Insights provided by DeciBio, a strategy consultancy focused on the life science and biopharma industry.

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Highlights & Summary

This Month: Cell Therapy M&A activity accelerates despite disappointing clinical results, Gene Therapy sees Ultragenyx complete its second Rare Pediatric Disease Priority Review Vouchers sale, Oligos show promise with a personalized mRNA cancer vaccine in melanoma, and ADCs advance with encouraging TROP2-targeted results in endometrial cancer

Happy Reading!  

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Cell Therapy

Caribou Biosciences halts CAR-T pipeline to pursue sale | Commercial

Caribou Biosciences said it will stop developing both of its allogeneic CAR-T candidates, vispa-cel and CB-011, and cut the bulk of its workforce by the fourth quarter. The board retained Wedbush Securities to explore strategic alternatives, including a merger, acquisition or asset sale, even though CEO Rachel Haurwitz said vispa-cel remains trial-ready with FDA alignment on a Phase 3 design. The move reflects the difficulty of raising capital for allogeneic CAR-T programs and shares fell soon after this announcement

Repligen completes $1.5 billion acquisition of BioLife Solutions | M&A

Repligen closed its roughly $1.5 billion acquisition of BioLife Solutions, maker of the CryoStor biopreservation media and other cell-processing tools used across 18 commercially approved cell and gene therapies. BioLife shareholders received $11.25 in cash plus 0.1442 Repligen shares apiece, with the deal split 64% stock and 36% cash. The deal will support Repligen’s top-line growth as it builds out a broader tools and consumables franchise for the cell and gene therapy manufacturing supply chain; the company’s balance sheet remains north of $300 million in cash, keeping options open to additional strategic opportunities.

Lakefront (formerly Galapagos) sells preclinical CAR-T pipeline to ACT | M&A

Lakefront Biotherapeutics, the rebranded Galapagos, completed the sale of its early-stage "armed" CAR-T pipeline, originally developed by and acquired from AboundBio, to the newly formed Armed Cell Therapeutics (ACT). Lakefront will receive an €8 million upfront payment plus up to $230 million in milestones and a low-single-digit royalty; the buyer is chaired by former Galapagos CEO Paul Stoffels and cell-therapy executive Willemijn Kremer. The deal closes out a sale process that failed to secure interest in 2025, letting Lakefront focus on its newly built T-cell engager-led immunology pipeline.

Bristol Myers Squibb discloses patient death in autoimmune CAR-T trial | Clinical Trial

Bristol Myers Squibb disclosed that a patient with highly active relapsing multiple sclerosis died of septic pneumonia, after the data cutoff and about 44 days after infusion with zola-cell, a CD19-directed CAR-T therapy, in the Phase 1 Breakfree-2 study. Presented alongside interim data at MSToronto 2026, the death followed unresolved symptoms after standard treatment. Otherwise, no additional Grade 5 events occurred, and efficacy data was also encouraging, with all 60 patients at the higher dose in the trial off other standard MS therapies (DMTs) at last follow-up.

Artiva Biotherapeutics halts AlloNK after two patient deaths, cuts 94% of staff | Clinical Trial

Artiva Biotherapeutics discontinued development of AlloNK (AB-101), after longer-term follow-up showed response durability fell short of its threshold and two patients died during the trial. One fatality was assessed as likely related to the regimen, while a second was deemed unrelated to treatment. In response, the FDA asked the company to pause enrollment and dosing. Artiva will cut about 94% of its workforce, will not start its Phase 3 trial, and has engaged Leerink Partners to evaluate alternatives including a merger, sale, licensing or wind-down, despite holding roughly $319 million in cash.

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Gene Therapy

Ultragenyx sells Sanfilippo gene therapy priority review voucher for $203M | M&A

Ultragenyx entered an agreement to sell the Rare Pediatric Disease Priority Review Voucher received following FDA approval of FAYUVI, its AAV9 gene therapy for Sanfilippo syndrome Type A, for $203 million. The transaction represents the company's second PRV sale within one week, providing additional non-dilutive capital following its recent gene therapy approvals.

Neurogene reports new AAV gene therapy data for Rett syndrome | Preclinical Data

Neurogene presented new preclinical data at the ESGCT annual congress supporting rapid and sustained expression of full-length MECP2 following single-stranded AAV delivery, the approach used in its NGN-401 gene therapy for Rett syndrome. The company also presented findings comparing transgene regulation strategies, including limitations of miRNA-based autoregulation, as it advances NGN-401 through clinical development.

4DMT outlines clinical and commercial strategy for 4D-150 | Commerical

4D Molecular Therapeutics held an investor day focused on the clinical development and potential commercialization of 4D-150, its intravitreal AAV gene therapy for wet AMD and diabetic macular edema. The program includes two Phase 3 wet AMD trials with topline data expected in 2027, alongside an additional Phase 3 program in diabetic macular edema.

REGENXBIO highlights sura-vec development in wet AMD | Clinical Trial

REGENXBIO announced an October investor event focused on surabgene lomparvovec (sura-vec), its AAV8-based anti-VEGF gene therapy being developed with AbbVie for retinal diseases. The companies have been advancing the Phase 3 ATMOSPHERE and ASCENT studies in wet AMD, with topline results anticipated in the fourth quarter of 2026.

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Oligos

Merck and Moderna’s intismeran cuts melanoma recurrence or death by 42% in Phase 3 | Clinical Trial
The first pivotal trial of a customized mRNA cancer treatment showed that intismeran, combined with Merck’s Keytruda, reduced the risk of recurrence or death by 42% versus Keytruda alone in resected melanoma. The results were presented at ESMO 2026 in Madrid, and experts are now debating how widely personalized mRNA vaccines can be deployed across tumor types.

FDA approves GSK’s bepirovirsen, the first antisense oligonucleotide for chronic hepatitis B | Regulatory
On October 20, the FDA approved GSK’s bepirovirsen (Hibsago) for use alongside existing antivirals in a subset of chronic hepatitis B patients without cirrhosis. It is the first antisense oligonucleotide approved for this indication, giving the field its first ASO option for the disease.

Novartis licenses Abogen’s mRNA T-cell engager in a deal worth up to $7.8B | Partnership
Novartis will pay $575 million upfront for rights to an mRNA-encoded T-cell engager from China’s Abogen Biosciences, with potential total value of up to $7.8 billion. The therapy prompts the patient’s own body to produce the therapeutic molecule for autoimmune disease.

Merck KGaA to acquire Silence Therapeutics for ~$1.3B | M&A
Merck KGaA agreed on October 19 to acquire Silence Therapeutics in an all-cash deal at a 130% premium to Silence’s October 16 closing ADS price. The acquisition adds divesiran, an siRNA targeting TMPRSS6 in Phase 3-ready development for polycythemia vera, following promising Phase 2 data.

Novartis takes a $1.9B valuation hit after del-desiran fails in late-stage DM1 trial | Clinical Trial
Novartis disclosed in its third-quarter filings that del-desiran, a targeted RNA-based therapy in myotonic dystrophy type 1, failed a late-stage trial and cut roughly $1.94 billion from the company’s valuation. CEO Vas Narasimhan said Novartis is still determining next steps for the program.

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ADCs

Hansoh Pharma Enters Up to $15.5B ADC Collaboration With Roche | Partnership

Roche and Hansoh Pharma signed a second ADC collaboration covering a portfolio of early-stage programs: two preclinical Hansoh ADCs plus multiple programs on Roche-proposed targets. Roche will pay $400 million upfront, $110 million on the first anniversary, and more than $15 billion in potential milestones; Hansoh leads development through Phase 1a before Roche takes over outside Greater China.

Bristol Myers Squibb’s Iza-Bren Nearly Doubles PFS in EGFR-Mutated NSCLC | Clinical Trial

Bristol Myers Squibb and SystImmune’s iza-bren, a first-in-class bispecific ADC, nearly doubled progression-free survival versus chemotherapy (11.1 vs. 5.7 months) and improved response rate (60.2% vs. 34.5%) in EGFR-mutated NSCLC, per Phase 3 data presented at ESMO 2026 and published in The Lancet.

Merck’s Sac-TMT Shows First Survival Benefit for a TROP2 ADC in Endometrial Cancer | Clinical Trial

Merck’s sac-TMT became the first TROP2 ADC to show an overall survival benefit in endometrial cancer, cutting the risk of death by 32% versus chemotherapy in Phase 3 TroFuse-005. The FDA has accepted a Priority Review BLA, with a PDUFA date of June 15, 2027.

Zai Lab’s Zoci Posts 81.8% Response Rate in First-Line Small Cell Lung Cancer | Clinical Trial

Zai Lab’s zoci, its DLL3-targeting ADC, paired with atezolizumab as a chemo-free first-line regimen for small cell lung cancer, achieved an 81.8% response rate and 81.9% six-month progression-free survival in first global Phase 1b data. A Phase 3 trial is planned by year-end 2026.

AbbVie’s Temab-A Wins Two FDA Breakthrough Therapy Designations in Colorectal and Lung Cancer | Regulatory

AbbVie’s Temab-A, a c-Met-directed ADC, received two FDA Breakthrough Therapy Designations: one with bevacizumab in refractory metastatic colorectal cancer, one as monotherapy in EGFR wild-type, c-Met-expressing NSCLC. These are its first BTDs, bringing AbbVie’s ADC portfolio to four total breakthrough designations.