Starter en tråd om Sangamo Therapeutics, Inc. (SGMO) siden en del har den i favoritt-listen eller i porteføljen sin
Sangamo Therapeutics, Inc. focuses on translating ground-breaking science into genomic therapies that transform patients’ lives using platform technologies in genome editing, gene therapy, gene regulation, and cell therapy.
The company’s zinc finger DNA-binding protein (ZFP) technology enables specific genome editing and gene regulation. The ZFPs could be engineered to make ZFP nucleases (ZFNs), proteins that could be used to specifically modify DNA sequences by adding or knocking out specific genes; and ZFP transcription factors (ZFP TFs), proteins that can be used to turn genes on or off.
Its therapeutic products include SB-728-T, a ZFN-mediated autologous T-cell product for human immunodeficiency virus and acquired immunodeficiency syndrome (HIV/AIDS), which is in Phase II and Phase I clinical trials; and SB-728-HSPC that is in Phase I/II clinical trials for HIV/AIDS. The company also engages in Phase I/II studies of in vivo genome editing applications of ZFP Therapeutics for hemophilia B, Hemophilia A, and Mucopolysaccharidosis I (MPS) and MPS II, which are lysosomal storage disorder (LSD); preclinical programs in other LSDs; and research stage programs in central nervous system disorders and cancer immunotherapies.
It has collaborative partnerships with Biogen Inc. to develop therapeutic genome editing products in hemoglobinopathies; and Shire International GmbH to develop the preclinical development program in Huntington’s disease, as well as license agreements with Sigma-Aldrich Corporation to develop ZFP-based laboratory research reagents and Dow AgroSciences, LLC to modify the genomes or alter protein expression of plant cells and plants.
The company also has a research collaboration and license agreement with Pfizer Inc. The company was formerly known as Sangamo BioSciences, Inc.
and changed its name to Sangamo Therapeutics, Inc. in January 2017.
Sangamo Therapeutics, Inc. was founded in 1995 and is headquartered in Richmond, California.
La oss starte diskusjonen! @Yogz @koe @pdx
Er nok grunnen til at sgmo kjørte emisjon for en stund tilbake. Og det kan tolkes bullish at ikke sgmo aksjer inngår i oppgjøret.
Abstract til konferansen 5. September
BACKGROUND: Mucopolysaccharidosis type II (MPS II, Hunter syndrome), is a rare, X-linked metabolic disease caused by mutations in the IDS gene, which leads to a deficiency of the lysosomal enzyme iduronate-2-sulfatase (IDS). MPS II is characterized by progressive respiratory and cardiac disease, skeletal abnormalities, and premature death with neurodegeneration in the severe form. SB-913 is a new type of gene therapy being developed for the treatment of MPS II. SB-913 uses zinc finger nuclease (ZFN)-mediated in vivo genome editing to insert a normal copy of the IDS transgene into liver cells, delivered via AAV2/6 vectors. The precision and specificity of the ZFNs allow for integration at a specified genomic location and result in high, continuous production of IDS, as demonstrated in an MPS II mouse model. SB-913 aims to provide lifelong production of IDS, with the goal of therapeutic benefit and eliminating the need for weekly enzyme replacement therapy.
METHODS: A Phase 1/2 clinical trial (CHAMPIONS) is ongoing in the U.S. to determine if SB-913 is safe and tolerable in patients with MPS II. The study is a multicenter, open-label, dose ranging trial with one-time peripheral intravenous infusion of SB-913, followed by three years of observation and testing. IDS activity and clinical endpoints will also be assessed. Key eligibility criteria include age ≥18, diagnosis of attenuated MPS II, and lack of pre-existing antibodies to AAV6.
RESULTS: Four subjects have received a single infusion of SB-913, with two subjects in each of two dose cohorts (5e12 vg/kg and 1e13 vg/kg). The infusions were generally well-tolerated and no serious adverse events related to the study drug were reported, with maximum exposure of up to 5 months post-study drug administration. Additional analysis of the trial data will be presented as available.
DISCUSSION: Initial safety data from this clinical trial support further development of SB-913 for the treatment of MPS II.
Mildt sagt ikke det peneste chartet:
Noen flere i sgmo? Er visst trigger på vei 5. September
Fikk vel akkurat bekreftet at det skjer noe binært 5. september. Conference call er enten damage control eller hypefest.
Noen ganger så er resultater så kompliserte og vanskelig å tolke at man trenger en slik conference call etterpå bare for å kunne spørre “hva i helvette betyr alt dette?”.
Vil dere lese haussing? For da skal dere få haussing!
Den var knallbra
Ble nødt til å kjøpe litt på impuls
Greit å ha noe etter stengetid (ose og Sverige) å følge med på.
Har tidligere gjort en brukbar kule på Enanta «over there». Solgte litt for tidlig dog.
Vet liksom aldri helt hva jeg skal tro når jeg leser sånt. Sliter med et alvorlig kognitivt Fox mulder bias: i want to believe
Den går vertfall mye om dagen, så mulig å hente på en trade on news uansett spennende saker, skulle bare ønske jeg ikke måtte spare penger til emisjonen i PCI, kan vel ikke være inne i alt
Same same. Må selge om kort tid her pga Pcib.
God timing da. Hvis det kommer gode nyheter her 5 september
Hehe, ja så lenge det går rette veien får se ann i morgen om det blir en liten trade
Fiffig detalj at markedet i usa er stengt på mandag.
Hver måned kårer jeg det jeg kaller Teksperter™ for noen av de mest populære investeringene våre
Det er de 3 medlemmene som har fått flest likes på innleggene sine de siste 90 dagene. Teksperter™ får også en unikt merke på profilen sin og et trofé-ikon ved siden av navnet sitt. Du kan bli Tekspert™ i flere aksjer/investeringer, og troféet vil bare vises i tråder der du er Tekspert™.
Her er denne månedens Teksperter™ og det mest likte innlegget deres fra de siste 90 dagene:
@Savepig (7 likes)
@Yogz (4 likes)
@koe (1 likes)
Resten av topp 10:
@Mr.Oncoinvest (1 likes)
@larsmkn (1 likes)