Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
Nanoscope advances gene-agnostic therapy for severe vision loss in RP
The FDA has accepted Nanoscope Therapeutics’ Biologics License Application for MOGENRY (sonpiretigene isteparvovec), an investigational optogenetic gene therapy for people with retinitis pigmentosa (RP) and severe vision loss. If approved, MOGENRY would be the first gene-agnostic treatment for this stage of RP, allowing patients to receive treatment independent of their individual mutation. RP encompasses more than 100 genes and more than 1,000 known mutations, creating a challenge for therapies designed around individual genetic causes. MOGENRY instead delivers a synthetic opsin gene to surviving bipolar retinal cells, making them sensitive to light after photoreceptor loss. The BLA is supported by positive results from the Phase 2b/3 RESTORE trial, which met its primary and key secondary endpoints for visual acuity at weeks 52 and 76, alongside longer-term data from the REMAIN follow-up study. – Bree Foster
Merck cuts another 54 jobs in New Jersey as its patent cliff approaches
Merck & Co. filed a new WARN notice trimming 54 positions at its Rahway, New Jersey headquarters in two phases, December 2026 and January 2027, just days after a separate cut of 88 employees took effect on September 4. Both are part of Merck's push to save 3 billion dollars annually by 2027 through a broader plan expected to eliminate around 6,000 jobs worldwide; Merck still employs more than 8,000 people statewide. The cuts come as Keytruda's (pembrolizumab) patent protection lapses in 2028, a drug that generated 31.7 billion dollars in 2025, nearly half of Merck's total revenue, with several pembrolizumab biosimilars already in development. Merck has also cut 163 jobs tied to a Pennsylvania manufacturing plant closure and 154 more from a North Carolina vaccine facility amid falling Gardasil demand. More broadly, 17 of the largest pharma companies cut more than 22,000 jobs in 2025 ahead of a projected 300 billion dollar wave of patent expirations, and Merck's New Jersey neighbors, including Novartis, Johnson & Johnson, BioMarin, Novo Nordisk, and Bristol Myers Squibb, have made comparable headquarters cuts in the past year. – Andrea Corona
BMS notches Phase 2 win for myeloma CAR T cell therapy
A week after Novartis and Bristol Myers Squibb (BMS) paused CAR T trials in autoimmune disease due to safety concerns, BMS announced positive topline results for their potential first-in-class directed CAR T cell therapy in the context of multiple myeloma. The therapy met the primary endpoint (overall response rate) and the secondary endpoint (complete response rate) in patients with quadruple-class exposed relapsed and refractory multiple myeloma (RRMM) who had already received four or more previous therapies. “As combination treatment regimens are now frequently used in earlier lines of therapy, an increasing number of people with multiple myeloma are quadruple-class exposed and resistant to currently available therapies earlier in the treatment journey, creating a critical need for new therapeutic approaches,” said Lynelle Hoch, President, Cell Therapy Organization at BMS in the press release. The positive results mean BMS could challenge Gilead’s CAR T cell therapy for RRMM with a PDUFA set for December 23rd. – Allison Whitten
Novartis’ RNA drug misses Phase 3 goal in myotonic dystrophy
Novartis’ investigational antibody-oligonucleotide conjugate del-desiran has failed to significantly improve hand function in a Phase 3 trial of myotonic dystrophy type 1 (DM1), putting one of the key assets from the company’s $12 billion acquisition of Avidity Biosciences under pressure. The HARBOR study enrolled around 150 patients with the progressive neuromuscular disease, which currently has no approved treatments, and found no statistically significant improvement in hand opening time, its primary endpoint. Novartis said the therapy showed evidence of clinical activity in secondary and exploratory measures, although it did not disclose the underlying data and plans to assess the full dataset before determining the program’s next steps. Del-desiran is designed to deliver an siRNA into muscle cells, where it targets toxic DMPK mRNA that drives DM1. The setback follows Friday’s failure of pelacarsen, the company’s cholesterol-lowering therapy, and the announcement of three deaths linked to its investigational CAR T therapy. – Bree Foster
Researchers uncover new bacterial pump behind antibiotic resistance
Researchers at the University of Osaka identified a previously unknown protein complex, FoeAB, that allows Streptococcus pneumoniae, a leading cause of pneumonia and meningitis, to pump the antibiotic fosfomycin out of its cells — a mechanism not previously linked to bacterial drug resistance. The team found that bacteria producing more FoeAB became more resistant to fosfomycin, then isolated the complex from a related species, Streptococcus thermophilus, and reconstituted it in artificial membranes to directly confirm it transports the drug. Using cryogenic electron microscopy, the researchers captured FoeAB in two distinct conformations, one open toward the cell's interior and one open toward the exterior, revealing the regions responsible for recognizing fosfomycin and how the pump shifts shape to draw the drug in and expel it. Antibiotic-resistant bacterial infections are already tied to more than a million deaths worldwide each year, according to a 2022 Lancet analysis, and fosfomycin has drawn renewed interest as a treatment of last resort for multidrug-resistant infections, making a newly identified export route for the drug directly relevant to how long that option remains effective. Atsushi Taguchi, the study's corresponding author, said in the press release that the discovery of a pump directly involved in antibiotic export marks a meaningful step toward understanding drug resistance more broadly. The finding also adds fosfomycin to the list of compounds known to be removed by bacterial efflux pumps, pointing to more diversity in these resistance mechanisms than previously recognized. – Andrea Corona
AstraZeneca’s breast cancer pill clears FDA
Despite a recent FDA advisory committee voting no against AstraZeneca’s SERD (selective estrogen receptor degrader) pill back in May, the FDA has now approved Etcamah in combination with a CDK4/6 inhibitor to treat first-line advanced HR-positive HER2-negative breast cancer. The approval is based on the SERENA-6 trial, in which Etcamah and a CDK4/6 inhibitor showed a reduction in the risk of disease progression or death by 56 percent compared to standard of care, but also was likely spurred by additional data that AstraZeneca submitted on ctDNA levels after the FDA’s request. “This combination provides an important new option for the one in three patients with this form of advanced breast cancer whose tumours develop ESR1 mutations before clinical or radiographic disease progression. Today’s approval will enable clinicians to promptly intervene and change therapeutic strategy at an earlier opportunity ahead of disease progression, rather than waiting until the cancer becomes harder to treat, and patient outcomes and quality of life worsen,” said Kevin Kalinsky, an investigator on the trial at the Winship Cancer Institute at Emory University, in the press release. – Allison Whitten
