Syncona Share Discussion Threads

[ltinvestor] 30/5/2020 09:47

Excellent Autolus results totally overlooked by UK investors .Expecting much more US interest in Autolus as trials proceed.Surely this is going to be taken out by a major Pharma for multiples of its present price..

[luxaeterna1] 29/5/2020 13:08

Autolus update today

DLBCL trial going well by the sounds of it. 50-70% complete response, ongoing durable responses.

If I read this correctly, the safety results have been good enough that they are moving to an outpatient model (i.e. not keeping the patient in the hospital during their treatment), which really underpins their potential.

I am not sure if anyone has had a safe CAR-T suitable for outpatient use trialled yet?

Good impetus. Best of luck to the team.

[sev22] 29/5/2020 11:06

(Alliance News) - Syncona Ltd on Friday said portfolio company Achilles Therapeutics Ltd has dosed the first patient in its Phase I/II study of its treatment for patients with recurrent or metastatic malignant melanoma.

The healthcare-focused investment firm explained that Achilles's tumour-infiltrating lymphocyte therapy has been designed to target clonal neoantigens, which are the protein markers on the surface of all cancer cells that are unique from patient to patient.

Achilles will use its bioinformatics platform PELEUS as well as its TRACERx data set derived from cancer patients over many years, to identify a patient's unique tumour profile and manufacture bespoke T-cells, called cNeTs, aimed at targeting and destroying tumours without harming healthy tissue.

Syncona said the study is a single-dose Phase I/II trial designed to evaluate the safety, tolerability and clinical efficacy of cNeT therapy in 20 patients with recurrent or metastatic malignant melanoma. Test results are expected to be published in the first half of 2021.

"We are delighted that Achilles has dosed its first patient with its proprietary cNeT cell therapy. This is the first TIL therapy to enter clinical trials that is specifically designed to target all cancer cells, and that represents an exciting prospect for science and for patients. Importantly, today's news demonstrates Achilles' capability to manufacture an entirely personalised T-cell therapy, and therefore its potential to deliver a commercially viable therapy that could dramatically improve patient outcomes," said Martin Murphy, chief executive of Syncona Investment Management Ltd & non-executive director of Achilles Therapeutics.

Shares in FTSE 250-listed Syncona were trading flat at 204.60 pence each on Friday morning in London.

[sphere25] 26/5/2020 13:27

Watching for a breakout here. Threatening one with the stock currently up 5% at 210p.

[luxaeterna1] 07/5/2020 16:26

There is a new corp presenation on the Syncona website. Some nice information on it.

Not sure if this had been published before re Freeline:

"Dose optimisation continuing in Haemophilia B; six patients have completed follow-up for six months, and amongst those, three have FIX activity levels over 50%."

50% was basically within "normal" healthy patient parameters. So encouraging data on that front.

[brexitplus] 10/3/2020 10:01

Freeline, a biotechnology company focused on developing curative gene therapies for chronic systemic diseases, today announces that the European Commission (EC) has granted orphan drug designation for FLT190 for the treatment of Fabry Disease, based on a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA).

[luxaeterna1] 04/3/2020 10:03

Bloody virus temporarily interrupting our uptrend.

I recommend AUTO12NG takes on a new viral target !

[acuere] 23/2/2020 13:21

Mentioned in the Mail today.

[milehouse] 20/2/2020 15:23

Back to £2.50 and back to green. I expect this will steady at £2.55/£2.60 until we have some news from Freeline/Gyroscope. 30% premium to NAV has been normal for this stock in the past, hopefully we have now blown the Woodford cobwebs away and this can resume.

[sev22] 18/2/2020 13:55

On a technical basis and analysing Syncona's latest chart it looks like a break out is about to occur.

[sev22] 17/2/2020 08:08

SYNCONA INVESTOR RELATIONS JUST RELEASED THIS UPDATE - Developing a pipeline of small molecule therapeutics for patients with immunological and genetic disorders - Oxford, 17 February 2020 – OMass Therapeutics (“OMass”), a biotechnology company harnessing native mass spectrometry to drive drug discovery in high definition, today announced completion of an extended Series A financing of £27.5 million from existing investors Syncona Ltd (“Syncona̶1;) and Oxford Sciences Innovation (“OSI”) who contributed £16.6 million and £10.4 million respectively. They were joined in the round with £0.5 million from the University of Oxford.

This comes after an initial Series A investment of £14 million in 2018 bringing the total series A to £41.5 million.OMass is leveraging its world-class biophysical discovery platform to deliver high-resolution data on how a putative ligand engages with a membrane protein target, focusing on GPCRs and solute carriers. Proceeds from this financing will allow the Company to progress a pipeline of small molecule therapeutics for patients suffering from immunological and genetic disorders providing funding for two years and enabling OMass to progress its lead program into preclinical development.

“It is an exciting time in our progression as we discover, develop and ultimately commercialize novel medicines to bring life-changing benefits to patients with immunological and genetic diseases”, said Ros Deegan, Chief Executive Officer, OMass Therapeutics. “I would like to thank our investors, Syncona, OSI and the University of Oxford for their belief in the technology’s ability to deliver new biological insights and new chemical starting points for membrane targets.”

“We are pleased to continue to support OMass, a world-leader in native mass spectrometry that allows non-covalent interactions with membrane protein targets to be studied in high-definition,R21; said Dr Edward Hodgkin, Chairman, OMass Therapeutics and Partner at Syncona. “The Company has now initiated programs against three high impact GPCR targets with a focus on genetically defined patient populations as well as immunological dysfunction, and we look forward to OMass progressing its products towards clinical development.”

“Emerging biophysical technologies like native mass spectrometry can meaningfully improve drug discovery and impact global health outcomes,” said Lachlan Mackinnon, Principal, Oxford Sciences Innovation. “We formed OSI to turn Oxford’s world beating science into world changing companies and believe that this funding puts us in a position to take full advantage of OMass Therapeutics’ capability to identify novel drugs against membrane proteins.”

[sev22] 15/2/2020 11:13

The Autolus share price was up 6.6% in the US last night.

[ltinvestor] 15/2/2020 09:26

It’s truly amazing that such a great company is overlooked by the PI community.....

[sev22] 14/2/2020 09:23

Published 14 Feb 2020:

Gyroscope Therapeutics, a company developing gene therapies and surgical delivery systems for retinal diseases, announces the strengthening of its leadership team with the hiring of Dr. Nadia Waheed from Tufts University School of Medicine in Boston as Chief Medical Officer (CMO) and the promotion of Dr. Jane Hughes, formerly VP Translational Research, to Chief Scientific Officer (CSO).

These additions to the Gyroscope Executive Committee come as the company is currently enrolling its initial Phase I/II clinical trial for the lead investigational gene therapy GT005 and preparing to move into its proof of concept Phase II clinical program.
Khurem Farooq, Chief Executive Officer, Gyroscope Therapeutics said: “We are delighted to welcome Nadia as CMO and Jane to her new role as CSO. Nadia’s expertise in ophthalmology and understanding of the patient experience will be invaluable at this stage in our development, as we continue to move our clinical programme forward and broaden our reach across the United States and the rest of the world. Jane’s expertise has been critical in building the biological understanding to support our advancement into the clinic. She has a proven track record as a translational biologist who has effectively bridged between pre-clinical and clinical programs. I welcome both to the Gyroscope Executive Committee and look forward to working together to develop our gene therapy for one of the leading causes of vision loss, dry age-related macular degeneration.”

Dr Nadia Waheed said: “This is an exciting time for me to join Gyroscope. As a retinal surgeon, and a frequent collaborator with innovative companies, my passion has been to find meaningful solutions for patients facing retinal diseases. GT005 represents a unique approach as a one-time gene therapy that may slow the progression of a blinding disease for patients for whom currently there is no good option. I’m excited to be part of a team that I believe will make a difference for people with dry AMD.”

Dr Jane Hughes said: “Over the past year I’ve been privileged to work with the researchers and scientific founders at Gyroscope to better understand the complement system and its role in dry AMD to bring GT005 from an idea in a laboratory to an investigational gene therapy. I’m excited about the next phase of research growth that lies ahead in building the Gyroscope pipeline of medicines.”

[luxaeterna1] 14/2/2020 09:16

New autolus and freeline presentations up on their websites.

Check out slides 12-13 of the Freeline one at for big smiles!

Factor IX activity well into 100-150% territory

[sev22] 12/2/2020 17:18

(Alliance News) - Syncona Ltd on Wednesday said its investee Freeline Therapeutics has revealed "promising" data from its gene therapy studies for the treatment of Fabry and Gaucher diseases.

Syncona holds a 79% stake in Freeline.

Fabry disease is a rare genetic disease that affects many parts of the body including the skin, eyes, gastrointestinal system, kidney, heart, brain, and nervous system. Gaucher is an inherited genetic disorder that results in the build-up of certain fatty substances in organs such as the spleen and liver, causing the affected organs to enlarge, thereby affecting their function.

Syncona said that preliminary data from the FLT190 study programme for Fabry showed that Freeline's adeno-associated viral gene therapy can deliver sustained levels of the alpha-galactosidase A enzyme from a single treatment. It added that a dose-escalating Phase 1/2 study in adult males with the disease provided encouragement that higher levels of the enzymes could be achieved through dose-escalation.

The alpha-galactosidase enzyme can help digest sugars and fats. When it's missing, complex sugars and fats can easily build up within the gut and throughout the body. The enzyme helps the body break carbohydrates, depriving gas production fuel and toxic bacteria.

Data from the dose escalation study showed a three to four-fold increase in enzyme activity was achieved by week four, and sustained through the data cut-off at week 20.

The biotechnology investment company also said that pre-clinical data in Gaucher disease suggests that the adeno-associated viral gene therapy has therapeutic potential. Data suggests a single injection of its treatment would result in sustained steady levels of fully active beta-glucocerebrosidase enzymes in the bloodstream.

Beta-glucocerebrosidase is an enzyme that helps break down a large molecule called glucocerebroside into glucose and simpler fat molecules.

"As of the data cut-off date, there have been no infusion-related adverse events. Two adverse events were reported in the study. The patient had elevations in ALT levels and levels returned to normal range within 3 weeks following immune suppression. A grade 2 myocarditis was observed but repeat MRI scans showed no evidence of changes compared to base line and it resolved without specific intervention," said Derralynn Hughes, clinical director of haematology & oncology at the Royal Free Hospital London.

Syncona shares were trading 0.6% higher in London on Wednesday afternoon at 237.00 pence each.

[sev22] 12/2/2020 14:10

ANNOUNCED AT 2.00 PM:

Freeline presents clinical data at 16th Annual WORLDSymposiumTM

12 February 2020

Syncona Ltd, a leading healthcare company focused on founding, building and funding a portfolio of global leaders in life science, notes that its portfolio company Freeline today announced data at the 16th annual WORLDSymposiumTM in Orlando, Florida, 10th - 13th February 2020. Preliminary data were presented on the starting dose in a Phase 1/2 dose escalation study for Fabry Disease, the first AAV gene therapy study in Fabry Disease globally, and pre-clinical data was presented in Gaucher Disease.

· Promising preliminary data presented in Fabry Disease; demonstrating for the first time that AAV gene therapy can deliver sustained levels of the enzyme αGLA[1] from a single infusion

· Freeline has demonstrated that it is able to deliver high protein levels in its Phase 1/2 trial in Haemophilia B at higher doses, which provides encouragement that higher αGLA levels could be achievable in Fabry through dose escalation

· Encouraging preclinical data in Gaucher Disease suggesting that AAV liver-directed gene therapy has therapeutic potential

The announcement can be accessed on Freeline's website at:

[sev22] 12/2/2020 08:48

I am not sure whether someone has already posted a feature from John Rosier in the Investors Chronicle about three weeks ago.

He holds 3% of Syncona in his 2020 portfolio and said that the Autolus overhang, which depressed the share price last year, had now cleared and he was expecting an imminent re-rating in the stock.

Apparently Syncona's shares historically manage a 30% plus premium to net asset value (NAV)which equates to a minimum of £2.60.

The other reason he continues to hold is the company's excellent record of creating real value in the life sciences industry.

[ltinvestor] 09/2/2020 09:44

Freeline alone in which Syncona own 80% could be worth the present mkt cap in 24 months time!

[luxaeterna1] 08/2/2020 10:03

Video update on the Freeline RNS.

[luxaeterna1] 07/2/2020 21:08

Yeah, this level of response is nearing the "will survive serious surgery" levels of FIX activity.

[ltinvestor] 07/2/2020 16:10

Wonderful results for those suffering from the illness

[luxaeterna1] 07/2/2020 14:04

Interesting freeline RNS out, if I'm not mistaken, this is the first trial ever to "cure" the haemophilia FIX to normal levels.

Time will tell and there are several hurdles to go.