Watch Our Video Summary Capturing Top Cell and Gene News from the Last Two Weeks
From Biogen’s higher-dose Spinraza setback and AAV exit to AMT-130’s promising Huntington’s data—signals are robust. They span regulatory shifts, neurology, rare disease, hematology, and more, with pivotal trial readouts and policy advances underscoring both strategic portfolio realignment and accelerating pathways for gene and cell therapies.
Top Stories Covered in This Video
🧴 FDA rejects higher-dose Spinraza (nusinersen) filing [1] [US • 24 Sep 2025]
Key point: FDA declined Biogen’s application, requesting manufacturing section revisions; resubmission planned.
Context: Higher dose approved in Japan; under EU review (per report).
Implication: Regulatory/generics: Introduces competition that may affect pricing and formulary access.
🧬 Biogen ends AAV programs; staff reshuffle [2] [US • 29 Sep 2025]
https://www.biospace.com/business/biogen-shuffles-20-staff-after-ending-aav-work
Key point: Company discontinued AAV gene therapy efforts; around 20 roles affected, many reassigned.
Context: Part of portfolio refocus and cost-cutting targeting ~$1B savings by 2025.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.
🧠 AMT-130 slows Huntington’s in pivotal Ph1/2 (reported) [3] [24 Sep 2025]
Key point: Report cites 75% slowing at 36 months for high dose vs external control; full dataset not peer-reviewed.
Context: AAV-delivered miRNA to lower mutant huntingtin; company announcement basis.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.
🩸 Valoctocogene roxaparvovec durability analysis (Hem A) [4] [24 Sep 2025]
https://bioengineer.org/long-term-durability-of-valoctocogene-roxaparvovec-in-hemophilia-a/
Key point: Modeling/extrapolation suggests sustained FVIII activity for years after one-time infusion.
Context: Advances in Therapy paper synthesizing clinical data; acknowledges need for longer-term evidence.
Implication: Observational/RWE: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
🇪🇺 Uplizna (inebilizumab) recommended for IgG4-RD [5] [EU • 23 Sep 2025]
https://www.medscape.com/viewarticle/europe-b-cell-therapy-moves-igg4-related-disease-2025a1000pdk
Key point: CHMP positive opinion for adult IgG4-RD; first EU therapy if approved.
Context: Phase 3 MITIGATE (n=135) showed fewer flares and higher steroid-free remission vs placebo (per report).
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.
🏛️ FDA drafts to streamline CGT development/approval [6] [US • 25 Sep 2025]
Key point: Encourages single-arm baseline-controlled designs, use of historical/RWD controls, adaptive methods, and robust early CMC.
Context: Three CBER draft guidances; also outlines post-approval RWE/EHR/claims use.
Implication: Observational/RWE: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
💪 Capricor’s Deramiocel BLA path clarified (DMD) [7] [US • 25 Sep 2025]
Key point: FDA aligned that HOPE-3 can fulfill CRL “additional study” within current BLA; PUL v2.0 primary, LVEF key secondary; topline mid-Q4 2025 planned.
Context: CRL in July 2025; majority of BLA previously reviewed; CMC items addressed per company.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.
👁️ Opus Genetics posts pediatric LCA5 data; FDA talks next [8] [US • 30 Sep 2025]
Key point: First three adolescents averaged ~0.3 logMAR improvement; no drug-related AEs; FDA meeting planned in Q4.
Context: AAV8 LCA5 augmentation; prior adult data showed early gains and durability through 18 months (per report).
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.
Why it matters
- Regulatory shifts (FDA CGT drafts; CHMP opinion) could accelerate approvals and expand options in rare/autoimmune disease.
- Sponsor updates (Capricor, Opus) sustain momentum across neuromuscular and ocular gene therapies.
- Biogen’s moves show strategic reshaping across modalities amid competitive and CMC pressures.
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FAQ
What did FDA request in the higher-dose Spinraza filing? [1]
Manufacturing section revisions. Biogen plans to resubmit; higher dose is approved in Japan and under EU review (per report).
How significant is CHMP’s Uplizna opinion for IgG4-RD? [5]
It would establish the first authorized EU therapy for IgG4-RD if the European Commission approves; based on MITIGATE Phase 3 results.
What changed for Capricor’s Deramiocel after the CRL? [7]
FDA agreed HOPE-3 can serve as the additional study within the current BLA, with PUL v2.0 as primary and LVEF as key secondary; topline planned mid-Q4 2025.
What pediatric outcomes did Opus report in LCA5? [8]
An average ~0.3 logMAR improvement across three adolescents, with no drug-related AEs; FDA discussions planned for Q4.
What do the FDA CGT drafts mean for trial design? [6]
They support single-arm baseline-controlled trials, model-based disease progression assessments, historical/RWD controls, adaptive designs, and emphasize early CMC rigor; RWE is encouraged post-approval.
Entities / Keywords
Biogen (Spinraza/nusinersen higher dose; AAV exit); AMT-130 (Huntington’s; external control; cUHDRS); Valoctocogene roxaparvovec (Hemophilia A; FVIII durability); Uplizna/inebilizumab (IgG4-RD; CHMP; MITIGATE); FDA CBER CGT drafts (RWD; adaptive; single-arm; CMC); Capricor (Deramiocel; DMD; HOPE-3; PUL v2.0; LVEF; CRL); Opus Genetics (LCA5; AAV8; logMAR).
References
- https://pharma.economictimes.indiatimes.com/news/pharma-industry/fda-rejects-biogens-higher-dose-sma-treatment-amid-rising-competition/124085007
- https://www.biospace.com/business/biogen-shuffles-20-staff-after-ending-aav-work
- https://www.newsonair.gov.in/experimental-gene-therapy-amt-130-slows-huntingtons-disease-progression-by-75-in-pivotal-phase-1-2-study/
- https://bioengineer.org/long-term-durability-of-valoctocogene-roxaparvovec-in-hemophilia-a/
- https://www.medscape.com/viewarticle/europe-b-cell-therapy-moves-igg4-related-disease-2025a1000pdk
- https://www.biospace.com/fda/fda-details-thinking-for-streamlining-development-of-cell-gene-regenerative-therapies
- https://www.globenewswire.com/news-release/2025/09/25/3156168/0/en/Capricor-Therapeutics-Provides-Regulatory-Update-on-Deramiocel-Program-for-Duchenne-Muscular-Dystrophy-Following-Type-A-Meeting.html
- https://www.fiercebiotech.com/biotech/opus-sets-sights-fda-talks-after-posting-pediatric-gene-therapy-data