Syncona Share Discussion Threads

[sev22] 09/12/2021 15:07

Published today:

Achilles Therapeutics Presents Positive Data at ESMO I-O Congress 2021 on High-Dose Manufacturing Process for Precision T Cell Therapies Targeting Clonal Neoantigens.

Over 10-fold dose increase at GMP scale with VELOS™ Process 2 manufacturing.

cNeT maintain CD4+ and CD8+ T cell subsets with a highly potent polyclonal phenotype.

Dosing of patients with high-dose cNeT expected in 1H 2022.

LONDON, Dec. 09, 2021 (GLOBE NEWSWIRE) -- Achilles Therapeutics plc (NASDAQ: ACHL), a clinical-stage biopharmaceutical company developing precision T cell therapies to treat solid tumors, today presented positive data at the ESMO Immuno-Oncology Congress 2021 (ESMO I-O) that further demonstrate that Achilles’ VELOS™ Process 2 manufacturing increases clonal neoantigen-reactive T cell (cNeT) doses by more than 10-fold over Process 1 at GMP scale and maintains a highly potent polyclonal phenotype. These data add to the pilot scale proof-of-concept study recently reported at the 2021 Society for Immunotherapy of Cancer (SITC) Annual Meeting.

“We are extremely pleased to have demonstrated the ability to generate a significant boost in cNeT dose over Process 1 in clinical-scale runs and to have successfully completed the technology transfer of Process 2 into clinical manufacture,” said Dr Ed Samuel, SVP Technical Operations of Achilles. “These Process 2 GMP data validate our previously reported R&D data and confirm retention of critical phenotypic characteristics and the ability to identify the active drug component of our products without adding to end-to-end manufacturing time. We anticipate dosing patients with high-dose Process 2 cNeT in the first half of 2022, with 6-week clinical and translational science data available in the second half 2022.”

Key highlights from the presentation entitled “Achilles VELOS Process 2 generates a >10-fold improvement in cNeT dose over Process 1 with a highly potent polyclonal phenotype and has been successfully validated at GMP scale for clinical use in solid cancer,” include:

Reporting that GMP validation of VELOS Process 2 has successfully been completed and transferred into clinical manufacture in Achilles’ ongoing Phase I/IIa CHIRON and THETIS clinical trials.

Showing that VELOS Process 2 generates a cell product that retains both CD4+ and CD8+ T cell subsets and maintains polyclonal cNeT reactivity with a favorable cell fitness phenotype.

Demonstrating the quantification of the active cNeT drug component with Achilles’ proprietary potency assay which further underlines the strength of the Achilles platform.

About Achilles Therapeutics
Achilles is a clinical-stage biopharmaceutical company developing precision T cell therapies targeting clonal neoantigens: protein markers unique to the individual that are expressed on the surface of every cancer cell. The Company has two ongoing Phase I/IIa trials, the CHIRON trial in patients with unresectable locally advanced and metastatic non-small cell lung cancer (NSCLC) and the THETIS trial in patients with recurrent or metastatic melanoma. Achilles uses DNA sequencing data from each patient, together with its proprietary PELEUS™ bioinformatics platform, to identify clonal neoantigens specific to that patient, and then develop precision T cell-based product candidates specifically targeting those clonal neoantigens.

[chc15] 09/12/2021 09:14

What happened here, rose for no reason, then plummeted.

[sev22] 14/11/2021 16:12

The Syncona Investment Trust, a member of Interactive Investor’s ACE 40 rated list of ethical investments, has reported its latest half-yearly results (11th November 2021):

Syncona Ord (SYNC) investment trust, which invests in life science companies, reported a decline in net asset value of 11.4% for the first six months of its financial year – from the end of March to the end of September.

Over this period, the trust reported that its net assets sat at £1.15 billion, down from £1.3 billion at the end of March 2021.

Figures from FE Analytics show that over this period its share price total return was a loss of 33.6%, suffering from its high premium notably declining.

The decline, the report notes, was driven predominantly by the decline in the share price of two of the trust’s listed holdings, Freeline Therapeutics Holdings ADR FRLN
and Achilles Therapeutics ADR ACHL.

Freeline’s poor performance is put down to “operational challenges” owing to the Covid-19 pandemic. Syncona says these concerns have now been addressed.

Achilles’ share price decline was driven by “market sentiment towards cell and gene therapies”. Syncona says that it believes the business is performing well and in line with expectations.

The trust’s strategy is to establish, build and fund companies to turn exceptional science into a dynamic portfolio of global leaders in life sciences. The aim is for the companies to deliver their product to market. The trust has a long-term target of owning between 15 and 20 companies. It currently has 10.

Martin Murphy, chief executive of Syncona Investment Management Limited, said: “While we are disappointed by the decline in NAV during the period, we are continuing to build a diverse portfolio across the development cycle and therapeutic areas and remain confident in our companies' potential. The substantial capital that a number of our companies have accessed so far this year validate the significant opportunity ahead for them.”

The trust also said that it planned to deploy another £100 million to £175 million into both existing companies and new opportunities this year.

Murphy said: “With clinical data the key driver of value and risk for Syncona, we believe our companies are well positioned and on track to further validate our model and strategy in the next 12 months with the potential for a rich seam of data.”

The trust is a member of Interactive Investor’s ACE 40 rated list of ethical investments. It was placed under formal review in the summer due to the volatility of its share price, but it kept its place on the list.

Dzmitry Lipski, head of funds research at Interactive Investor, notes: “We remain positive on the trust outlook and see it as a strong choice for adventurous investors prepared to tolerate high volatility in the short term, but potentially reap rewards over the longer term.

[sev22] 08/11/2021 19:36

This was before today's two positive announcements.

[the shuffle man] 08/11/2021 13:59

Two well received announcements today and increased news flow over the last few weeks has certainly moved this off the bottom. Great track record here worth backing IMO.

[sev22] 08/11/2021 10:03

Peel Hunt Limited have a new price target of £2.79.

[chc15] 29/10/2021 15:44

Pretty decent recovery here, must be strong institutional support.

[sev22] 22/10/2021 15:01

Achilles Therapeutics Presents Data at the 2021 European Society for Gene and Cell Therapy (ESGCT) Congress Demonstrating its Proprietary Manufacturing Process Can Generate Potent, Personalized Anti-Cancer Cell Therapy Candidates in Multiple Tumor Types (22nd October 2021).

Achilles Therapeutics plc (NASDAQ: ACHL), a clinical-stage biopharmaceutical company developing precision T cell therapies to treat solid tumors, today delivered an oral presentation (OR54) at the 2021 European Society for Gene and Cell Therapy (ESGCT) Congress. In the presentation entitled ‘Multicentre, prospective research protocol for development of a clonal neoantigen-reactive T cell (cNeT) therapy pipeline across multiple tumour types,’ Dr. Michael Grant, Associate Medical Director at Achilles, reviewed initial data from the Company’s Material Acquisition Platform (MAP)​ showing that Achilles’ proprietary VELOS™ manufacturing process is able to extract tumor infiltrating lymphocytes (TIL) and generate potent clonal neoantigen-reactive T cells (cNeT) across a range of solid tumor types. cNeT target clonal neoantigens, which are unique proteins expressed on every cancer cell within a patient but not on healthy tissue.

MAP is a unique prospective study that facilitates the procurement of patient material across a range of solid tumor types, enabling a comprehensive evaluation of indications prior to clinical development. MAP was developed to enable the detailed genomic and cellular characterization of different cancer tissue types and assess the ability to produce significant doses of potent cNeT. The study is currently collecting patient material at eight sites in the United Kingdom, European Union, and United States from patients with a range of cancers including lung, melanoma, head and neck, renal, bladder and breast cancer.

“This important research shows that our bioinformatics and manufacturing processes together produce fit, potent and neoantigen-specific cells and supports the potential use of our cNeT in a broad range of solid tumor indications beyond our current clinical trials in non-small cell lung cancer (NSCLC) and metastatic melanoma, and into indications including head and neck cancer,” said Dr Sergio Quezada, Chief Scientific Officer of Achilles. “MAP showcases the analytical strength of the Achilles platform, delivers vital translational science insights, and highlights our organizational and supply chain expertise. It has been extremely successful in accumulating a broad set of tumour-related material and continues to expand into new countries and indications.”

Data presented show that the VELOS manufacturing process delivers higher neoantigen-specificity and potency, in both CD4+ and CD8+ T cell activity, relative to traditionally manufactured TIL products from the same tumor source material. These enhancements can be seen in specificity and potency assays in which IFN-γ and TNF-α cytokine secretion and production of inflammatory cytokines is triggered in response to clonal neoantigen presentation. Additionally, analysis of CD4+ and CD8+ cells in cNeT products shows favorable cell phenotypes consistent with high cell fitness and reduced cell exhaustion. At the time of submission, 74 patient samples had been processed across five solid tumor indications. The proprietary PELEUSTM platform identified a median of 107 clonal neoantigens in NSCLC samples, 156 in melanoma samples, and 71 in head and neck squamous cell carcinoma samples, consistent with publicly available data sets, illustrating the accuracy of the platform. Additional data for renal and bladder tumor samples were still in process at the time of submission.

The presentation delivered by Dr. Grant is available in the Events & Presentations section of the Achilles Therapeutics website.

About Achilles Therapeutics
Achilles is a clinical-stage biopharmaceutical company developing precision T cell therapies targeting clonal neoantigens: protein markers unique to the individual that are expressed on the surface of every cancer cell. The Company has two ongoing Phase I/IIa trials, the CHIRON trial in patients with unresectable locally advanced and metastatic non-small cell lung cancer (NSCLC) and the THETIS trial in patients with recurrent or metastatic melanoma. Achilles uses DNA sequencing data from each patient, together with its proprietary PELEUS™ bioinformatics platform, to identify clonal neoantigens specific to that patient, and then develop precision T cell-based product candidates specifically targeting those clonal neoantigens.

[acuere] 22/10/2021 10:52

Return of interest here for last couple of weeks. I was looking for 160p, so not tempted at this level to buy back though. The last reported results gave NAV of 178p as at 30 Jun. The share price was about 200p so trading at a 13% premium.
At that time Freeline was trading at $8.15 and Autolus was $6.64. They are currently $3.30 and $5.79 respectively. Both are 12-14% down in the last month. So we are likely to see a NAV to end Sept of around 160p. So, by my reckoning it’s currently trading at a 17% premium to NAV.
Not justified imho. I’ll wait to see results and look for Autolus and Freeline to reverse their downward trends before buying again.

[sev22] 19/10/2021 11:39

SwanBio Therapeutics Announces New Preclinical Data Demonstrating Proof of Principle for SBT101 Gene therapy as a Potential Treatment of Adrenomyeloneuropathy (19th October 21).

- SBT101 Demonstrated Dose-Dependent Improvement of AMN Disease Markers in Mouse Models

- SBT101 Well-Tolerated in Non-Human Primates at 3-Months Post-Treatment

- Data Support Continued Advancement of SBT101 in IND-Enabling Studies

SwanBio Therapeutics, a gene therapy company focused on developing treatments for patients with genetically defined neurological conditions, today announced new preclinical efficacy and safety data supporting the potential of the company’s lead program, SBT101, as a disease-modifying treatment for adrenomyeloneuropathy (AMN). The data will be presented in two posters, “Intrathecal delivery of an AAV encoding human ABCD1 shows dose-responsive expression and activity in a mouse model of Adrenomyeloneuropathy,” and, “Three-month preclinical safety data of AAV9-hABCD1 following intrathecal delivery in non-human primates,” during the European Society for Gene and Cell Therapy (ESGCT) Virtual Congress. The Congress is being held October 19-22, 2021.

AMN, the adult-onset degenerative spinal cord disease occurring in patients with adrenoleukodystrophy (ALD), is characterized by progressive loss of mobility, incontinence and debilitating pain. ALD is caused by mutations in the ABCD1 gene, which encodes a protein essential to the processing and breakdown of very long chain fatty acids (VLCFA). Dysfunction of this protein leads to VLCFA accumulation in tissue and the progressive axonopathy of AMN. SBT101, a novel AAV-based gene therapy, is designed to compensate for the disease-causing ABCD1 mutation, increasing ABCD1 expression and reducing VLCFA levels.

“We are excited to share the first data on SBT101, which demonstrate its potential to directly address the underlying cause of AMN by establishing ABCD1 expression in the spinal cord,” said Karen Kozarsky, Ph.D., Swan’s chief scientific officer. “AMN is a devastating disease with no approved treatments, and we are pleased to have observed improvements in markers of AMN disease as well as good tolerability in both mice and NHPs. Taken together, these findings support the continued evaluation of SBT101 as a potential disease-modifying treatment for AMN as we advance towards the clinic.”

Summary of Findings

The safety and efficacy of SBT101 were evaluated in mouse models of AMN and non-human primates (NHPs). Key findings include:

Dose-dependent expression of hABCD1 and reduction of VLCFA levels after transduction of SBT101 in glial cell cultures from ABCD1 knockout mice.

Durable increase in hABCD1 protein expression and significant reductions in VLCFA levels at 13 weeks post-treatment with intrathecal delivery of SBT101, which were maintained at 24 weeks post-treatment, in an ABCD1 knockout mouse model.

Well-tolerated in NHPs at 3 months with expected transient dose-dependent increases in ALT and AST observed that resolved within 2 months post-treatment. Further, no advanced grade histopathological findings were observed in tissues, including the dorsal root ganglion.

Based on these findings, SwanBio plans to continue its ongoing investigational new drug (IND) enabling studies for SBT101 to support a planned IND submission.

About Adrenomyeloneuropathy

Adrenomyeloneuropathy (AMN), the adult-onset spinal cord disease of adrenoleukodystrophy (ALD), is a progressive neurodegenerative disorder. It is caused by mutations in a single X-linked gene, ABCD1. The ABCD1 gene encodes a protein that resides in the cells' peroxisomal membrane. Dysfunction of this protein results in accumulation of very long chain fatty acids (VLCFA) in the blood, tissue and cerebrospinal fluid and progressive neurodegeneration in the spine. Symptoms of AMN typically begin in men between the ages of 20 and 30. Women can experience symptoms later in life. Early symptoms for people living with AMN include leg weakness, such as difficulty walking and balance issues. Later in the progression of the disorder, patients lose mobility and bladder and bowel dysfunction have a significant impact on quality of life. Beyond physical symptoms, people living with AMN report economic hardship and loss of dignity and self-esteem as the disease progresses over time.

About SwanBio Therapeutics

SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, genetically defined neurological conditions. SwanBio is advancing a pipeline of gene therapies, designed to be delivered intrathecally, that can address targets within both the central and peripheral nervous systems. This approach has the potential to be applied broadly across three disease classifications – spastic paraplegias, monogenic neuropathies and polygenic neuropathies. SwanBio’s lead program is being advanced toward clinical development for the treatment of adrenomyeloneuropathy (AMN). For more information, visit SwanBioTx.com.

[sev22] 30/9/2021 16:03

Gyroscope announces additional positive interim data from Phase I/II FOCUS trial.

[chc15] 30/9/2021 08:07

Yeh these biotechs are just a gamble. Only have a small bit in here because of that.

[grim] 29/9/2021 20:24

I'm hoping mm's are accumulating in anticipation of major news/results from investee trials - but it's only a hope. These were flavour of the month with many Fund Mgrs & pundits a couple of years ago and nothing majorly negative has been announced but wtfdik

[chc15] 29/9/2021 19:55

Wow this is plummeting fast, any ideas or buyers?

[salpara111] 17/8/2021 14:01

Glad I took a small loss a couple of days ago as opposed to a bigger loss today!
NAV is falling at quite a rate.

[davemac3] 02/8/2021 22:08

is this the reason for today's rise?

[tomps2] 16/7/2021 11:56

John Rosier mentions Syncona (SYNC) in the latest PIWORLD interview at 12m14s

Watch the video here:

Or listen to the podcast here:

[1seanshare] 24/6/2021 19:09

SYNC sinking further. Why so negative?

[luxaeterna1] 21/5/2021 11:01

Depends how you want to look at it. CAR-T sales are probably growing 40-80% per year at the moment, with a substantial number of trials with people like Autolus.

Heck, Autolus are even on the lower end of the risk/safety profile, so they have some 1st mover advantages there. Although I am not clear how Autolus will scale long term. CAR-T are not simple processes.

[chc15] 13/5/2021 15:27

T Henderson, check director rns.

[1seanshare] 13/5/2021 10:16

Yes that's correct. My concern is about CAR-Ts in general. They aren't the be all they were promised to be, there is considerable toxicity for patients who are already heavily pretreated, and they are very complex and onerous to deliver. It could be that CAR-Ts in general are not being used as much as first envisaged