Hemophilia B cure by gene therapy

 
Hemophilia B gene therapy pipeline
Company Project Mechanism Notes Trial details
Baxalta (Shire) BAX 335 AAV8-Padua FIX Phase III to start 2016 NCT01687608
UniQure/Chiesi AMT-060 AAV5-wild-type FIX Low-dose phase I/II data presented at EHA; high-dose data H2 2016 NCT02396342
Spark/Pfizer SPK-9001 Bio-engineered AAV-Padua FIX Low-dose phase I/II data presented at EHA NCT02484092
Dimension Therapeutics/ Regenxbio DTX101 AAVrh10-wild-type FIX Initial phase I/II data due H2 2016 NCT02618915
Sangamo Biosciences SB-FIX In vivo protein replacement using zinc finger nucleases Phase I trial started in 2016 NCT02695160
Freeline Therapeutics hemophilia B Gene
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Gene therapy for osteoarthritis ?

GeneQuine develops drugs that are based on gene therapy. The concept of gene therapy is to introduce genetic material into the patient’s own cells in the body. The cells produce then a therapeutic protein according to the template that the introduced genetic material (DNA) provides

GeneQuine is focused on the development of gene therapy agents for the treatment of osteoarthritis. Osteoarthritis is a degenerative joint disorder characterized by cartilage loss and inflammation. Patients affected by osteoarthritis experience joint pain as well as swelling and stiffness of the joints leading to … Read more

Gene editing target disease ?


Genome Editing with CRISPR-Cas9

Here’s a short list of some common diseases that might be curable or preventable with gene editing:

Autism
Breast cancer
Colon cancer
Hemophilia
Huntington’s disease
Marfan, Parkinson’s
Prostate cancer
Retinitis pigmentosa
Sickle cell
Skin cancer
Tay-Sachs
Wilson
Duchenne muscular dystrophy
Crohn’s
Color blindness
Cystic fibrosis
Down syndrome
Polycystic kidney
Turner syndrome.

There are hundreds of other more rare genetic disorders.… Read more

Gene therapy for spinal muscular atrophy (SMA) Type 1

Spinal-muscular-atrophy

Gene therapy is a well-suited approach for the treatment of SMA due to the monogenic nature of the disease—meaning it’s caused by the deletion of or mutations in a single gene. AVXS-101 is our clinical-stage, proprietary gene therapy candidate of a one-time, intravenous treatment for SMA Type 1—designed to prevent further muscle degeneration caused by SMA through:

Delivery of a fully functional human SMN gene into target motor neuron cells
Production of sufficient levels of SMN protein required to improve motor neuron function
Rapid onset of effect in addition to … Read more

New Genetic Therapy Could Erase Down Syndrome

We need to keep eye on http://elixirgen.com/ This is new company so not alto of detail but they are trying.

https://lejeuneusa.org/blog/curing-down-syndrome#.Vm8-o0orJpg

http://elixirgen.com/

http://www.nature.com/news/researchers-turn-off-down-s-syndrome-genes-1.13406

I will keep this in my alert list.

http://hereandnow.wbur.org/2013/07/17/therapy-down-syndrome… Read more