Company: NCEL
Filing Date: 2025-06-23
Form Type: F-4/A
Source: 0001213900-25-056787
Chunk: 373

Company: NewcelX Ltd.
Filing Date: 2025-06-23
Form: F-4/A
Chunk 373
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 of first -in-man intrathecal injection of human astrocytes (AstroRx ® ) in ALS patients: phase I/IIa clinical trial results. This paper describes the results of Phase 1/2a NCT03482050 clinical trial (further described on page 203). PMID: 29871694: Safety and efficacy of human embryonic stem cell -derived astrocytes following intrathecal transplantation in SOD1 G93A and NSG animal models. This paper describes the development of good manufacturing practice -gradeprotocol for generation of astrocytes from human embryonic stem cells (AstroRx ®), their in vitro function, their efficacy in ALS animal model (hSOD1 G93Atransgenic mice) and their 201 safety and biodistribution in a 9 -monthstudy conducted in immunodeficient NSG mice under good laboratory practice conditions. In vitro, AstroRx ®possess the activities of functional healthy astrocytes, including glutamate uptake, promotion of axon outgrowth and protection of MNs from oxidative stress. A secretome analysis shows that AstroRx ®also secretes several inhibitors of metalloproteases as well as a variety of neuroprotective factors (e.g. TIMP -1, TIMP -2, OPN, MIF and Midkine). Intrathecal injections of the AstroRx ®into transgenic hSOD1 G93Amice and rats significantly delayed disease onset and improved motor performance compared to sham -injectedanimals. A safety study in immunodeficient mice showed that intrathecal transplantation of AstroRx ®demonstrated positive results. Transplanted AstroRx ®attached to the meninges along the neuroaxis and survived for the entire duration of the study without formation of tumors or teratomas. Cell -injectedmice gained similar body weight to the sham -injectedgroup and did not exhibit clinical signs that could be related to the treatment. No differences from the vehicle control were observed in hematological parameters or blood chemistry. PMID: 32848579: Rising Stars: Astrocytes as a Therapeutic Target for ALS Disease This paper describes the role of Astrocytes malfunction in several neurodegenerative diseases and specifically in LAS. Astrocytes impaired biological functionality were implicated in multiple neurodegenerative diseases, including ALS, FTD, Parkinson’s and Alzheimer’s. In ALS disease patients, A1 reactive astrocytes were found to play a key role in the pathology of ALS