Member Info for Haywain

cant seem to get a timetable for the speakers but the conference goes on until Wednesday with companies presenting on all 3 days.

Maybe it doesn’t take a month - it’s just the time scale I use for development batches normally approx a month to do analytical testing and write the reports

JHFH; every batch gets tested otherwise there is no point in running the batch (in my opinion) : might not be full testing and doesn’t have to be validated methods at this stage but I am pretty certain they will want the data even if it is just for information only or to develop the methods further. Agree with you on economy of scales and I have no idea what the cocoon system costs but I hope they do plan to work in parallel as you have said.

Should know in a couple of weeks whether the pilot run for car-t was successful : takes up to a month to perform the analytical work but I imagine they will wait for the data before making a decision to go ahead with engineering and demonstration batches so I don’t think they will be running in parallel.

I Imagine there is a lot that Vlad would like to share with the shareholders must be very frustrating to have to wait until all the i's are dotted and t's are crossed but we will get to know soon enough. Have a good weekend all.

I have posted this before but what you see them using for their process development run is lonza's cocoon system - impressive piece of kit and very scalable - check it out here for those interested: https://www.youtube.com/watch?v=S1p1Nf5HU5g

personally I am happy with small daily tick ups for a while before news - if it makes a big jump it attracts the nutters from the sharerisers board

the procedure is outlined here: https://www.fda.gov/drugs/investigational-new-drug-ind-application/ind-application-procedures-overview

You are right - when I say reviewed I mean absence of clinical hold letter within 30 days of receipt of the IND. FDA dont approve the IND- If they object they will send a clinical hold letter within the 30 days of receipt of the IND.

It could be but I have just had an IND reviewed in 30 days maybe it is specific ot CAR-t?

Normally 30 day FDA review for IND (not that it makes much difference). Also FDA's review in the early stages is normally more focused on the clinical protocol rather than the chemistry manufacturing control (although I havn't worked on a CAR-t)- If they tick the boxes in terms of the CMC data requested and they have listened to FDA's guidance on the clinical protocol then we shouldn't have any FDA related hold ups - when you are developing a process for the first time however obviously there can be set backs along the way for a multitude of reasons- that said I too think they are in a good spot from here in the run up to the end of the year.

I am in a call at the same time as this so unfortunately will not be able to attend - hopefully I will read at least some of the discussions on here. I see it as a positive that Vlad is open to this sort of shareholder interaction and it can only be a good thing.

I think you just have to ask yourself what has changed - if nothing then it doesnt really matter what the share price does on a day to day basis - it jsut becomes relatively cheaper for the proposition on the table or relatively more expensive but the proposition is the same: Only new information (scientific or financial) can change your judgement on the long term future of the company. I appreciate that it might be frustrating if your timeframe is a shorter one but it really doesnt do you any good to sweat the short term movements.

nice - thankyou JHFH

There is a useful draft guidance as to the requirements to meet FDA expectations for IND for CAR-T's that can be accessed here: https://www.fda.gov/media/156896/download.
It also seems that the manufacturing process can be developed on the system that they are currently using "cocoon system" - see youtube video here: https://www.youtube.com/watch?v=S1p1Nf5HU5g. The manufacturing process will use patients T-cells obtained through leukophoresis. The lentiviral transfection strain is being developed and characterised by WuXi (announced in December 2021) : https://advancedtherapies.com/platform-cell-and-gene-therapies/lentiviral-vector-platform/ - It is difficult to predict when they will have done sufficient testing to get the go ahead from FDA but significant pieces of the puzzle appear to be in place - fingers crossed for a 2022 initiation of phase 1

Apologies if already posted but the intro to Vlads talk tomorrow at 9.35 was published in the conference prospective there are some heavy hitters presenting so it should be at least a good networking opportunity:
9:35 Anti-FLT3 CAR T and Bispecific Antibody Therapies for
Effective Elimination of AML and Conditioning for Hematopoietic
Stem Cell Transplantation
Vladislav M. Sandler, PhD, CEO & Co-Founder, Hemogenyx Pharmaceuticals
Relapsed/refractory acute myeloid leukemia (R/R-AML) has a poor prognosis
even with hematopoietic stem cell (HSC) transplantation. Surface expression
of FLT3 in leukemic cells and in HSCs provides an opportunity for targeted
therapy to eradicate FLT3+ R/R-AML and facilitate bone marrow conditioning.
We have developed chimeric antigen receptor modified T cells (CAR T)
targeting FLT3 and a humanized bispecific antibody (CDX) that recruits T cells
to eliminate FLT3-expressing cells.

oh yeah I forgot to mention solvaldi was the name of the drug that gilead bought (RNA polymerase inhibitor) and it generated 19 billion dollars of revenue on a single indication.

What I particularly like about CBR is that it is potentially a platform to generate treaments targeted to specific viruses - This fits the model of big pharma although it is slightly disruptive because big pharma likes to inhibit a viral infection to manage the disease rather than cure (more profitable). Personally I was encouraged all those years ago when pharmasset was procured by Gilead for their HCV treatment as this was the first instance of big pharma being interested in a cure for hepatitis C. However, a broad spectrum antiviral was created by MIT's Dr Todd Ryder called double stranded RNA activated caspase oligomerizer (DRACO for short) this involved a protein that recognises very long double stranded RNA (which is unique to viruses as they hijack the cell to drive viral replication) binds and activates caspase (the enzyme that signals apoptosis) to kill any cell infected by a virus. This was tested in-vitro on multiple viruses and in-vivo (mice/flu) and worked yet no funding was recieved + no interest from big pharma- I can only imagine because it is broad spectrum and is a cure therefore didn't fit the pharma model of going after a single indications and managing the disease. Todd Ryder recently sold his patent to a start up company called Kimermed based in NewZealand and they are carrying on his work. The reason Hemo's offering in this space is different is that it isnt broad spectrum but a platform that would need to be tailored to each virus (unless viruses share binding proteins targets for cell entry) and therefore would (I imagine) appeal to big pharma.

In theory if it works on corona viruses then that is exactly what he has done Hemotruth as the common cold is caused by corona/rhino viruses. But is is not some small molecule that could be delivered as a capsule or tablet to millions at first sign of a cold. It will be expensive to make and I presume therefore limited to serious viral/bacterial disease where a patient has run out of treatment options or that no treatment options exist.

You can just bed down ISA which essentially means that your shares will be sold the broker will sit on the money for a couple of days as it clears and then they repurchase shares in you ISA account - you will lose out on spread and fee but essentially you can just instruct them to do it