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40+ results for “AAV9 FOLR1 gene therapy cerebral folate deficiency”
- Regulatory & PolicyCMS finalizes scaled-back international drug pricing model for Medicare Part BThe Centers for Medicare & Medicaid Services (CMS) has finalized the Global Benchmark for Efficient Drug Pricing (GLOBE) Model, a mandatory Medicare Part B...
- BusinessFoghorn cuts 40% of staff as Lilly pulls out of FHD-909 program Foghorn Therapeutics (Nasdaq: FHTX) and Lilly have decided not to advance FHD-909 (LY4050784), a selective SMARCA2 ATPase inhibitor, into the clinical...
- Regulatory & PolicyFDA seeks fresh scientific and consumer expertise for advisory committeesThe FDA launched a nationwide recruitment drive on September 30, 2026 to fill current and anticipated vacancies across its advisory committees, ahead of the...
- Regulatory & PolicyPierre Fabre, Atara resubmit Ebvallo BLA after two FDA CRLsPierre Fabre Pharmaceuticals, with support from Thousand Oaks, California-based Atara Biotherapeutics (Nasdaq: ATRA), resubmitted a Biologics License...
- DevelopmentProgentos takes oral remyelination drug PRO-1562 into first human MS trialCambridge, Massachusetts-based Progentos Therapeutics has registered a Phase I/II first-in-human study of PRO-1562, an oral, central nervous system...
- Regulatory & PolicyFDA expands Bristol Myers Squibb's Camzyos into pediatric obstructive hypertrophic cardiomyopathyBristol Myers Squibb (NYSE: BMY) won US FDA approval for an expanded indication for Camzyos (mavacamten), a selective cardiac myosin inhibitor, covering...
- BusinessBioMarin taps Profluent AI models for novel enzyme therapiesEmeryville, California-based Profluent and BioMarin Pharmaceutical (Nasdaq: BMRN) entered a research collaboration under which Profluent will apply its AI...
- BusinessSanofi commits up to USD 8b for Regeneron’s long-acting immunology antibodiesRegeneron Pharmaceuticals (Nasdaq: REGN) and Sanofi (Euronext Paris: SAN; Nasdaq: SNY) have expanded their global antibody alliance to co-develop and...
- BusinessWave lands USD 12m as GSK advances SpiNA siRNA programCambridge, Massachusetts-based RNA specialist Wave Life Sciences, Inc. (Nasdaq: WVE) revealed that GSK has selected an undisclosed liver disease candidate...
- DevelopmentLilly's Ebglyss wins Phase IIIb trial in hand and foot atopic dermatitis, seeks label expansionEli Lilly and Company's Ebglyss met its primary and secondary endpoints in the Phase IIIb ADtouch study in adults and adolescents with moderate-to-severe...
- DevelopmentLilly’s retatrutide delivers 20.8% weight loss in Phase III diabetes-obesity trialEli Lilly and Company (NYSE: LLY) reported detailed Phase III data from TRIUMPH-2, one of the studies supporting its planned US regulatory filing for...
- DevelopmentVividion’s WRN inhibitor shows first clinical proof of synthetic lethality in MSI-high cancersFirst peer-reviewed clinical data for San Diego-based Vividion Therapeutics, Inc.'s WRN inhibitor VVD-214 showed antitumor activity and prolonged disease...
- Regulatory & PolicyRoche’s fenebrutinib gets FDA priority review for RMS and PPMSRoche had its New Drug Application accepted by the US FDA under priority review on September 30, 2026, for investigational fenebrutinib in relapsing...
- BusinessAnaCardio raises EUR 62.5m to push ghrelin agonist AC01 toward Phase IIISweden-based AnaCardio has raised EUR 62.5 million in a Series B financing co-led by Novo Holdings and the Ljungström family office to advance lead...
- DiscoveryNIH backs Jillion’s oral small-molecule tau program with USD 1.12m awardBrooklyn, New York-based Jillion Therapeutics Inc. received a USD 1.12 million Phase II Small Business Innovation Research (SBIR) award from the National...
- Business4BIO Capital leads EUR 40m seed round for Aptadir's gene-reactivating RNA platformMilan-based Aptadir Therapeutics raised EUR 40 million (USD 45 million) in seed financing led by 4BIO Capital to advance its pipeline of disease-modifying...
- BusinessImmix Bio offering to raise USD 125m after 89% CR rate for BCMA CAR T in amyloidosisLos Angeles-based Immix Biopharma, Inc. (Nasdaq: IMMX) priced a USD 125 million underwritten public offering to support development of its BCMA-directed...
- DiscoveryMD Anderson study identifies two mechanisms shaping allogeneic CAR T successResearchers at The University of Texas MD Anderson Cancer Center have identified two distinct mechanisms governing whether allogeneic CAR T cells are...
- DevelopmentHutchMed puts GSK-partnered KRAS-EGFR conjugate into Phase I in ChinaChina-based HUTCHMED (China) Limited (Nasdaq/AIM: HCM; HKEX: 13) dosed the first patient in a global Phase I trial of HMPL-A830, the KRAS-EGFR...
- DevelopmentArvinas enters immuno-oncology with HPK1 PROTAC degrader in Phase I/II trialArvinas, Inc. (Nasdaq: ARVN) dosed the first patient in a Phase I/II trial of ARV-6723, marking the New Haven, Connecticut-based company's entry into...
- DevelopmentIASO Bio’s BCMA/GPRC5D dual-targeted CAR-T RD140 shows 88.9% response rate in Phase IIASO Bio, a biopharmaceutical company with operations in Shanghai, Nanjing, and California, presented preliminary Phase I data for RD140, a fully human...
- Regulatory & PolicyAlume wins FDA Breakthrough Therapy Designation for nerve-visualization agent bevonesceinSan Diego-based Alume Biosciences received US FDA Breakthrough Therapy Designation for bevonescein (ALM-488), an investigational fluorescent peptide-dye...
- Regulatory & PolicyNutshell Therapeutics wins FDA IND clearance for NRF2 degrader NTS231Nutshell Therapeutics (Shanghai) Co., Ltd. received US FDA clearance for an investigational new drug application covering NTS231, a covalent allosteric...
- DevelopmentAdicet’s prula-cel shows 50% complete renal response rate in Phase I lupus nephritis studyAdicet Bio (Nasdaq: ACET) reported that its allogeneic gamma delta CAR-T therapy prulacabtagene leucel (prula-cel) produced complete renal responses in 50%...
- DevelopmentChugai resurrects emugrobart in SMA after Roche exits following obesity failureJapan-based Chugai Pharmaceutical (Tokyo: 4519) is preparing to resume development of emugrobart (GYM329) in spinal muscular atrophy (SMA) after Roche ended...
- DevelopmentMirum's brelovitug aces Phase III in chronic hepatitis delta virus with 56% response rateMirum Pharmaceuticals (Nasdaq: MIRM) reported that the Phase III portion of the AZURE-1 study of brelovitug, an anti-hepatitis B surface antigen (HBsAg)...
- BusinessADARx raises USD 446m in upsized Nasdaq IPOSan Diego-based ADARx Pharmaceuticals priced an upsized initial public offering of 26.25 million shares at USD 17 per share, raising approximately USD 446.3...
- DevelopmentCarvykti sustains five-year remissions in half of early-line relapsed myeloma patientsJohnson & Johnson (NYSE: JNJ) reported five-year follow-up data from the Phase II CARTITUDE-2 study showing that half of patients with early-line relapsed...
- BusinessTolerance Bio adds to thymus pipeline with anti-IL-33 antibody license from Tanabe Philadelphia-based Tolerance Bio, Inc. has licensed TLB-33 (MT-2990), a clinical-stage anti-IL-33 monoclonal antibody, from Japan-based Tanabe Pharma...
- Regulatory & PolicyAstraZeneca wins Priority Review for Imfinzi-EV regimen in bladder cancerThe US FDA granted Priority Review to AstraZeneca’s supplemental BLA for perioperative Imfinzi (durvalumab) with neoadjuvant enfortumab vedotin...
- Regulatory & PolicyAbbVie lands FDA approval for Cerevel-origin Parkinson’s drug JuvmoAbbVie (NYSE: ABBV) received US FDA approval for Juvmo (tavapadon) for the treatment of Parkinson's disease in adults, with the drug approved for use with...
- Regulatory & PolicyUMass Chan, Mass General Brigham advance first gene therapy trial in mucolipidosis type IVThe US FDA has allowed an investigational new drug (IND) application to proceed for AAV-MCOLN1, an adeno-associated virus gene therapy for mucolipidosis...
- Regulatory & PolicyIncyte/Mirum take FDA approval for Atebrioz as first oral ALK2i for fibrodysplasia ossificans progressivaThe US FDA approved Atebrioz (zilurgisertib), an oral ALK2 inhibitor developed by Incyte (Nasdaq: INCY) and commercialized by Mirum Pharmaceuticals (Nasdaq:...
- DevelopmentFDA keeps Newron’s ENIGMA-TRS 2 study on clinical holdItaly-based Newron Pharmaceuticals (SIX: NWRN, XETRA: NP5) said the US FDA’s clinical hold on new patient enrollment at US sites in the Phase III ENIGMA-TRS...
- DiscoverynChroma Bio's epigenetic silencer therapy shows sustained HBV suppression in preclinical modelsResearchers at Boston-based genetic medicines company nChroma Bio published preclinical data in Nature Biomedical Engineering supporting CRMA-1001, an...
- Regulatory & PolicyPrime Medicine receives FDA clearance for PM647 in alpha-1 antitrypsin deficiencyCambridge, Massachusetts-based Prime Medicine, Inc. (Nasdaq: PRME) received US FDA clearance to begin first-in-human testing of PM647, a one-time in vivo...
- DiscoveryAmplifyer Bio humanized priming agent boosts ctDNA recovery up to 100-fold in miceAmplifyer Bio, an MIT and Broad Institute spinout developing agents designed to increase the amount of circulating tumor DNA (ctDNA) recoverable from blood,...
- Regulatory & PolicyFDA approves Gazyva to reduce relapse risk in pediatric idiopathic nephrotic syndromeThe US FDA approved Roche/Genentech's Gazyva (obinutuzumab) to reduce the risk of relapse in adult and pediatric patients 2 years of age and older with...
- DevelopmentIASO Bio’s in vivo BCMA CAR-T posts 90% response rate in first human studyChina-based IASO Biotechnology reported a 90% objective response rate (ORR) and 90% minimal residual disease (MRD) negativity rate in 10 patients with...
- Regulatory & PolicyDaiichi Sankyo, Merck withdraw ifinatamab deruxtecan BLA in ES-SCLCA Biologics License Application (BLA) for ifinatamab deruxtecan in extensive-stage small cell lung cancer (ES-SCLC) has been voluntarily withdrawn after...