Drug intelligence / Profile preview

ALT001

Development stage
Phase 1
Lead developer
DarwinCell
Modality
Small Molecules, Stem Cell Therapies → Cell Therapies
Administration
Intra-articular Injection, Intravenous
01

Overview

ALT001 is a designation used for two unrelated drug candidates. The first is a mesenchymal stem cell (MSC)-based biologic developed by DarwinCell, which utilizes proprietary protein polymer extraction technology (ECIWEP) to treat neurological conditions including amyotrophic lateral sclerosis (ALS), acute ischemic stroke, and Alzheimer's disease. This candidate has demonstrated safety and preliminary efficacy in investigator-initiated trials involving over 40 ALS patients and is progressing toward IND filings in the United States and China. The second candidate is a small molecule mitophagy enhancer developed by Altmedical. It is a synthetic isoquinoline derivative that activates the ULK1/Rab9-mediated alternative mitophagy pathway to address Alzheimer's disease and serve as an antiviral agent against herpes simplex virus type 1 (HSV1) by reducing neuroinflammation and restricting viral replication.

Other names
nerve repair protein
02

Targets

RAB9A (Ras-related protein Rab-9A)

Beyond the preview

Go deeper on ALT001.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Full profile access

Follow clinical development from study design and recruitment through results.

  • Trial phase
  • Status
  • Readouts

Indications & development

Full profile access

Explore development by indication, patient population, and geography.

  • Indications
  • Development status
  • Countries

Licensing & deals

Full profile access

Trace asset ownership, licensing agreements, and commercial partnerships.

  • Partners
  • Deal terms
  • Milestones

Patents & exclusivity

Full profile access

Explore the patent landscape and regulatory exclusivity around an asset.

  • Patents
  • Expiration dates
  • Exclusivity

Competitive landscape

Full profile access

Compare development programs by target, modality, and indication.

  • Competing assets
  • Targets
  • Development stage

Research & analysis

Full profile access

Connect source evidence and development news to your research questions.

  • Publications
  • News
  • Analysis

Bring the full picture into focus.

See how Gosset can support your research on ALT001.

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call