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Insights provided by DeciBio, a strategy consultancy focused on the life science and biopharma industry.
Highlights & Summary
Shifting priorities among cell therapy companies, the first approved gene therapy for Sanfilippo syndrome type A, an FDA approval for an oligonucleotide therapy in Alexander disease, and a $2B investment from large pharma in ADCs.
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Cell Therapy
Novartis and BMS pause autoimmune CAR-T programs after patient deaths | Clinical Trial
Novartis halted eight autoimmune trials of rapcabtagene autoleucel (rap-cel), a CD19 CAR-T, across lupus, myasthenia gravis, and other indications after three deaths from immune effector cell-associated hemophagocytic syndrome (IEC-HS). BMS separately paused autoimmune trials of its own CD19 CAR-T, zolacabtagene autoleucel (zola-cel), after “transient and reversible” inflammatory events; both use rapid-manufacturing platforms.
ArsenalBio pivots entirely to in vivo CAR-T | Commercial
Arsenal Biosciences, which has raised roughly $630 million developing autologous CAR-T for solid tumors, announced a shift to focus exclusively on in vivo CAR-T across oncology and autoimmune disease. The pivot winds down clinical programs including AB-1015 (ovarian cancer) and AB-2100 (kidney cancer), leveraging the company's existing T-cell engineering, synthetic biology, and computational toolkit toward in vivo delivery instead.
BMS ends Cellares manufacturing deal after automated platform fails to scale Breyanzi production | Manufacturing
Bristol Myers Squibb terminated its up-to-$380 million manufacturing deal with Cellares after finding the CDMO's automated platform couldn't produce commercial Breyanzi at scale. The decision prompted roughly 100 job cuts at Cellares, ten months after it raised $257 million for global expansion.
Cellectis exits CAR-T, pivots to in vivo gene editing | Commercial
Cellectis' board approved a shift to become an in vivo gene editing company, discontinuing CAR-T candidates lasme-cel and eti-cel in favor of two metabolic-disease programs: HEAL-101 (base editing APOC3, for severe hypertriglyceridemia) and HEAL-201 (epigenetic editing PCSK9, for severe hypercholesterolemia), each showing 70%+ and 90%+ target knockdown preclinically, with Phase 1 China trials planned for H2 2027 and H1 2028. The pivot comes months after lasme-cel's FDA RMAT designation and extends cash runway into second-half 2028.
Cilta-cel delivers five-year treatment-free remission in half of early-line myeloma patients | Clinical Trial
New long-term follow-up from CARTITUDE-2 Cohort A, presented at the International Myeloma Society Annual Meeting, showed 50% of patients (10 of 20) treated with a single infusion of cilta-cel (Carvykti) in early-line relapsed/refractory multiple myeloma remained alive and progression-free at five years without maintenance therapy. The data builds on CARTITUDE-1 and suggests earlier-line use may increase the odds of durable, treatment-free remission.
Gene Therapy
Ultragenyx wins FDA approval for FAYUVI in Sanfilippo syndrome | Regulatory
The FDA granted full approval to Ultragenyx’s FAYUVI (rebisufligene etisparvovec-hopf), a single-dose AAV9 gene therapy for pediatric patients with Sanfilippo syndrome Type A. FAYUVI becomes the first FDA-approved treatment for MPS IIIA and Ultragenyx’s second approved gene therapy, with the approval supported by clinical and biomarker data extending to nearly eight years of follow-up.
Beacon’s laru-zova meets primary endpoint in pivotal XLRP trial | Clinical Trial
Beacon Therapeutics reported that laru-zova met the primary endpoint in the Phase 2/3 VISTA trial in X-linked retinitis pigmentosa, with 31.0% and 24.1% of patients in the high- and low-dose groups achieving a ≥15-letter improvement in low-luminance visual acuity at 12 months versus no responders in the untreated control group. Beacon plans to begin a rolling BLA submission later in 2026 based on the results.
Encoded raises $275M to advance ETX101 through pivotal development | Financing
Encoded Therapeutics raised $275 million in a Series F financing co-led by GV and another healthcare fund to fund pivotal and expansion studies of ETX101, its gene therapy for Dravet syndrome. The financing will also support commercial-scale manufacturing and development of Encoded’s broader neurological genetic medicine pipeline.
REGENXBIO reports three-year durability for sura-vec in diabetic retinopathy | Clinical Trial
REGENXBIO reported three-year follow-up from the Phase 2 ALTITUDE study of surabgene lomparvovec (sura-vec), an AAV8-based anti-VEGF gene therapy for diabetic retinopathy. At the selected dose, 60% of evaluable patients achieved a ≥2-step improvement in DRSS without additional diabetic retinopathy treatment, with no new treatment-related safety signals or intraocular inflammation observed through three years.
PTC completes acquisition of Fabry gene therapy ST-920 | M&A
PTC Therapeutics completed its acquisition of ST-920 (isaralgagene civaparvovec), a one-time AAV gene therapy for Fabry disease, from Sangamo Therapeutics. PTC expects to complete the rolling BLA submission for accelerated approval in Q4 2026; the program has received FDA RMAT, Fast Track and Orphan Drug designations.
Oligos
Ionis' Zanvastro (zilganersen) wins FDA approval as first treatment for Alexander disease | Regulatory
The FDA approved Zanvastro, an antisense oligonucleotide that reduces production of the GFAP protein, as the first disease-modifying treatment for Alexander disease in pediatric and adult patients. The approval came 19 days ahead of its PDUFA date. The pivotal trial showed gait speed stabilized in treated patients while the control group declined by 33%, and the approval marks Ionis' first independent neurology launch, following its June 2026 ex-US licensing deal with Recordati.
Moonwalk Biosciences raises $70M to advance adipose-targeted RNAi therapies | Financing
Moonwalk Biosciences closed a $70 million Series B co-led by Alpha Wave and YK Bioventures, with Eli Lilly among the participants, to advance RNAi therapies that direct gene silencing to fat tissue instead of the liver. Lead candidate MW101 targets obesity and cardiometabolic disease and is set to enter first-in-human studies in late 2027.
Envisagenics and Boehringer Ingelheim ink up to $1B+ RNA-splicing oncology deal | Partnership
Envisagenics and Boehringer Ingelheim entered a multi-year research collaboration to validate tumor-specific targets mined from alternative RNA splicing using Envisagenics' AI-powered SpliceCore platform. Envisagenics is eligible for more than $1 billion in payments, including an upfront fee, research funding, option fees and milestones. Boehringer holds an option to license selected targets for use in ADCs, T-cell engagers and multispecific antibodies.
ADARx Pharmaceuticals raises $535M in Nasdaq debut for siRNA pipeline | Financing
ADARx priced an upsized IPO at $17 a share, raising $446.3 million, plus an $89 million concurrent private placement from partner AbbVie, for combined proceeds of $535.2 million. It is the first traditional U.S. IPO by an RNAi-focused biotech in over a decade. Proceeds will fund the pivotal trial of onvuzosiran in hereditary angioedema and Phase 2 development of agazisiran.
ADCs
GSK and Hansoh's Ris-Rez wins first Phase 3 survival benefit for a B7-H3 ADC in relapsed small cell lung cancer | Clinical Trial
GSK licensor Hansoh Pharma presented full interim data from its Phase 3 trial, ARTEMIS-008, for risvutatug rezetecan (Ris-Rez) in the WCLC Presidential Symposium in Seoul, South Korea. The data demonstrated a medial overall 54% reduction in risk of death in 461 Chinese patients with relapsed SCLC. This is the first Phase 3 B7-H3 ADC data that shows a survival benefit in any tumor type, with topline data first disclosed in July, and is a rare case of an ADC beating cytotoxic standard of care on both efficacy and tolerability. GSK holds rights outside of mainland China, Hong Kong, Macau and Taiwan and is running a global Phase 3 study with pivotal data expected next year.
Note: topline data was first disclosed in July, with the full dataset presented on September 13.
Roche and MediLink's Tam-Peli matches the 54% survival gain with a cleaner lung-toxicity profile | Clinical Trial
Results from MediLink's Phase 3 TAISHAN-302 trial of tambotatug pelitecan (Tam-Peli), a B7-H3 ADC built on its TMALIN activatable-linker platform, demonstrated an almost identical result to GSK’s rival B7-H3 ADC in the same weekend. In the 451-patient China-only trial, tam-peli cut the risk of death by 54% and the risk of progression by 71%, extending median overall survival and delivering a 59.1% response rate versus 9.7% for chemotherapy. The asset came to Roche through an expanded MediLink licensing deal earlier this year, where Roche has global rights outside of China, Hong Kong, and Macau and is now planning global Phase 3 studies.
AstraZeneca invests $2 billion in Summit Therapeutics to pair ivonescimab with its ADC portfolio | Partnership
AstraZeneca took a roughly 12% equity stake in Summit Therapeutics, starting a clinical collaboration to test Summit's PD-1/VEGF bispecific ivonescimab with AstraZeneca's antibody-drug conjugates. Currently in various gastrointestinal cancer trials, the Claudin-18.2-directed ADC sonesitatug vedotin (Sone-Ve) will be the first target. Each company will contribute its own medicine and share trial costs while retaining full rights and the deal follows positive Phase 3 CLARITY-Gastric01 survival data for Sone-Ve, signaling that AstraZeneca sees bispecific combinations as the route to extending its ADC franchise into new settings.
Merck and Daiichi Sankyo pull ifinatamab deruxtecan filing after FDA rejects Phase 2 evidence | Regulatory
The two partners voluntarily withdrew their BLA for ifinatamab deruxtecan (I-DXd) in previously treated extensive-stage SCLC after FDA discussions indicated the Phase 2 IDeate-Lung01 data, showing a 48.2% objective response rate, would not support accelerated approval. The withdrawal came twelve days after two rival B7-H3 ADCs posted randomized survival data at WCLC. Patient enrollment continues in the IDeate-Lung02 Phase 3 trial however, and I-DXd is also being evaluated in Phase 3 studies for advanced/metastatic CRPC (prostate) and ESCC (esophageal) cancers.
The EMA's CHMP recommended Enhertu (trastuzumab deruxtecan) as an adjuvant monotherapy for resected HER2-positive breast cancer with residual invasive disease after neoadjuvant treatment. This builds on the Phase 3 DESTINY-Breast05 trial, in which AstraZeneca and Daiichi Sankyo's ADC cut the risk of invasive disease recurrence or death by 53% versus T-DM1, with a three-year IDFS rate of 92.4% against 83.7%. Already cleared in this setting in the U.S., Brazil, Canada, and India, an EU authorization would extend the world's best-selling ADC further into early-stage, curative-intent treatment.
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