior atopic dermatitis (AD) studies with biologics: 60.0% of dupilumab-experienced AD patients treated with 400mg eblasakimab weekly achieved EASI-90 (at least a 90% reduction in their Eczema Area Severity Index (EASI) score) and 66.7% achieved a vIGA score of 0 or 1 (clear or almost clear skin) after 16 weeks, versus 14.3% of patients on placebo. 20% of...Read more
CALGARY, Alberta, April 22, 2024 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANUA WKN: A3UNZ), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, is pleased to announce a research paper titled “Raising serum uric acid with a uricase inhibitor worsens PKD in rat and mouse models" has been accepted for...Read more
GLEN ALLEN, Va., April 22, 2024 (GLOBE NEWSWIRE) -- Adial Pharmaceuticals, Inc. (NASDAQ: ADIL) (“Adial” or the “Company”), a clinical-stage biopharmaceutical company focused on developing therapies for the treatment and prevention of addiction and related disorders, today announced patent number 11957664 was issued April 16, 2024 by the United States Patent and Trademark Office. This patent expands Adial’s intellectual property protection...Read more
Azitra will present two oral presentations at ASGCT (American Society of Gene and Cell Therapy) Baltimore, MD, May 7-11, 2024 related to ATR-12, Azitra’s candidate for Netherton syndrome Topical application of ATR-12 to ex vivo human skin demonstrates the potential for superior LEKTI delivery compared to topical LEKTI application Preclinical data suggests ATR-12 can significantly reduce IL-36g, a pro-inflammatory cytokine that drives...Read more
Lead product candidate, VCN-01 in combination with liposomal irinotecan demonstrated enhanced anti-tumor effects in a human pancreatic mouse xenograft The observed synergy emphasizes VCN-01’s potential in diverse chemotherapy combinations for improved efficacy in the treatment of pancreatic cancer ROCKVILLE, Md., April 22, 2024 (GLOBE NEWSWIRE) -- Theriva™ Biologics (NYSE American: TOVX), a diversified clinical-stage company...Read more
Injection in murine tumors shows the compound is well tolerated Studies support ongoing clinical trial of PH-762 as a neoadjuvant therapy for treatment of cSCC, melanoma, or Merkel cell carcinoma MARLBOROUGH, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a clinical stage biotechnology company whose proprietary INTASYL™ siRNA gene silencing technology is designed to make immune cells more...Read more
FREEHOLD, N.J., April 22, 2024 (GLOBE NEWSWIRE) -- Avalon GloboCare Corp. (“Avalon” or the “Company”) (NASDAQ: ALBT), a developer of innovative cell-based technology, cellular therapy and precision diagnostics, today announced the official launch of the KetoAir™ breathalyzer device and related accessories in the U.S. at the Hack Your Health by KetoCon 2024 Conference being held from May 31 to June 2, 2024 in Austin, Texas. KetoAir™ is a...Read more
NEWTON SQUARE, Pa., April 22, 2024 (GLOBE NEWSWIRE) -- ArriVent BioPharma, Inc., a clinical-stage company dedicated to accelerating the global development of innovative biopharmaceutical therapeutics, today announced the appointment of Kristine Peterson to its Board of Directors. Ms. Peterson brings over 30 years of biopharmaceutical leadership experience, and currently serves on the Boards of public companies Immunocore, Inc. and Enanta...Read more
Alvotech, in agreement with Teva Pharmaceuticals, has signed an agreement with a strategic partner to bring to the U.S. market the newly FDA approved high-concentration interchangeable biosimilar to Humira® The agreement broadens patient access and further commitment to availability of vital biologics in the U.S. and globally REYKJAVIK, Iceland, April 19, 2024 (GLOBE NEWSWIRE) -- Alvotech (NASDAQ: ALVO), a global biotech company...Read more
MALVERN, Pa., April 19, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that dosing is complete in the second cohort of its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA)—a modifier gene therapy candidate being developed for geographic atrophy (GA), an...Read more
WALTHAM, Mass., April 19, 2024 (GLOBE NEWSWIRE) -- TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell therapies (TCR-T) for the treatment of patients with cancer, today announced the closing of an underwritten public offering of 2,472,581 shares of its voting common stock at a public offering price of $7.1300 per share, which was equal...Read more
BERKELEY HEIGHTS, N.J., April 19, 2024 (GLOBE NEWSWIRE) -- CorMedix Inc. (Nasdaq: CRMD), a biopharmaceutical company focused on developing and commercializing therapeutic products for life-threatening diseases and conditions, today announced that the Center for Medicare & Medicaid Services (CMS) has determined that DefenCath® meets the criteria for a Transitional Drug Add-On Payment (TDAPA) in the anti-infective functional category,...Read more
GAITHERSBURG, Md., April 19, 2024 /PRNewswire/ -- YS Biopharma Co., Ltd. (NASDAQ: YS) ("YS Biopharma" or the "Company"), a global biopharmaceutical company dedicated to discovering, developing, manufacturing, and delivering new generations of vaccines and therapeutic biologics for infectious diseases and cancer, today announced its unaudited consolidated financial results for the first nine months of the fiscal year ended March 31,...Read more
Intranasal foralumab attenuated microglial activation in patients with non-active secondary progressive multiple sclerosis and progression independent of relapse (PIRA) Data presented in a platform session at the Annual Meeting of the American Academy of Neurology in Denver, Colorado NEW YORK, April 19, 2024 (GLOBE NEWSWIRE) -- Tiziana Life Sciences, Ltd. (Nasdaq: TLSA) (“Tiziana” or the “Company”), a biotechnology company...Read more
TORONTO and HAIFA, Israel, April 19, 2024 (GLOBE NEWSWIRE) -- NurExone Biologic Inc. (TSXV: NRX) (Germany: J90) (the “Company” or “NurExone”), a pioneering biopharmaceutical company, is pleased to announce that Dr. Lior Shaltiel, CEO and Director at NurExone, was invited to present on "Revolutionizing Spinal Cord Injury Treatment: Harnessing the Potential of Exosomes for Advanced Therapy in Comprehensive Approach" at the Exosomes of...Read more
| Company | Change | Last Trade |
|---|---|---|
| Pieris Pharmaceuticals | 11.85 8,113.55 | $12.00 |
| Biophytis | 9.94 4,050.71 | $10.19 |
| BeiGene | 8.18 6.25 | $139.12 |
| Regeneron Pharmaceuticals | 7.16 0.80 | $907.32 |
| Neurocrine Biosciences | 6.43 4.81 | $140.09 |
| argenx | 6.33 1.72 | $375.08 |
| Vertex Pharmaceuticals | 4.99 1.25 | $404.91 |
| Madrigal Pharmaceuticals | 4.89 2.29 | $218.38 |
| Krystal Biotech | 4.79 3.05 | $162.04 |
| Third Harmonic Bio | 3.85 53.47 | $11.05 |
| Moderna | 3.43 3.28 | $107.89 |
| SIGA Technologies | 3.32 61.03 | $8.76 |
| Nuvalent | 3.11 4.82 | $67.60 |
| Belite Bio | 2.83 8.21 | $37.31 |
| Ascendis Pharma | 2.73 1.92 | $144.83 |
Assertio is a leading commercial pharmaceutical company bringing differentiated products to patients. The Company has a robust portfolio of branded prescription products in three areas: neurology, hospital, and pain and inflammation. Assertio has grown through...
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Cue Biopharma is developing the first-ever class of therapeutics for the treatment of cancer that mimic the natural signals, or “Cues”, of the immune system. This novel class of injectable biologics selectively engages and modulates tumor-specific T cells directly within the patient’s body to transform...
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