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GSK will enlist regulatory RNA biotech Camp4 Therapeutics to help find and create new neurodegenerative and kidney disease medicines. The deal is worth $17.5 million upfront, and as much as $440 million in development and commercial payments. The partners will work on antisense oligonucleotide (ASO) drug candidates for various genetic targets. “Protein under-expression plays a critical role in diseases such as neurodegenerative and kidney disease,” Camp4 CEO Josh Mandel-Brehm said in a press release. He said the collaboration will focus on the “rapid identification of novel targets and ASO treatments that increase the expression of validated genetic targets.” The Cambridge, MA-based drug developer expects to enter the clinic next year with CMP-002, its in-house program for neurodegenerative ailments caused by a genetic mutation, known as SYNGAP-related disorders. It has also been looking for a partner for CMP-001, its urea cycle disorders drug candidate that’s in Phase 1. GSK has executed a slew of partnerships this year with LTZ , Empirico , Quotient, ProFound, Hengrui Pharma , Chugai and others. Similarly, Camp4 is working with BioMarin, Fulcrum Therapeutics and Eli Lilly. It previously had pacts with Biogen and Alnylam. It went public in October 2024 in a $75 million IPO .

Sana Biotechnology shifted its plans for SC291 last year.\n Sana Biotechnology, CAMP4 Therapeutics and Crinetics Pharmaceuticals have all used the third quarter to refocus their pipelines on the most promising candidates.For Sana, that means halting work on a pair of HIP-modified allogeneic CAR-T cell therapies. One of these, dubbed SC291, was being evaluated in a phase 1 study for autoimmune disease, namely systemic lupus erythematosus and anti-neutrophil cytoplasmic antibody-associated vasculitis.The other asset, called SC262, is a CD22-directed CAR-T Sana had been assessing in a phase 1 study for patients with non-Hodgkin lymphoma.In its earnings release, the biotech pointed to research it recently published highlighting the potential of HIP-modified cells to evade immune attack. But Sana explained that work on the allogeneic CAR-T candidates must be sacrificed to keep funding flowing to SC451, a preclinical HIP-modified stem-cell-derived pancreatic islet cell therapy for Type 1 diabetes, as well as its fusogen platform for optimizing in vivo cell-specific delivery of genetic material.Sana CEO Steve Harr, M.D., explained that the goal for SC451 is to become a single treatment for Type 1 diabetes that results in “normal blood glucose with no need for further insulin treatment or immunosuppression.”“Separately, we have increased the potency of [our] in vivo CAR-T platform, and based upon preclinical data, believe we have the opportunity to develop best-in-class therapies with a single treatment and no conditioning chemotherapy for a range of B-cell cancers and B-cell mediated autoimmune diseases,” the CEO said.“Based upon our momentum and given the resources required to fully exploit these opportunities, we have made the difficult decision to suspend our allogeneic CAR-T programs, including SC291 and SC262,” Harr added.The biotech already shifted its plans for SC291 last year when the company halted development in cancer in order to focus the asset on B-cell-mediated autoimmune diseases. The restructuring also saw Sana end work on its pluripotent-stem-cell-derived glial progenitor cell product that was in preclinical development for central nervous system disorders. It was a similar story over at CAMP4, where the biotech is going all-in on getting CMP-002, a treatment for SYNGAP1-related disorders, into the clinic. That means the company will deprioritize CMP-001, its clinical-stage antisense oligonucleotide for urea cycle disorders.“As we prioritize our SYNGAP1 lead program, we have made a strategic decision to pursue partnerships for further development of CMP-001,” CAMP4 CEO Josh Mandel-Brehm said in an earnings release.“We continue to believe CMP-001 has potential to be the first disease-modifying therapy for the most prevalent urea cycle disorders and were encouraged by the safety and pharmacokinetics data we observed in our phase 1 SAD/MAD clinical trial,” Mandel-Brehm added.Commenting on CAMP4’s decision, analysts at William Blair acknowledged that the phase 1 data for CMP-001 in healthy volunteers “demonstrated sound safety and pharmacokinetics.”However, the analysts said they “view the variability in ureagenesis data as somewhat disappointing given we would like to see clinical validation that targeting RegRNA can impart pharmacodynamic changes.”Another biopharma tweaking its pipeline yesterday was Crinetics, which has decided to take its thyroid-stimulating hormone receptor (TSHR) antagonist back to the drawing board. After mulling data emerging from preclinical studies, the company said it has “shifted to bringing forward an alternative candidate with a superior profile.”Until now, the TSHR program had revolved around CRN12755 (PDF), a selective oral TSHR antagonist aimed at thyroid eye disease and the autoimmune disorder Graves’ disease.