VG801 is the first FDA IND clearance for novel mRNA trans-splicing gene therapy, delivering the full-length functional ABCA4 gene to target the genetic root cause of retinal dystrophies associated with ABCA4 mutations VG801 IND clearance expands ViGeneron’s clinical-stage pipeline to two programs, utilizing its novel gene therapy delivery technology platforms vgAAV capsid and REVeRT Selected by FDA for the Rare Disease Endpoint Advancement (RDEA) Pilot Program Dec. 18, 2024 -- ViGeneron GmbH, a

Data from lowest dose level cohorts demonstrate a favorable safety and efficacy profile Data includes complete results from cohort 1 and initial results from cohort 2 Early exercise of common stock warrants from stockholder generates cash proceeds of approximately $12.7 million Company to host conference call and webcast today at 8:00 a.m. ET / 5:00 a.m. PT Dec. 18, 2024 -- Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company, today announced interim data from

EYLEA HD demonstrated non-inferior vision gains with an every 8-week dosing regimen compared to EYLEA® (aflibercept) Injection 2 mg dosed every 4 weeks Safety data remains consistent with the known EYLEA HD and EYLEA safety profiles Supplementary biologics license application planned for submission to the U.S. Food and Drug Administration in the first quarter of 2025 Dec. 17, 2024 -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the primary endpoint was met in the Phase 3 QUASA

Duvakitug positive phase 2b results demonstrate best-in-class potential in ulcerative colitis and Crohn’s disease Primary endpoints met in ulcerative colitis (UC) and Crohn’s disease (CD), the most common forms of inflammatory bowel disease (IBD) Primary endpoint results in UC and CD for high dose represent the highest achieved with any TL1A monoclonal antibody Sanofi and Teva plan to initiate phase 3 development in IBD, pending regulatory discussions Program underscores Sanofi's Immunology lea

PDE6b-related Retinitis Pigmentosa is a rare inherited retinal dystrophy affecting up to 3,000 people in the US Symptoms often start in childhood leading to blindness by midlife with no approved therapy HORA-PDE6b has demonstrated clinically meaningful benefits in visual functions and good safety profile in PDE6b patients at 24-month follow-up from ongoing Phase I/II trial Rare Pediatric Disease Designation provides eligibility to receive a Priority Review Voucher upon future marketing authoriza

In 33 heavily pretreated NSCLC EGFR wild-type (EGFRwt) patients the combination of AFM24 and atezolizumab shows an overall response rate (ORR) of 21% (6 confirmed responses, 1 response awaiting confirmatory scan) and a disease control rate (DCR) of 76%; tumor shrinkage was observed in 48% of patients; preliminary median progression free survival (PFS) is 5.6 months and 36% of patients remain on treatment In 17 heavily pretreated NSCLC EGFR mutant (EGFRmut) patients the combination shows an ORR o

KYN-5356, an innovative, first-in-class small molecule inhibitor of KAT-II, a key enzyme in the kynurenine pathway implicated in schizophrenia, was shown to be safe and well-tolerated in the phase 1 study in healthy volunteers Initial data show clear evidence of target engagement and suggestive evidence of cognitive improvement, adding to body of evidence that supports further study of KYN-5356 in patients with cognitive impairment associated with schizophrenia (CIAS), which is among the most d

Preclinical studies with ACCG-2671 demonstrate potent target engagement, robust weight loss, favorable safety profile and PK properties supportive of once-daily dosing in humans ACCG-2671 is the most advanced oral small molecule amylin-based drug candidate, with Phase 1 study initiation expected by year end 2025 Company to host conference call today at 4:30 p.m. Eastern Time Dec. 17, 2024 -- Structure Therapeutics Inc. (NASDAQ: GPCR), a clinical-stage global biopharmaceutical company developin

Dec. 16, 2024 -- Merus N.V. (Nasdaq: MRUS), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics® and Triclonics®) for cancer, today announced that the first patient has been dosed in the Company’s phase 2 trial evaluating petosemtamab monotherapy in heavily pretreated (3L+) metastatic colorectal cancer (mCRC). Petosemtamab is a Biclonics® targeting EGFR and LGR5. The phase 2, open-label trial will evaluate the safety and antitumor activity o

Dec. 16, 2024 -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced dosing has been completed in Part A of the LIGHTHOUSE study, a Phase I/II clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a best-in-class gene therapy product candidate, leverages AAV.SPR, the company’s novel spreading capsid, to achieve