Syncona Share Discussion Threads

[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.

[rambutan2] 05/2/2020 20:57

Tiger Blue, that's a good and fair write up. It was the emphasis on the platforms which I thought very enlightening, and encouraging. But much else also well worth listening to.

[sev22] 05/2/2020 12:12

Syncona is a 'strong buy' having just hit its 200 day moving average of 231.00.

[ltinvestor] 05/2/2020 12:03

TB.I believe that Autolus will be taken out for many times it’s present mkt cap but time will tell....

[tiger blue] 05/2/2020 11:42

DL - lol, but in this case I think heavier is better

[davidlloyd] 05/2/2020 11:32

Is he on a diet then? Seems keen to get on the scales.......

DL

[tiger blue] 05/2/2020 10:49

CM day was indeed successful. Webcast recording on SYNC website, as always DYOR but do please watch if you have more than a passing interest. These are not annual or even bi-annual events for SYNC, and there is a lot of insight and an opportunity to learn about the whole current portfolio, which certainly gave me reassurance about my holding.

Positives: there will be lots of newsflow to come from Freeline, Achilles and Gyroscope over the next 12-24 months as these are scaling rapidly. I think the reason share price has responded is the focus on the platforms and manufacturing they are building, that enable new products to be rolled out quicker and more efficiently. The takeover of Spark Therapeutics by Roche for $4.3bn was referenced, the price paid was far too much for the Haemophilia candidates they have, they bought it mainly for the platform. All of Syncona’s companies have a strong focus on the manufacturing of product in reliable, closed processes. Note that the recent Autolus data showed vastly superior survival rates from the closed manufacturing process vs the open.
The presentation and panel discussions showed the value of being close to the science (literally 200 yds from UCL) and the benefit of sharing ideas and experience among the companies, as well as their long term approach which the scientists & founders prefer to venture cap, who always put the exit strategy on the first page.

Concerns/negatives: Not many, though Brexit could be a pain in adding extra costs for different regulations in UK/EU, and long term may require manufacturing to be set up in EU as well as UK/US. Attracting European recruits may also be harder. Autolus (my view & I still hold) - I think you have to say given the share price that the jury is out as to whether they will ever get a product to market, the data is encouraging but there are other players. Price already heavily down so only 11% of SYNC portfolio and I think Freeline will be much bigger soon, but scope for a big bounce should AUTL succeed.

Share price – I wouldn’t have been brave enough but someone asked Martin Murphy about the share price and I’ll end with a direct quote from him – “We can’t manage the share price, what we can do is manage the business….in the long run we all get weighed, and I’m looking forward to being weighed, because frankly the value will come out”.

[1seanshare] 05/2/2020 10:37

What causes that type of spike?

[ltinvestor] 05/2/2020 09:29

Looks like a very successful Capital Markets Day judging by the share price movement

[sev22] 04/2/2020 09:46

Syncona Ltd, a leading healthcare company focused on founding, building and funding global leaders in life sciences, announces the appointment of Lorenz Mayr, Ph.D. as Entrepreneur in Residence, from 1 January 2020. In this role, he will have a particular focus on sourcing new Syncona companies, developing and driving portfolio company business plans and strategy.

Lorenz has 25 years of experience in the pharmaceutical and biotechnology industry, spanning drug discovery and clinical development in areas including molecular biology, molecular imaging, antibodies, cell and gene therapy, and gene editing. Lorenz was most recently Chief Technology Officer at GE Healthcare, where he was responsible for leading the company’s global R&D strategy. Prior to this, he held senior leadership roles at AstraZeneca Plc, Novartis Pharma AG and Bayer AG. Lorenz is a lecturer for Biochemistry at the Martin-Luther University, Germany, and is a member of several Scientific Advisory Boards.

Martin Murphy, Chief Executive of Syncona Investment Management Limited, said: “Lorenz is a highly regarded member of the scientific community and is a fantastic addition to the Syncona team. His knowledge and experience in the sector will enhance our search for best-in-class science that has commercial prospects and the potential to make meaningful differences to patients.”

Lorenz Mayr, Entrepreneur in Residence of Syncona Investment Management Limited, added: “I look forward to joining the team at Syncona and helping them seek opportunities to found new companies. Syncona has an excellent track record and I’m excited to be part of a process that brings exceptional science from concept to clinic.”

[sev22] 04/2/2020 09:40

Life sciences company investor Syncona said its net asset value total return had crept up 0.2% in its fiscal third quarter.

The rise had slightly reduced to 7.0% the fall in the company's net asset value total return for the nine months through December.

Syncona's life science portfolio was valued at £516.6m at the end of December, up from £481.3m at the end of September.

A total of £39.m of capital was deployed in the quarter and £167.1m over the nine months from the end of March.

'We have had a productive third quarter, funding Freeline as it continues to scale and leading a financing in a new Syncona company, Azeria, where there is an exciting opportunity to build a world-class oncology company,' Syncona Investment Management chief executive Martin Murphy said.

'We have also seen strong clinical progress across the portfolio with Autolus, Freeline and Gyroscope progressing seven clinical trials.'

"We continue to take a hands-on approach to capitalise on the significant opportunities ahead.'

'We have a strong pipeline of opportunities to found new companies and will ambitiously fund our growing portfolio.'

[tiger blue] 01/2/2020 16:12

My bad, Capital Markets Day should read Tuesday, not Monday.

[tiger blue] 31/1/2020 11:47

Don't forget the Capital Markets Day on Monday from 13:00. Was going to attend but they are showing a live webcast (requires registration) which saves a trip to London.
Pleased to see that the opening address will be by the Chair of the Wellcome Trust which underlines my belief that they will remain a long term supportive holder having already taken their cost out.

There will be no inside info but nevertheless it will provide valuable insight into all of Syncona's existing projects and future vision, and a very rare opportunity to see some of the leading experts and founders such as Martin Pule (Autolus and whose work was the subject of the BBC programme) discuss their work and where the science is today.

[luxaeterna1] 31/1/2020 10:08

I'm trying to find some details on competitor products.

Yescarta – 51% of patients had complete recovery from DLBCL measured at 7.9 months median (101 patients)
AUTO3 – 57% of patients had the same at approx. 6 months median (18 patients).

While Yescarta has the ‘normal’ potentially fatal side effects, AUTO3 has the safety ‘off switch’.