Syncona Share Discussion Threads

[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.

[ltinvestor] 07/9/2019 08:07

I think that Woodford will be forced to sell his total holding in Autolus.There appears to be no shortage of buyers for his stock.....He can now sell at $14

[luxaeterna1] 06/9/2019 16:12

Worth watching on the monitor, Autolus is up 65% on yesterday

$9.85 to $16+ in 24 hours.

[tiger blue] 06/9/2019 15:35

Citywire reporting that Woodford has sold Autolus from his Equity Income & Income Focus funds. They think he has retained the Patient Capital holding. AUTL up again today.

[ltinvestor] 06/9/2019 08:00

TB. Certainly looks that way...

[tiger blue] 05/9/2019 22:02

Autolus up 21.7% today, nice but nothing to celebrate given the extent of the decline. More important was the volume of 7.8m shares, methinks some stock overhang has finally moved on, hopefully Woodford into safer hands

[luxaeterna1] 05/9/2019 10:22

Autolus updated corporate presentation a couple of days ago. Seems all systems are go and the management team are keen to push things along now hxxps://autolus.gcs-web.com/static-files/d053be07-f6a1-4762-b96f-2eac212b811d

[alan@bj] 03/9/2019 11:36

Autolus Therapeutics today announced that the journal Nature Medicine has published both pre-clinical results and clinical data from the ongoing Phase I CARPALL trial of AUTO1, demonstrating the potential of the company’s novel CAR T therapy targeting CD19 in development for the treatment of pediatric acute lymphoblastic leukemia (ALL). The paper reports that AUTO1, or CAT Chimeric Antigen Receptor T cells (CAT CAR T), utilizes a binder with a fast off rate and showed both increased proliferation/ cytotoxicity in vitro and enhanced proliferative capacity and anti-tumor activity when compared to FMC63 CAR T therapies in vivo. In the Phase 1 clinical trial, 86% (n=14) of recurrent/refractory pediatric ALL patients achieved molecular complete remission after a single dose, with a median duration of remission of 7.4 months and no severe cytokine release syndrome (CRS; ≥ grade 3 or 4), in this relapsed and/or refractory patient population.

[ltinvestor] 03/9/2019 10:51

Sorry I meant overlooked by PI’s!!

[kristini2] 03/9/2019 09:17

overlooked? It's constantly traded at a massive premuim to NAV ????How's that overlooked

[ltinvestor] 03/9/2019 08:51

Pity there aren’t more companies like Syncona listed on the market yet Syncona continues to be overlooked by the investment community...

[brexitplus] 03/9/2019 07:49

Findings from Phase I CARPALL Trial Demonstrate that Autolus’ Novel CAR T Therapy, AUTO1, Induces Enhanced Anti-tumor Response Without Severe Cytokine Release Syndrome 86% molecular complete response rate after a single dose of AUTO1

Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, today announced that the journal Nature Medicine has published both pre-clinical results and clinical data from the ongoing Phase I CARPALL trial of AUTO1, demonstrating the potential of the company’s novel CAR T therapy targeting CD19 in development for the treatment of pediatric acute lymphoblastic leukemia (ALL). The paper reports that AUTO1, or CAT Chimeric Antigen Receptor T cells (CAT CAR T), utilizes a binder with a fast off rate and showed both increased proliferation/cytotoxicity in vitro and enhanced proliferative capacity and anti-tumor activity when compared to FMC63 CAR T therapies in vivo. In the Phase 1 clinical trial, 86% (n=14) of recurrent/refractory pediatric ALL patients achieved molecular complete remission after a single dose, with a median duration of remission of 7.4 months and no severe cytokine release syndrome (CRS; ≥ grade 3 or 4), in this relapsed and/or refractory patient population.

“The safety profile emerging from this pediatric study is encouraging. AUTO1 was well-tolerated and we did not see severe cytokine release syndrome or neurotoxicity seen in other ALL programs,” said Sara Ghorashian, PhD, Molecular and Cellular Immunology Section, UCL Great Ormond Street Institute of Child Health and a co-author of the paper. “It is very promising to see these strong remission rates and excellent CAR T cell expansion and persistence, which give us hope that AUTO1 could improve outcomes for these patients.”

[luxaeterna1] 16/8/2019 15:06

Seems like the market got wind of the 5 month delays on the Autolus manufacturing site, unavoidable in biotech to some extent, could create a value opportunity as AUTL is trading pretty terribly ($45 to $9 in a year!)

[luxaeterna1] 01/8/2019 15:38

..Jan 2020! (cough, typo)

[luxaeterna1] 01/8/2019 15:28

Gyroscope dosed their first patient in February 2019, it sounds like they are running a 7-clinic UK trial that is intended to last until January 2019. Looking forward to that, although it may be slightly difficult to interpret the results on AMD/vision.