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Insights provided by DeciBio, a strategy consultancy focused on the life science and biopharma industry.
Highlights & Summary
Intro
This month saw strong momentum across next-gen therapeutics, driven by major clinical readouts, partnerships, and funding rounds. The Cell Therapy space was rich with trial updates from players like Bristol Myers Squibb and Kynerna Therapeutics. In Gene Therapy, Lilly’s announcement for acquisition of Adverum Biotechnologies leads a wave of M&A updates. The Oligonucleotide space was also defined by strategic deals and financings, including GSK–Empirico’s licensing agreement, Soufflé's $200M Series A funding raise, and Novartis’s $12B Avidity acquisition. Meanwhile, the ADC field remained red-hot with clinical wins and major investments, such as GSK’s $350M licensing Syndivia deal to Boehringer Ingelheim's near-$1B AimedBio commitment and Tubulis’s $361M Series C funding raise.
Happy Reading!
Cell Therapy
- Bristol-Myers Squibb Reports Phase 1 Data for CD19 NEX-T CAR-T in Autoimmune Diseases | Clinical Trial
Gene Therapy
- SparingVision Doses First Advanced Retinitis Pigmentosa Patient | Clinical Trial
- Nanoscope Shows Sustained Vision Improvements at 3 Years | Clinical Trial
- FDA Grants Orphan Designation to Stargardt Asset | Regulatory
- AviadoBio Licenses Chinese Asset for RP | Partnership
- Genethon Provides Update on Low-Dose DMD Prospect | Clinical Trial
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Oligo Therapy
- Peptide-siRNA conjugates reach the primate brain | Preclinical
Antibody-Drug Conjugates
- Padcev Plus Keytruda Reduce Risk of Death in Phase 3 MIBC Trial | Clinical Trial
- Datroway Demonstrates Unprecedented Improvements in 1L mTNBC | Clinical Trial
Cell Therapy
Bristol-Myers Squibb Reports Phase 1 Data for CD19 NEX-T CAR-T in Autoimmune Diseases | Clinical Trial
Bristol-Myers Squibb (BMS) presented preliminary Phase 1 results from its Breakfree-1 Study of the CD19-targeted CAR-T therapy BMS-986353 (CC-97540) in 71 patients across three chronic, refractory autoimmune disease cohorts: systemic sclerosis (SSc), systemic lupus erythematosus (SLE), and idiopathic inflammatory myopathies (IIM). At the time of analysis, 94% of evaluable patients remained off chronic immunosuppressive therapy, suggesting the potential for a durable “immune reset.” In the SSc cohort, patients with interstitial lung disease showed a median ~10 % improvement in predicted forced vital capacity at six months, along with meaningful skin-thickness improvements. In the SLE group, all but one evaluable patient achieved resolution of clinical symptoms by six months, and 92% remained off disease-specific immunosuppressants. The IIM cohort achieved a 91% major-or-moderate response rate, with median muscle-strength improvements of +17% at three months and +22% at six months. The safety profile was consistent with CAR-T therapy, with most cytokine release syndrome events reported as low-grade and resolving in a median of two days, while transient grade 3 neurotoxicity events resolved fully.
CARsgen Presents Early Satri-cel Data in Pancreatic Cancer Adjuvant Setting | Clinical Trial
CARsgen Therapeutics reported preliminary data from its Phase Ib registrational trial (CT041-ST-05) of satricabtagene autoleucel (“satri-cel”), a Claudin 18.2-targeted autologous CAR-T therapy administered as adjuvant treatment in patients with resected, CLDN18.2-positive pancreatic ductal adenocarcinoma (PDAC). Among treated patients (follow-up median ~6 months), only one experienced disease recurrence. In five of the six patients, the tumor marker CA19-9 declined by 51.3%–96.1% following infusion. All patients experienced grade 1–2 cytokine release syndrome (CRS) after the first infusion; one patient who received a second infusion developed grade 3 CRS with hypotension, which resolved within three days. The company plans to extend evaluation of satri-cel into adjuvant gastric and gastro-esophageal junction adenocarcinoma settings.
Kyverna Therapeutics Posts 100% Response in Phase 2 gMG Trial for KYV-101 | Clinical Trial
Kyverna Therapeutics reported interim data from the Phase 2 portion of its open-label, single-arm registrational KYSA-6 trial of KYV-101, a fully human CD19 CAR-T therapy, in six patients with moderate to severe generalized myasthenia gravis (gMG) who had failed prior immunosuppressant therapies. All six patients achieved clinically meaningful reductions in MG-ADL, QMG, and MGC scores at 24 weeks, with mean reductions of approximately 8.0, 7.7, and 12 points, respectively. All patients discontinued non-steroidal immunosuppressants, high-dose steroids, and FcRn or complement inhibitors. The therapy was well tolerated, with no Grade 3 or higher cytokine release syndrome or ICANS events reported; one patient experienced Grade 4 neutropenia that improved to Grade 1. With follow-up extending to 36 weeks, these data strengthen confidence in the upcoming Phase 3 portion of the trial planned for later this year.
Starna Therapeutics Raises $44 M to Propel In Vivo CAR-T Platform | Financing
Starna Therapeutics, a Suzhou-based biotech startup, has closed a US $44 million Series B financing round to advance its in-vivo CAR-T programs following early human data. In investigator-initiated studies, a single infusion of the company’s LNP-based in-vivo CAR-T therapy resulted in complete elimination of circulating B cells within 72 hours in a small number of lupus patients and a non-Hodgkin lymphoma patient, with only “super mild” cytokine release reported. The company plans to treat six lupus patients by year-end, transition into Phase 1 trials in China and the US in 2026, and evaluate a dual-CAR (CD19 + BCMA) approach and a circular RNA platform in upcoming studies. The financing was co-led by LYFE Capital, Lilly Asia Ventures and Hillhouse Capital, and the proceeds will support the company’s pivot from broader mRNA/LNP programs to a dedicated in-vivo CAR-T focus.
Cellipont Bioservices & Ernexa Therapeutics Partner to Drive ERNA-101 Production | Manufacturing
Cellipont Bioservices, a dedicated cell-therapy CDMO, has entered a partnership with Ernexa Therapeutics to advance ERNA-101, Ernexa’s lead allogeneic iMSC cell therapy targeting ovarian cancer. The agreement focuses on Engineering, Differentiation and Production (EDP) activities, as well as the cGMP development and scale-up of the ERNA-101 manufacturing process. ERNA-101 is designed as an off-the-shelf induced mesenchymal stem-cell (iMSC) therapy derived from engineered iPSCs, which in pre-clinical models has shown the ability to convert immunologically “cold” tumors into “hot,” increasing immune cell infiltration and suppressing tumor growth. Through the collaboration, Cellipont will leverage its manufacturing infrastructure and expertise to enable clinical-grade production in preparation for future trials. This milestone marks a key execution step for Ernexa’s iMSC platform as it moves from pre-clinical promise toward clinical stages.
Gene Therapy
Lilly to Acquire Adverum Biotechnologies | M&A
Eli Lilly will acquire Adverum Biotechnologies for up to $12.47 per share, adding Ixo-vec, a Phase 3 intravitreal gene therapy for wet age-related macular degeneration. Ixo-vec is designed as a one-time treatment to provide continuous aflibercept expression and reduce the burden of repeated anti-VEGF injections. The deal includes $3.56 per share in cash plus up to $8.91 per share in contingent value rights tied to approval and sales milestones, with closing expected in Q4 2025.
SparingVision Doses First Advanced Retinitis Pigmentosa Patient | Clinical Trial
SparingVision announced that the first patient has been dosed in its NYRVANA first-in-human clinical trial evaluating SPVN20, a gene-agnostic, intravitreal AAV-based gene therapy for retinal diseases such as advanced retinitis pigmentosa (RP). SPVN20 is designed to restore vision by reactivating dormant cone cells through delivery of a gene encoding a GIRK channel, functioning independently of the disease’s genetic cause. The multicenter, dose-escalation study will assess safety, tolerability, and preliminary efficacy over six months, with long-term follow-up through 2031, and is expanding recruitment across Belgium, France, and Ireland. The field has coalesced around ocular indications with many clinical-stage programs for various forms of RP, including NanoScope, who is advancing their rolling Biologics License Application for MCO-010 in RP.
Nanoscope Shows Sustained Vision Improvements at 3 Years | Clinical Trial
Nanoscope Therapeutics reported positive three-year follow-up results from the REMAIN study of its optogenetic therapy MCO-010 in patients with severe vision loss from retinitis pigmentosa. Participants maintained an average three-line vision gain from baseline through 152 weeks with no serious ocular adverse events, supporting durable efficacy and safety after a single intravitreal dose. The data form the basis of Nanoscope’s rolling BLA.
FDA Grants Orphan Designation to Stargardt Asset | Regulatory
AAVantgarde Bio announced that the FDA has granted Orphan Drug Designation to AAVB-039, its investigational gene therapy for Stargardt disease caused by biallelic ABCA4 mutations, and that the UK MHRA has approved its Clinical Trial Authorization. The therapy enables expression of the full-length ABCA4 protein to address the root genetic cause of the disease and holds Fast Track Designation in the U.S. AAVB-039 is currently being evaluated in the Phase 1/2 CELESTE trial to assess safety, tolerability, and preliminary efficacy.
AviadoBio Licenses Chinese Asset for RP | Partnership
AviadoBio and UgeneX Therapeutics announced an exclusive option and license agreement for UGX-202, an investigational AAV-based optogenetic gene therapy in clinical development for retinitis pigmentosa, with a second indication expected to enter the clinic by year-end. UGX-202 reprograms surviving retinal cells to restore light sensitivity and vision regardless of genetic cause. Under the deal, AviadoBio can obtain worldwide rights outside Greater China, with UgeneX eligible for up to $413 million in milestones and royalties. The partnership expands AviadoBio’s portfolio into retinal diseases alongside its neurodegenerative programs.
Genethon Provides Update on Low-Dose DMD Prospect | Clinical Trial
Genethon announced it will present long-term follow-up data from the dose-escalation phase of its Phase 1/2/3 trial of GNT0004, an AAV-based gene therapy for Duchenne muscular dystrophy (DMD), at the ESGCT 2025 Annual Congress. The company has begun dosing in the Phase 3 trial, supported by earlier safety and efficacy results at a 3x lower dose than competing DMD programs. Genethon will also deliver three additional oral presentations including clinical data on overcoming AAV8 immunity using Hansa Biopharma’s imlifidase pretreatment in Crigler-Najjar syndrome.
Oligo
GSK and Empirico enter license agreement for respiratory disease therapy | Partnerships
GSK signed a worldwide exclusive license for Empirico’s siRNA EMP-012, now in phase I for COPD and designed to enable longer dosing intervals via proprietary chemistry. The deal includes $85 million upfront and up to $660 million in milestones, with Empirico running the ongoing phase I before GSK takes over development and commercialisation; royalties are tiered on global net sales. EMP-012 targets a distinct inflammatory pathway with potential utility across COPD subtypes, including non-type 2 inflammation, and can be used as monotherapy or in combinations within GSK’s respiratory pipeline.
Ribo granted EMA Orphan Drug Designation for hepatitis D siRNA drug | Regulatory
The European Medicines Agency granted Orphan Drug Designation to Suzhou Ribo Life Science’s siRNA candidate RBD1016 for the treatment of hepatitis delta virus (HDV) infection. RBD1016 is designed to selectively silence key viral factors involved in HDV and is currently being evaluated in a Phase 2a clinical trial in Sweden (NCT06649266). The designation provides development incentives, including market exclusivity upon approval.
RNA biotech Soufflé raises $200M Series A backed by pharma and VC | Financing
Soufflé Therapeutics emerged with a $200 million series A to develop targeted, cell-specific siRNA medicines, co-founded by Robert Langer with Susan Langer as founding president and CBO and Amir Nashat as CEO. The company cites more than $3.5 billion secured across fundraising and partnerships, lists Big Pharma partners including AbbVie, Amgen, Bayer and Novo Nordisk, and counts investors such as Bessemer, Arch, Vida and Polaris. Lead programs aim to enter the clinic in 2026 across metabolism, facioscapulohumeral muscular dystrophy, and phospholamban-mutation cardiomyopathies, supported by an experienced RNA leadership team from Merck, J&J, Third Rock, Alnylam and CRISPR networks.
Wave Life Sciences shows positive clinical data for obesity drug WVE-007 | Clinical Trial
WVE-007, a GalNAc-siRNA targeting INHBE, was generally safe and well tolerated in the recent INLIGHT clinical trial, and the independent monitoring committee backed expanding to a 600 mg cohort and further escalation. Wave reported dose-dependent, single-dose reductions in circulating Activin E at day 29 in its INLIGHT obesity study: 56% at 75 mg, 75% at 240 mg, and 85% at 400 mg, with durability through six months in the lowest dose cohort. Additional INLIGHT readouts will include body composition and weight metrics beginning in Q4 2025.
Peptide-siRNA conjugates reach the primate brain | Preclinical
ProteinQure reported non-human primate data showing efficient CNS delivery and widespread brain distribution following intrathecal dosing of proprietary peptide–siRNA conjugates that use receptor-mediated transport. In a 28-day head-to-head study versus leading lipid-based CNS delivery platforms in clinical development, the conjugates were described as competitive and superior in certain cell types. The company will present these data, plus early results from its blood-brain barrier shuttle program, at the Oligonucleotide Therapeutics Society Annual Meeting in Budapest in October 2025.
Novartis strengthens neuro pipeline with $12B Avidity acquisition | M&A
Novartis agreed to acquire Avidity Biosciences for USD 72.00 per share in cash ($12B equity value; $11B enterprise value), with closing expected in H1 2026 subject to regulatory and shareholder approvals and completion of an Avidity SpinCo separation for early-stage precision cardiology assets. The deal adds Avidity’s muscle-directed Antibody Oligonucleotide Conjugates (AOCs) platform and late-stage programs in DM1, FSHD, and DMD to Novartis’ neuroscience franchise, with planned product launches before 2030. Novartis raised its expected 2024–2029 sales CAGR from +5% to +6% on the back of the transaction. Avidity shareholders are slated to receive SpinCo consideration via a distribution (one SpinCo share per ten Avidity shares) and/or pro rata cash from any pre-close SpinCo sale.
ADCs
GSK Licenses ADC from Syndivia in a Deal Worth up to $350M | Commercial
GSK has acquired exclusive worldwide rights from Syndivia to a pre-clinical antibody-drug conjugate (ADC) candidate targeting metastatic castration-resistant prostate cancer. The ADC uses Syndivia’s GeminiMab conjugation platform and showed strong tumor-shrinkage in pre-clinical studies without a corresponding increase in significant side-effects, supporting best-in-class potential. Under the deal, GSK takes full responsibility for development, manufacturing and global commercialization, while Syndivia will receive an upfront payment, milestone payments up to £268 million (~$350 million) and tiered royalties. The acquisition strengthens GSK’s solid-tumor oncology pipeline and complements its existing B7-H3-targeting ADC program.
Padcev Plus Keytruda Reduce Risk of Death in Phase 3 MIBC Trial | Clinical Trial
Pfizer and Astellas Pharma reported that the combination of their ADC PADCEV™ (enfortumab vedotin) and immunotherapy KEYTRUDA™ (pembrolizumab) significantly reduced the risk of recurrence, progression or death by 60% and the risk of death by 50% in cisplatin-ineligible patients with muscle-invasive bladder cancer when given before and after surgery. The Phase III EV-303 (KEYNOTE-905) trial showed that the median event-free survival was not yet reached for the combination arm versus 15.7 months for surgery alone, and roughly 74.7% of treated patients were event-free at two years compared to 39.4% in the surgery-alone group. Overall survival data showed about 79.7% of patients on the combination were alive at two years compared to 63.1% in the control arm. The safety profile was consistent with prior studies, and grade 3+ adverse-events occurred in ~71% of patients on the combination versus ~46% with surgery alone. The encouraging survival results show potential for this combination to impact standard of care.
Tubulis Raises $361M Series C to Accelerate Development of Lead ADC Candidates | Financing
Tubulis has closed a €308 million ($361 million) Series C financing round, led by Venrock Healthcare Capital Partners with participation from new investors such as Wellington Management and Ascenta Capital alongside existing backers. The proceeds will be used to advance its lead ADC candidate TUB040—which targets NaPi2b and is being evaluated in a Phase I/IIa trial in platinum-resistant ovarian cancer and relapsed/refractory non-small cell lung cancer—and expand into earlier lines of treatment and additional tumor indications. The funding will also support the clinical-stage ADC candidate TUB030, various preclinical programs, and further development of Tubulis’ proprietary ADC platform technologies. This fundraising marks the largest Series C ever for a European biotech company and the largest financing round for a private ADC developer globally. The company aims to leverage this capital to accelerate its solid-tumor ADC pipeline and establish itself as a leader in the ADC space.
Boehringer Ingelheim Bets Nearly $1B for ADC from AimedBio | Commercial
Boehringer Ingelheim has entered a deal with AimedBio worth up to $991 million to license an antibody-drug conjugate (ADC) candidate, signaling the company’s strong commitment to expanding its oncology portfolio. The agreement includes an upfront payment along with development, regulatory, and commercial milestone payments, and AimedBio will also receive royalties on future net sales. Specific details about the ADC asset, including its target and indication, were not disclosed. The collaboration aligns with Boehringer Ingelheim’s broader strategy to strengthen its cancer pipeline through partnerships and investments in next-generation ADC technologies. This move highlights the company’s growing focus on ADCs as a central pillar of its oncology innovation strategy.
Datroway Demonstrates Unprecedented Improvements in 1L mTNBC | Clinical Trial
Datroway (datopotamab deruxtecan) demonstrated a 5-month improvement in median overall survival compared to chemotherapy (23.7 months vs 18.7 months) in the Phase III TROPION‑Breast02 trial for first-line treatment of metastatic triple-negative breast cancer (TNBC) patients not eligible for immunotherapy. The trial also met its second primary endpoint: median progression-free survival was 10.8 months with Datroway versus 5.6 months with chemotherapy. This marks the first time any therapy has shown a statistically significant overall survival benefit over standard chemotherapy in this high-risk patient population. The safety profile was consistent with prior Datroway studies, and the companies are preparing regulatory submissions. These results position Datroway as a potential new standard of care in the first-line setting for immunotherapy-ineligible metastatic TNBC.
