Syncona Share Discussion Threads

[chc15] 17/1/2020 11:51

Any point holding onto these, not doing much, money put better eleswere?

[luxaeterna1] 21/12/2019 09:58

New AUTL presentation up on the website (Dec 19th) hxxps://www.autolus.com/investor-relations/news-and-events/presentations

[brexitplus] 19/12/2019 09:39

Healthcare company Syncona said it had made an $80m (£61.1m) funding commitment to biotech company Freeline.
The first tranche of $40m tranche would allow Freeline to expand its team, continue to develop its manufacturing platform generate further data in its clinical programmes for Haemophilia B and Fabry Disease, and progress its pipeline, Sycona said on Thursday.

Freeline has a pipeline of four disclosed programmes, two of which are clinical stage.

Syncona chief investment officer and Freeline executive chairman Chris Hollowood said: 'Freeline has developed a high-quality gene therapy platform with a novel capsid capable of driving high expression levels to treat systemic diseases.

'We are encouraged by the potential product profile in our lead programme in Haemophilia B and believe Freeline has the opportunity to achieve functional cures for patients across a broad pipeline of systemic diseases.'

Syncona said it would have a 79% fully diluted ownership stake in Freeline at the point full current commitments were invested.

[ltinvestor] 19/12/2019 07:54

Excellent progress being made by Freeline which is 79% owned by Syncona and valued at cost!

[milehouse] 10/12/2019 15:12

Interesting appointment of Julia Gregory to the board of Freeline. With experience in investment banking and taking biotech's to IPO it suggests that they will be preparing for an IPO.
Optimists view is that the Haemophilia B trial is looking good (read out due this financial year) and an IPO will be required to fund the Phase III.

Syncona shares seem to have dropped as a result though?

[brexitplus] 10/12/2019 10:19

Freeline, a biotechnology company focused on developing curative gene therapies for chronic systemic diseases, today announced the appointment of Julia P Gregory, to its Board of Directors. Ms. Gregory is a seasoned biopharmaceutical executive with CEO, CFO, Corporate Development, Board and Investment Banking experience.

“We are delighted to welcome Julia to Freeline’s Board of Directors,” said Chris Hollowood, Executive Chairman of Freeline. “She brings significant experience in biopharma governance, operations and investment banking. I look forward to working with her as we build the Company and progress our innovative gene therapy programmes through the clinic and towards commercialisation.”

“I am very pleased to be joining Freeline at this exciting time as the company brings curative gene therapies to patients suffering from the likes of Hemophilia, Fabry disease and Gaucher disease,” said Julia Gregory. “I look forward to working with the Board and management team to realise the full potential of these transformative therapies.”

Currently Chair and CEO of Isometry Advisors, Inc., a biotechnology advisory company, Ms. Gregory also serves as a non-executive director of several biopharmaceutical companies, including Biohaven Therapeutics, Cell Medica, Iconic Therapeutics, IMV, Nurix Therapeutics, and Sosei-Heptares. She is also a senior advisor to the Morgan Stanley’s Private Equity Growth Fund and M. M. Dillon & Co., Inc.

In her previous executive roles, Ms. Gregory served as Chief Executive Officer, Chief Financial Officer and Board Director of ContraFect Corporation; President and CEO of Five Prime Therapeutics; and Executive Vice President, Corporate Development and Chief Financial Officer of Lexicon Pharmaceuticals, Inc. Her corporate development experience includes leading transactions for significant strategic partnerships including development and licensing agreements with GlaxoSmithKline, Human Genome Sciences, Genentech, Bristol-Myers Squibb Company and Takeda Pharmaceutical Company Limited. As CFO at Lexicon, she led the company’s $220 million public offering and was involved in the creation of Lexicon’s $500 million private equity plan with Invus, LLP. Prior to her leadership positions in the biopharmaceutical industry, Ms. Gregory was an investment banker at Dillon, Read & Co. and subsequently at Punk, Ziegel & Company, where she served as the head of investment banking and head of its life sciences practice.

She received her B.A. in International Affairs from George Washington University ‘s Elliot School of International Affairs where she was elected to Phi Beta Kappa and her M.B.A. from The Wharton School of The University of Pennsylvania.

[ltinvestor] 08/12/2019 10:57

Data presented at the Ash conference yesterday shows the excellent progress being made by Autolus.Well worth reading .....

[ltinvestor] 07/12/2019 11:46

I expect to see a rerating of Syncona in 2020 as several of their investments enter the clinical stage.In addition, Autolus, which is around 30%, owned by Syncona should on positive data scale $20 plus over the coming months which should be reflected with an increase in the Syncona share price

[1seanshare] 07/12/2019 11:37

Don’t understand that whilst Autolus is much stronger, SYNC remains flat. My understanding was Autolus was dragging Sync but that shouldn’t be the case anymore?

[ltinvestor] 07/12/2019 11:19

Autolus and Freeline are both presenting updates at the ASH conference being held in Orlando this weekend.Looking forward in particular to more data being released by Autolus and progress to date...

[luxaeterna1] 03/12/2019 10:46

The NASDAQ market likes the recent updates from Autolus. Share price up 50% (valuation up $250m) in 1 month.

[brexitplus] 13/11/2019 16:11

LONDON, November 13, 2019 -- Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, and Noile-Immune Biotech, Inc., Tokyo, Japan, a biotechnology company focusing on the development of innovative cancer immunotherapies, today announced that they have entered into a license agreement whereby Autolus will have the right to develop CAR T cell therapies incorporating Noile-Immune’s PRIME (proliferation-inducing and migration-enhancing) technology secreting both IL-7 and CCL19. The PRIME technology is designed to improve proliferation and trafficking into solid tumors of both engineered CAR T cells as well as the patient’s own T cells.

[brexitplus] 05/11/2019 14:57

Autolus Therapeutics to Present Preclinical Data on AUT06NG at the SITC Annual Meeting

Autolus Therapeutics plc a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies for the treatment of cancer, announced today pre-clinical data on AUTO6NG, the company’s next generation GD2-targeting CAR T cell therapy, at the 34th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) being held November 6-10, 2019, in Washington, D.C.AUTO6NG: Next generation GD2-targeting CAR T-cell therapy with improved persistence and insensitivity to TGFb and checkpoint inhibition for relapsed/refractory neuroblastoma.

Background
Neuroblastoma is the most common extracranial solid cancer in children with poor long-term survival in those with high-risk disease.

A currently ongoing phase I clinical study of GD2-targeted CART for refractory/relapsed neuroblastoma (NCT02761915) shows activity against disseminated disease without inducing on target/off tumor toxicity. However, CART persistence was limited and clinical activity transient and incomplete.

Building on the GD2 CAR used in this study, we have developed a next generation T-cell product candidate termed AUTO6NG. The AUTO6NG product consists of 3 distinct populations of GD2-targeted CAR T-cells, produced by dual transduction of T-cells with two separate retroviral vectors. The first vector directs the expression of a GD2-targeting CAR, co-expressed with a constitutively signalling IL7 cytokine receptor (IL7R_CCR) (product A), while the second vector is a tri-cistronic retroviral vector encoding the same GD2 CAR, co-expressed with dominant negative TGFbRII (dnTGFbRII) and truncated SHP2 (dSHP2) (product B). dSHP2 confers resistance to inhibitory signals such as those from PD1.

Methods
Human T-cells were either dual transduced with both vectors yielding a mix of product A/B/A+B (AUTO6NG) or single transduced with each vector individually giving raise to product A or B. Both single and dual transduced CAR T-cells were extensively evaluated in vitro for redirected lysis, cytokine secretion, T-cell proliferation and survival and resistance to immunosuppressive pathways (including TGFb and PD1/PDL1 inhibition) in co-culture assays with GD2-positive and negative tumour cell lines. Additionally, anti-tumour activity of AUTO6NG was evaluated in vivo by intravenous administration in an established neuroblastoma xenograft model in NSG mice.

Results
AUTO6NG T-cells (product A/B/A+B) were highly potent in cytotoxicity assays against GD2 positive tumour cell lines with no differences observed compared with single transduced CAR T-cells (product A or B). Expression of the IL7R_CCR in both AUTO6NG and product A conferred exogenous-cytokine-independent viability and homeostatic proliferation of modified T-cells, without causing autonomous T-cell growth. Furthermore, AUOTO6NG T-cells and product B but not product A proved resistant to both TGFb- and PD1/PDL1-mediated immunosuppression in vitro due to the presence of dnTGFbRII and dSHP2 in those genetically engineered CAR T-cells. Finally, intravenous delivery of AUTO6NG exhibited potent anti-tumour activity and extended survival in NSG mice with established tumour burden.

Conclusions
These results demonstrate the feasibility, safety, and efficacy of AUTO6NG T-cells. The addition of IL7R_CCR, dnTGFbRII and dSHP2 modules to the AUTO6NG product augment its functions by extending T-cell persistence and rendering modified T-cells resistant to TGFb- and PD1/PDL1-driven immune inhibition.

[brexitplus] 05/11/2019 11:46

Thanks Tiger

Autolus Therapeutics to Report Third Quarter 2019 Financial Results and Host Conference Call on November 7

[tiger blue] 05/11/2019 11:27

Hi brexitplus

Not sure if that would include Autolus as already sold from the two unit trusts, only held in Patient Capital which now has Schroders in place to manage it.

That said, good to see a line being drawn under some of the Woodford wreckage and some better sentiment towards particularly the unquoted pharma/life sciences sector would be good all round.

[brexitplus] 05/11/2019 11:15

AUTOLUS???

A consortium led by a specialist life sciences investment bank is close to entering exclusive talks to buy £500m of healthcare assets accumulated by the fallen star fund manager Neil Woodford.

Sky News has learnt that WG Partners, which has assembled a cluster of financial and industry backers including pharmaceuticals investor Ricanto, is the frontrunner to buy roughly 15 positions held by Woodford Investment Management (WIM).

Insiders said the WG-led consortium could secure a period of exclusivity within which to conclude a deal as early as Monday.

The prospective deal comes weeks after Mr Woodford - arguably Britain's best-known retail fund manager - announced that he was shutting his firm amid a deepening crisis.

Other bidders for the portfolio of stakes in listed and privately owned healthcare companies included Rosetta Capital, a life sciences-focused venture fund, and Omega, a hedge fund.

The overall portfolio being pursued by the WG Partners consortium is understood to be valued at close to £500m, according to one insider.

It excludes WIM's holding in BenevolentAI, which uses artificial intelligence to aid drug discovery and which recently saw its valuation halve in a funding round led by the Singaporean state fund Temasek Holdings.

Among the companies included in the proposed transaction are Oxford Nanopore, a DNA sequencing specialist; 4D Pharma, a biotherapeutics specialist; Mereo BioPharma Group, a rare diseases specialist quoted in New York and London; and Rutherford Health, a listed proton beam therapy business formerly called Proton Partners.

The company stakes vary widely in size and value, and a deal is expected to take several weeks to conclude.

WG is said to be in the process of finalising the necessary financing to complete the acquisition.

Mr Woodford became Britain's most prominent biotech investor, but has seen many of those bets turn sour amid uncertainty about the companies' ultimate chances of success.

The sale, which is being overseen by PJT Park Hill Partners, also includes stakes in Arix Bioscience, Immunocore, Kymab, Verseon and 4D Pharma, according to insiders.

Once completed, the deal will mark a further step towards the winding-up of Mr Woodford's eponymous asset management firm.

[ny boy] 24/10/2019 11:52

Bull flag on the chart, I am watching closely but in (STX) which has gone very well since 30p’s, now 184p but US commercialization deal should see a run up to 250/275p this quarter, if that happens I will free up a bit of cash for a stake here.

[luxaeterna1] 22/10/2019 14:18

Gyroscope's Dry AMD eye clinical trials will have data from the the first cohort of patients from around December 2019. I am basing this from the RNS saying January 2019 had the first dosed patient, combined with a trial length of 48 weeks as per the official trial registration.

Interesting times - they now have 7 trial locations recruiting in the UK. I am not clear if these are referral centres, or surgery locations. Full data on P1/2 in early 2021.

[luxaeterna1] 16/9/2019 15:15

With the Freeline pipeline expanding, I am pleased to see progres and if they are getting more comfortable with the technology, I note they have planned pre-clinical trials for: Gaucher disease, Haemophilia A and an inflammation disease planned. Hopefully they can start a new trial every 4-6 months from now on. I'm hoping anyway!

[brexitplus] 12/9/2019 07:16

Freeline, a biotechnology company focused on developing curative gene therapies for chronic systemic diseases, today announced dosing of the first patient in its MARVEL1 study, a multi-centre Phase 1/2 clinical trial of its liver-directed AAV gene therapy for Fabry Disease.
The MARVEL1 study is the first clinical-stage adeno-associated virus (AAV) gene therapy study globally for Fabry Disease. The programme leverages Freeline’s proprietary gene therapy platform, including its novel capsid, which has already shown clinical benefit for Haemophilia B patients.

Fabry disease is a type of lysosomal storage disorder in which certain fatty molecules are not properly metabolised. Patients have a genetic mutation which leads to a deficiency of α-galactosidase A enzyme (αGLA) resulting in an accumulation of lipids, such as globotriaosylceramide (Gb3) and globotriaosylsphingosine (LysoGb3), throughout the body. This can cause highly debilitating progressive multi-organ disease.

It is estimated that Fabry Disease affects one in every 40,000* people. It is currently treated by enzyme replacement therapy (ERT), which requires regular and expensive infusions. The MARVEL1 study aims to deliver a replacement copy of the missing gene to the liver, which will then produce continuous high levels of αGLA, offering the potential for therapy with a single treatment.

MARVEL1 is a multi-centre, international, dose-escalating Phase 1/2 study in adult males with classic Fabry Disease. The study is focused on assessing the safety of FLT190, and its ability to lead to continuous high levels of αGLA production. In addition to safety, endpoints in the study include clearance of Gb3 and LysoGb3 from the plasma, changes in renal and skin biopsies, renal and cardiac function, GLA immune response, viral shedding and quality of life.

Freeline published preclinical data on its Fabry programme in February 2019 at the WORLD Symposium in Orlando and presented further data in May 2019 at the Update on Fabry Disease in Prague, showing that a single dose of its liver-directed AAV gene therapy was able to correct disease in Fabry knockout mice. Freeline has shown long-term follow-up of sustained levels of GLA activity in Fabry knock-out mice and significant reductions of Gb3 in plasma and urine as well as the key disease tissues of kidney and heart, with no adverse effects being observed.

Prof. Derralynn Hughes, Royal Free Hospital, London, UK

“The initiation of this clinical study is an important event for the patient community. I am hopeful that the promising preclinical data will translate into long term benefit for patients with Fabry Disease.”

“The initiation of our MARVEL1 study and dosing of the first patient is a significant milestone for Freeline,” said Chris Hollowood, Executive Chairman of Freeline. “Continuous high expression of alpha GLA holds the potential for better treatment outcomes than is seen with ERT, the current standard of care. We believe we can access high expression at relatively low doses. With two programmes in the clinic on a common proprietary gene therapy platform, Freeline are building a leading systemic gene therapy company using next-generation AAV technology. These innovative gene therapies have the potential to change patients’ lives.”

[brexitplus] 09/9/2019 07:31

Freeline, a biotechnology company focused on developing curative gene therapies for chronic systemic diseases, presented further follow-up data on FLT180a, its AAV gene therapy programme for Haemophilia B, for the first cohort of two patients at the Joint 10th BIC and the 3rd Inhibitor International Conferences (Italy), 6 – 8 September 2019.

Pratima Chowdary, the principal investigator for the FLT180a programme, presented the latest data from the first cohort of two patients who were treated with the lowest study dose (4.5x1011vg/kg). A single infusion at this low dose was well tolerated by patients with severe Haemophilia B with no infusion related reactions. FIX activity remains stable and consistent post steady state at 40 +-5.5 over 66 weeks and 74 weeks post-administration, respectively, with no evidence of transaminitis. Both patients remained free of spontaneous bleeding episodes and did not require any Factor IX supplementation. The Freeline FLT180a programme uses the company’s AAVS3 capsid and a gain of function variant of human factor IX (FIX). The therapy is being evaluated in collaboration with UCL in a Phase 1/2 trial known by Freeline as B-AMAZE, with the goal of normalising FIX activity in patients with moderate and severe Haemophilia B.

Principal investigator Pratima Chowdary said:

“The data is encouraging, showing durability of clinical results for FLT-180a. We are currently progressing the study to identify a dose level that leads to normalisation of FIX activity levels with minimal or no toxicity.”

[brexitplus] 07/9/2019 14:58

Will be interesting to see of Syncona has bought more.