Gene & Cell Therapy Update: Lilly Obesity Deal, RMAT for Stargardt, XLRP Gains and More
From major pharma collaborations to pivotal trial milestones, this week’s episode covers the most important developments shaping the future of gene and cell therapy. Whether your focus is rare disease, oncology, or advanced delivery technologies, these updates spotlight the innovations driving progress. 🔬💡
In Today’s Newsletter
🤝 Remedium Bio–Lilly gene therapy collaboration in obesity & T2D [1] [US • 08 Sep 2025]
Context: Platform: Prometheus™ dose-adjustable, subcutaneous gene delivery; one-time, tunable expression.
Key point: Multi-target R&D deal; Remedium receives upfront + equity + milestones + tiered royalties (amounts undisclosed).
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.
👁️ Nanoscope’s MCO-010 gets FDA RMAT (Stargardt disease) [2] [04 Sep 2025]
https://www.cgtlive.com/view/nanoscope-rmat-designation-retinitis-pigmentosa-gene-therapy-mco-010
Context: Ambient-light activatable MCO gene therapy; prior FDA ODD/Fast Track; EMA ODDs across retinal dystrophies.
Key point: RMAT applies to Stargardt; rolling BLA for RP ongoing (primary endpoints met in Phase 2b RESTORE per company).
Implication: Regulatory/generics: Introduces competition that may affect pricing and formulary access.
👁️ Beacon’s laru-zova shows sustained Phase 2 XLRP gains (DAWN & SKYLINE) [3] [04 Sep 2025]
Context: Subretinal RPGR gene therapy; EURETINA 2025 presentations (Paris).
Key point: Durable LLVA/microperimetry improvements (9–36 mo); generally well-tolerated; pivotal VISTA uses LLVA primary.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.
🧬 uniQure AMT-191 boosts α-Gal A in Fabry; ERT stopped in Cohort A [4] [05 Sep 2025]
Context: AAV5 IV gene therapy; Phase I/IIa Cohort A (6×10^13 gc/kg), n=4; ICIEM 2025 presentation.
Key point: 27–208× α-Gal A above mean normal; all withdrew ERT with stable lyso-Gb3 through cutoff; manageable safety (SAEs noted).
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.
🧪 Ultragenyx DTX401 Phase 3: greater cornstarch reduction at Week 96 in GSDIa [5] [US • 08 Sep 2025]
Context: AAV8 liver-directed gene therapy; Phase 3 met primary at Week 48; ICIEM 2025 update.
Key point: Week 96: 61% mean daily cornstarch reduction (both immediate and crossover groups); low hypoglycemia maintained; tolerability acceptable.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.
🧫 Vyriad licenses Weizmann in vivo viral targeting tech [6] [03 Sep 2025]
Context: Exclusive license via Yeda; reversible adapter peptide to retarget viral vectors without genetic envelope edits.
Key point: Aims to preserve potency while improving specificity; integration across Vyriad’s viral/nanoparticle platforms.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.
🧫 Lyell starts Phase 3 H2H CAR-T (ronde-cel) vs liso-cel/axi-cel in 2L LBCL [7] [US • 03 Sep 2025]
Context: Ronde-cel (CD19/CD20 dual-target) with RMAT/Fast Track; PiNACLE-H2H randomized 2L LBCL; ~400 pts planned.
Key point: Primary endpoint EFS; investigator’s choice comparator (liso-cel or axi-cel); US/Australia sites initiating.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.
🫁 ImmunityBio ANKTIVA: Phase 2 NSCLC data link ALC reversal to longer OS [8] [US • 08 Sep 2025]
Context: QUILT-3.055 (single-arm) in CPI-resistant NSCLC; presented at IASLC 2025.
Key point: Subgroups with maintained higher ALC showed longer median OS (e.g., 21.1 mo in one analysis); overall mOS 14.3 mo; Phase 3 ResQ201A enrolling.
Implication: Observational/RWE: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
❤️ Capricor responds to FDA-posted CRL for Deramiocel (DMD cardiomyopathy) [9] [US • 09 Sep 2025]
Context: FDA publicly posted July 2025 CRL; company cites submitted preliminary response; awaiting Type A minutes.
Key point: Plan to share company response publicly; HOPE-3 topline expected Q4 2025 (to inform resubmission strategy).
Implication: Regulatory/generics: Introduces competition that may affect pricing and formulary access.
🇰🇷 Korea approves Yescarta as 2L DLBCL option [10] [South Korea • 09 Sep 2025]
https://www.koreabiomed.com/news/articleView.html?idxno=28893
Context: MFDS cleared axicabtagene ciloleucel for 2L DLBCL within 12 months of 1L relapse; ZUMA-7 underpins use.
Key point: Adds to Kymriah/Carvykti availability; reimbursement application submitted; access expected to expand.
Implication: Regulatory/generics: Introduces competition that may affect pricing and formulary access.
🚽 enGene hits target enrollment (100) in pivotal LEGEND cohort (NMIBC CIS) [11] [03 Sep 2025]
Context: Detalimogene voraplasmid (non-viral) in high-risk BCG-unresponsive NMIBC with CIS; open-label Phase 2.
Key point: Target enrollment reached; update planned Q4 2025; BLA timing guided to 2H 2026 (over-enrollment expected).
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.
Why it matters
Gene therapy momentum spans metabolism (obesity/T2D), ophthalmology (XLRP/Stargardt), and rare IMDs (Fabry, GSDIa).
Regulators are active: RMAT designations, Korea’s CAR-T approval, and CRL dynamics shape timelines and access.
Delivery innovation (adapter-peptide retargeting) and dual-antigen CAR-T trials aim to raise efficacy while managing safety.
Longer-term Phase 3 readouts (DTX401) emphasize functional/outcomes relevance alongside biomarkers.
📢 Stay Ahead in Cell and Gene Therapy!
✅ Contact LucidQuest at 📧 info@lqventures.com for strategic guidance on gene therapy innovations and clinical researchbreakthroughs.
FAQ
What is the scope of the Remedium–Lilly deal?
A multi-target R&D collaboration in obesity and type 2 diabetes using Prometheus™, with upfront/equity, milestones, and royalties (undisclosed amounts). [1]
Which indication did MCO-010 receive RMAT for?
Stargardt disease. Nanoscope is separately pursuing a rolling BLA for RP based on Phase 2b RESTORE. [2]
How durable are laru-zova’s Phase 2 signals?
Beacon reported sustained LLVA and microperimetry improvements to 9–36 months with overall tolerability; pivotal VISTA uses LLVA as the primary endpoint.
What did uniQure show in Fabry with AMT-191?
In Cohort A (n=4), α-Gal A rose 27–208× above mean normal; all discontinued ERT with stable lyso-Gb3 through cutoff; safety was manageable with noted SAEs.
What’s new from Ultragenyx’s DTX401 Phase 3?
At Week 96, mean daily cornstarch fell ~61% with maintained glycemic control; nighttime dosing reduced 70–75% in groups analyzed.
What changed for Yescarta in Korea?
MFDS approved second-line use in DLBCL relapsing within 12 months; reimbursement process has begun, potentially improving access.
Entities / Keywords
Remedium Bio; Eli Lilly; Prometheus™ • Nanoscope Therapeutics; MCO-010 (sonpiretigene isteparvovec); RMAT; Stargardt; RP • Beacon Therapeutics; laru-zova; XLRP; LLVA; microperimetry • uniQure; AMT-191; Fabry; α-Gal A; lyso-Gb3 • Ultragenyx; DTX401; GSDIa; AAV8 • Vyriad; Yeda; Weizmann Institute; adapter peptide; in vivo targeting • Lyell Immunopharma; ronde-cel (LYL314); CD19/CD20; PiNACLE-H2H; EFS • ImmunityBio; ANKTIVA (nogapendekin alfa inbakicept-pmln); ALC; OS; NSCLC • Capricor Therapeutics; Deramiocel; DMD cardiomyopathy; CRL; HOPE-3 • Kite/Gilead; Yescarta (axi-cel); MFDS; 2L DLBCL • enGene; detalimogene voraplasmid; LEGEND; NMIBC CIS.
References
https://www.cgtlive.com/view/nanoscope-rmat-designation-retinitis-pigmentosa-gene-therapy-mco-010
https://www.koreabiomed.com/news/articleView.html?idxno=28893