SNAPSHOT

Lilly bets big on RNA exon editing with a potential $1.9 billion partnership with Ascidian

Lilly and Ascidian have entered a research collaboration focused on developing treatments for genetically driven kidney diseases using Ascidian’s RNA exon editing technology. The deal could be worth up to $1.9 billion through upfront payments, milestones, and royalties.

Why it matters: Many inherited kidney disorders have few treatment options beyond symptom management, dialysis, or transplantation. RNA exon editing aims to address the root genetic cause of disease by repairing faulty RNA instructions, potentially restoring normal protein production without permanently altering DNA.

Big picture: The agreement highlights growing pharmaceutical interest in genetic medicines. Lilly has aggressively expanded its genetics portfolio through deals involving Verve Therapeutics, MeiraGTx, Seamless Therapeutics, and Engage Bio, signaling confidence that genetic treatments are becoming increasingly practical for diseases beyond rare disorders.

Zoom in: More than 60 genetic diseases affect the kidneys and over 3.5 million Americans are estimated to have severe inherited kidney disease. Ascidian’s platform replaces damaged RNA segments (exons) rather than editing DNA directly. The companies have not disclosed initial disease targets.

Yes, but: Ascidian is already testing a treatment for Stargardt disease and previously signed a collaboration with Roche that could be worth roughly $1.8 billion.

What’s next: The partners will identify specific kidney disease targets and begin research efforts aimed at advancing potential drug candidates into development.

SNIPPETS

What’s happening in biotech today?

🤝 Deal expansion: Regeneron Pharmaceuticals has expanded its partnership with CytomX Therapeutics, paying $37 million upfront to develop conditionally active bispecific antibodies against two additional targets and securing options on up to six more programs, increasing the collaboration’s potential value to about $4 billion. The original 2022 agreement included a $30 million upfront payment and up to $2 billion in milestones, but the new deal significantly broadens the scope through additional target nominations and development, regulatory, and commercial milestone opportunities. The expansion provides a boost for CytomX following recent partnership terminations by Astellas, Bristol Myers Squibb, Moderna, and Amgen. CytomX’s technology aims to keep biologics inactive until they reach tumors, potentially reducing toxicity, and the company remains well funded with $346.7 million in cash and a runway extending into at least the second half of 2028.

🔄 Aging reboot: NewLimit, a biotech focused on reversing aspects of cellular aging, has raised $435 million in a Series C financing round led by Founders Fund after advancing its first clinical candidate much faster than anticipated. The company uses combinations of mRNA-encoded transcription factors to reprogram aging cells, with its lead therapy targeting liver cells to restore youthful function. In preclinical studies, the candidate improved liver regeneration and alcohol tolerance in older mice, and NewLimit plans to begin a phase 1 trial in fatty liver disease next year. The funding will also support programs targeting blood vessel endothelial cells, T cells, chronic kidney disease, and other age-related conditions, while expanding the company’s cellular reprogramming platform.

📉 SERD stumble: Roche remains confident in the blockbuster potential of its oral SERD giredestrant despite the phase 3 persevERA trial failing to achieve statistically significant progression-free survival (PFS) improvement in first-line HR-positive, HER2-negative metastatic breast cancer when combined with Pfizer’s Ibrance. Although the trial showed only a numerical 11% PFS benefit and no overall survival advantage, Roche sees encouraging late separation in the PFS curves and believes this supports further development in early-stage breast cancer, where longer treatment durations may yield greater benefit. The company is planning a new adjuvant phase 3 study combining giredestrant with a CDK4/6 inhibitor, building on positive results from the lidERA trial, which showed a 30% reduction in recurrence risk. Roche has also filed for FDA approval of giredestrant in both early-stage breast cancer and second-line ESR1-mutated metastatic disease, while continuing studies with CDK4/6 inhibitors Verzenio and Kisqali.

😕 Stock sulks: Celcuity’s phase 3 VIKTORIA-1 trial showed that its experimental breast cancer drug gedatolisib significantly improved outcomes in patients with PIK3CA-mutated, HR-positive, HER2-negative advanced breast cancer, with both a three-drug regimen (gedatolisib, hormone therapy, and Pfizer’s Ibrance) and a two-drug regimen (gedatolisib plus hormone therapy) reducing the risk of disease progression or death by about 50% compared with Novartis’ Piqray plus hormone therapy. Median progression-free survival reached roughly 11 months for both gedatolisib regimens versus 5.6 months for the control group. Despite these strong clinical results, Celcuity’s stock fell more than 20% because investors had expected even better performance based on earlier, smaller studies, and because the triplet regimen offered little apparent advantage over the doublet. Investigators nonetheless described the findings as potentially practice-changing, citing meaningful efficacy and a relatively favorable safety profile compared with existing PI3K-targeted therapies.

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