biopharmeceutical therapeutic

User Generated

aznffnuv

Engineering

Description

I have picked KYMRIAH https://www.us.kymriah.com/acute-lymphoblastic-leu... as the biopharmeceutical therapeutic and need answers and research to answer the fallowing questions. I will need slides answering each of the fallowing bullets and also script for each slide explaining them.

Each group will research, compile, and present a 15-20 minute powerpoint presentation on a biopharmaceutical therapeutic, to address topics covered in class including but not limited to:

·Title slide- Drug name, manufacturer (and location)

·Diseases/disorders treated, mechanism of action

·Primary research- where completed, academia vs. industry, summary of findings

·Intellectual property status (patent year, patent type(s), indications, biosimilar status?)

·Development genetics

·Platform technology- protein or antibody

·Host cells and upstream production details (culture media/conditions, scale-up, process control strategies)

·Cell bank system(s) utilized

·Downstream product recovery and purification

·Product formulation, fill-finish

·Advanced strategies used (PAT, QBD, Continuous processing)

·Active substance testing, product stability, bio-burden

·Clinical trials and FDA approval process

·Annual sales revenue, market share, other drugs in class, US approval, EU approval? other approvals? Post-market research? Evidence of drug re-purposing? Future outlook?

·References slide

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Explanation & Answer

Attached.
Attached.

Surname 1
Student’s name:
Instructor:
Course:
Date:
KYMRIAH script
Slide one: Title slide
Slide two: Diseases/disorders treated
Tisagenlecleucel is the real name of the drug but it is marketed as KYMRIAH (Pharma
US, 2017). It is used to treat the B-cell acute lymphoblastic leukemia cancer (B-cell all).
In regard to mechanism of action, KYMRIAH reprograms the T-cells of a patient with transgene
encoding of chimeric antigen receptor (CAR). The CAR then identifies and destroys any CD19expressing tumor, malignant or normal cells. The CAR is a single-chain antibody with the ability
to recognize CD19. The CAR is fused in the intracellular signaling domains – CD3 zeta and 41BB (CD137) (Pharma US, 2017). The CD3 zeta is responsible for malignant destruction and
initiation of T-cell activation. The 4-1BB enhances the sensitivity, persistence, and expansion of
KYMRIAH. Once the CD-19 expressing cell is identified, the CAR signals T-cells to expand,
activate, and work on the destruction of the malignant cell.
Slide three: Primary research
KYMRIAH was once used in the study B2202 with 68 subjects. The major adverse effect
noted in the study was cytokine release syndrome (CRS). Hemophagocytic lymphohistiocytosis
(HLH) was also noted, with other side effects occurring due to CRS.
Grade 3 and 4 CRS was noted among 32 individuals. This was 47% of the sample (Pharma US,
2017). CRS resulted in no deaths. Other neurological toxicities included delirium,

Surname 2
ancephalopathy, seizure, hallucinations, mental status changes, cognitive disorder, dysphagia,
and somnolence. Another study revealed that KYMRIAH increased in the body with time.
After 28 days from infusion, KYMRIAH was found to be 44% in the patient’s bone marrow
(Pharma US, 2017). The product was found to be 67% and 69% respectively for 3 and 6 months
after infusion.
Slide four: Intellectual property status
Novartis and the University of Pennsylvania patent the product - KYMRIAH. The patent
was effected in 2012. The CAR-T technology is patented by Memorial Sloan Kettering Cancer
Center whereas Novartis (BioPharm In...


Anonymous
Just what I needed…Fantastic!

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