Today’s neuroscience weekly update spotlights major advances spanning cell and gene therapy manufacturing, AI-enabled drug discovery, brain delivery technologies, and global regulatory approvals. Momentum continues around CNS access, platform scalability, and high-value partnerships.
In Today’s Newsletter
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🧠 Aspen completes Cohort 3 and expands GMP for PD cell therapy [1] [US • 08 Jan 2026]
Context: ASPIRO Phase 1/2a of ANPD001 in moderate to advanced Parkinson’s; autologous DANPC therapy; earlier cohorts showed safety and tolerability at 6 months without immunosuppression.
Key point: Cohort 3 used commercial formulation with cryopreserved thaw-and-inject workflow; company finished San Diego GMP build-out entering qualification.
Implication: May influence prescriber choice and payer reviews pending full data.
🔭 Headlamp launches Lumos AI for neuroscience R&D decisions [2] [US • 07 Jan 2026]
Context: Neurosymbolic multi-agent platform integrating biological, behavioral, clinical data.
Key point: Aims to identify responder subtypes earlier and refine trial design to mitigate heterogeneity and placebo effects.
Implication: Could streamline initiation and adherence via remote prescribing and logistics.
🧩 Precision Neuroscience partners with Medtronic on BCI + navigation integration [3] [US • 12 Jan 2026]
Context: Integrates Layer 7 cortical interface with Medtronic StealthStation surgical navigation.
Key point: Seeks real-time functional and structural mapping for neurosurgery using high-resolution electrodes + 3D navigation.
Implication: Signals pipeline investment and modality expansion.
🚀 Novartis licenses SciNeuro brain-shuttle Alzheimer’s program [4] [12 Jan 2026]
https://www.fiercebiotech.com/clinical-data/novartis-pays-scineuro-165m-ticket-another-shuttle-brain
Context: BBB shuttling strategies remain a key industry focus.
Key point: Deal includes $165M upfront, up to $1.5B in milestones, worldwide rights to antibody program for brain delivery.
Implication: Signals pipeline investment and modality expansion.
🧬 Dyno unveils TfR1-mediated AAV capsid Dyno-yp2 for CNS [5] [US • 12 Jan 2026]
Context: AI-engineered AAV; evaluated in TfR-humanized mice vs BI-hTFR1 and AAV9.
Key point: Reported >94% neuronal transduction, 11-fold brain biodistribution gain vs BI-hTFR1, and strong liver detargeting.
Implication: Signals pipeline investment and modality expansion.
🇰🇷 Korea approves Ramzede (velmanase alfa) for alpha-mannosidosis [6] [Korea • 12 Jan 2026]
https://biz.chosun.com/en/en-science/2026/01/12/KJY46LNFAVEOVAO6PNFVL3R2FY/
Context: Enzyme replacement for non-CNS symptoms in alpha-mannosidosis; introduced by Kwangdong in Korea.
Key point: First approved therapy in Korea for this rare disease; MFDS set dose at 10 mg.
Implication: Introduces competition that may affect pricing and formulary access.
🧯 Evogene + Unravel to design remyelination small molecules [7] [IL/US • 07 Jan 2026]
Context: Combines Evogene ChemPass AI with Unravel’s BioNAV and patient-derived data.
Key point: Targeting a novel demyelination biology to enable brain-penetrant inhibitors aimed at restoring myelin.
Implication: Signals pipeline investment and modality expansion.
💊 Kallyope sets 2026 plan for migraine and metabolism assets [8] [US • 07 Jan 2026]
Context: Elismetrep, an oral TRPM8 antagonist, and K-554, a non-incretin peptide for metabolic disease.
Key point: Positive Phase 2b topline for elismetrep; planning to initiate registrational trials mid-2026. K-554 to enter Phase 1 with early data in H2 2026.
Implication: May influence prescriber choice and payer reviews pending full data.
🧪 Sarepta files CTA in NZ for SRP-1005 siRNA in Huntington’s [9] [US/NZ • 07 Jan 2026]
Context: SRP-1005 (formerly ARO-HTT) uses TfR1-mediated delivery; first-in-human Phase 1 dose escalation planned in ~24 participants.
Key point: CTA submitted to Medsafe; subcutaneous dosing intended for deep brain target engagement.
Implication: Signals pipeline investment and modality expansion.
Why it matters
- BBB delivery continues to be a central bottleneck and investment theme (Novartis–SciNeuro, Dyno-yp2, Sarepta SRP-1005).
- Surgical neurotech platforms are converging on integrated mapping and navigation (Precision–Medtronic).
- AI is moving upstream in CNS development for patient selection and trial design (Headlamp, Evogene–Unravel).
- Rare-disease access expands in Asia with enzyme replacement approvals (Ramzede in Korea).
- Novel mechanisms beyond incumbents advance toward pivotal stages (TRPM8 in migraine; non-incretin metabolic targets).
FAQ
What is ANPD001 and where is Aspen’s program now?
ANPD001 is an autologous dopaminergic neuronal precursor cell therapy for Parkinson’s. Aspen completed Cohort 3 dosing in Phase 1/2a and finished GMP expansion entering qualification for late-stage manufacturing [1]. Full efficacy data are not provided.
How does Novartis’s SciNeuro deal compare in scale and focus?
Novartis paid $165M upfront, with total potential value up to $1.7B, to license a BBB-shuttle Alzheimer’s antibody program, adding to industry efforts to improve brain delivery [4]. Specific endpoints were not disclosed.
What distinguishes Dyno-yp2 from other CNS AAV capsids?
In TfR-humanized mice, Dyno-yp2 showed >94% neuronal transduction, 11-fold brain biodistribution improvement vs BI-hTFR1, and marked liver detargeting, addressing efficacy and safety concerns for systemic CNS gene therapy [5]. Translation to humans is not established.
What is Kallyope’s elismetrep targeting, and what’s next?
Elismetrep is an oral TRPM8 antagonist for acute migraine. Kallyope reported positive Phase 2b results and plans to start registrational trials mid-2026; detailed endpoints are not stated in the release [8].
What is Sarepta’s SRP-1005 approach in Huntington’s?
SRP-1005 is a subcutaneous siRNA leveraging TfR1-mediated delivery to knock down huntingtin protein with intended deep-brain penetration. A CTA was filed in New Zealand for a first-in-human Phase 1 study [9]. Specific efficacy endpoints are not specified.
What did Korea approve for alpha-mannosidosis?
MFDS approved Ramzede (velmanase alfa) for non-CNS symptoms in alpha-mannosidosis, the first such therapy in Korea. The approved dose is 10 mg, with Kwangdong as local introducer [6].
Entities / Keywords
Aspen Neuroscience; ANPD001; Parkinson’s; GMP; Headlamp Health; Lumos AI; neurosymbolic AI; Precision Neuroscience; Medtronic; Layer 7; StealthStation; Novartis; SciNeuro; blood-brain barrier; brain shuttle; Alzheimer’s; Dyno Therapeutics; Dyno-yp2; TfR1; AAV; Ramzede; velmanase alfa; alpha-mannosidosis; MFDS; Evogene; ChemPass AI; Unravel Biosciences; BioNAV; remyelination; multiple sclerosis; Kallyope; elismetrep; TRPM8; K-554; non-incretin peptide; Sarepta; SRP-1005; siRNA; Huntington’s; Medsafe.
References
- https://www.prnewswire.com/news-releases/aspen-neuroscience-completes-phase-12a-aspiro-trial-cohort-three-dosing-and-state-of-the-art-manufacturing-expansion-paving-the-way-for-developing-transformative-therapies-for-patients-302655800.html
- https://www.prnewswire.com/news-releases/headlamp-health-launches-lumos-ai-to-de-risk-neuroscience-drug-development-302655200.html
- https://www.manilatimes.net/2026/01/13/tmt-newswire/globenewswire/precision-neuroscience-and-medtronic-partner-to-integrate-layer-7-brain-computer-interface-with-medtronic-neurosurgical-platform/2257513
- https://www.fiercebiotech.com/clinical-data/novartis-pays-scineuro-165m-ticket-another-shuttle-brain
- https://www.01net.it/dyno-therapeutics-launches-dyno-yp2-a-top-performing-tfr1-mediated-aav-capsid-to-further-diversify-cns-delivery-portfolio/
- https://biz.chosun.com/en/en-science/2026/01/12/KJY46LNFAVEOVAO6PNFVL3R2FY/
- https://www.prnewswire.com/news-releases/evogene-and-unravel-biosciences-announce-collaboration-to-develop-a-first-in-class-therapy-to-reverse-neurological-damage-in-demyelinating-disorders-302655069.html
- https://www.businesswire.com/news/home/20260107056893/en/Kallyope-Announces-2026-Strategic-Priorities-for-Migraine-and-Metabolism-Portfolio-Based-on-Novel-Neural-Circuits-Platform
- https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-submission-clinical-trial