Dec. 18, 2024 -- Tessera Therapeutics, a biotechnology company pioneering a new approach to genome engineering through the development of its Gene Writing™ and delivery platforms, announced today that it has entered into an agreement with the Bill & Melinda Gates Foundation to jointly fund the company’s in vivo program for sickle cell disease (SCD). The funding includes an initial investment, with potential total investment of up to $50 million, from the Gates Foundation intended to support the

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

December 16, 2024 Basilea Pharmaceutica Ltd, Allschwil (SIX: BSLN), a commercial-stage biopharmaceutical company committed to meeting the needs of patients with severe bacterial and fungal infections, announced today that it has entered into an exclusive distribution and license agreement with Innoviva Specialty Therapeutics, Inc., a wholly owned subsidiary of Innoviva Inc. (NASDAQ: INVA), for the commercialization of Basilea’s hospital anti-MRSA antibiotic Zevtera® (ceftobiprole) in the United

Company plans to meet with FDA regarding potential sNDA submission in 1H 2025 Call planned today at 8:30 a.m. (ET) to discuss company’s diabetes program progression Dec. 16, 2024 -- MannKind Corporation (Nasdaq: MNKD), a company focused on the development and commercialization of inhaled therapeutic products and devices for patients with endocrine and orphan lung diseases, today announced six-month results from its Phase 3 INHALE-1 study of Afrezza (insulin human) Inhalation Powder in children

— First-in-class, oral small molecule to prevent weight regain, without need for significant lifestyle modifications — Breakthrough potential to treat obesity-related comorbidities by regulating underlying dysfunction in metabolic syndrome — Program is currently in IND-enabling studies and on track to enter the clinic in Q3 2025 Dec. 16, 2024 -- Verge Genomics, a clinical-stage biotechnology company pioneering the use of artificial intelligence (AI) and human data to transform drug discovery an