Chapter 11:

Question # 2: What are the recent development in gene therapy?

1. Tigenix’s CX601 Allogenic Cell Therapy

 is a local injectable stem cell therapy for the treatment of Crohn’s disease is a chronic
inflammatory disease of the intestine. A complication of the disease is the development of
complex perianal fistulas. A perianal fistula is an abnormal connection between the perianal
space and the outside skin surface which causes a significant negative impact on quality of
life. It was formulated in Europe last March 2018.

2. Gilead/Kite Pharma’s Yescarta CAR T-cell Theraphy

 The highly personalized cancer treatment called CAR T-cell therapy (CAR is short for
chimeric antigen receptor) is made by Kite Pharma, a company that in August was acquired
by biotech giant Gilead Sciences and was approved by FDA.

 for treatment of adult patients with relapsed or refractory large B-cell lymphoma after two
or more lines of systemic therapy.

 It's a type of cancer immunotherapy, which harnesses the body's immune system to take on
cancer cells. In this case, the therapy removes a person's cells, reengineers them, then puts
them back in their body to attack cancer cells.

3. Spark Therapeutic Luxterna gene therapy

 Based on PHILADELPHIA, Sept. 21, 2018 (GLOBE NEWSWIRE),Spark Therapeutic

Luxterna gene therapy is a fully integrated, commercial gene therapy company dedicated to
challenging the inevitability of genetic disease today announced that the Committee for
Medicinal Products for Human Use(CHMP) of the European Medicines Agency (EMA)
adopted a positive opinion recommending approval of LUXTURNA® (voretigene
neparvovec), a one-time gene therapy for the treatment of adult and pediatric patients with
vision loss due to inherited retinal dystrophy caused by confirmed
biallelic RPE65 mutations and who have sufficient viable retinal cells.

4. Novartis’s Kymriah CAR T-cell Therapy

 This was approved by US Food and Drug Administration (FDA) last august 2017,
 This is use for the treatment of patients up to 25 years of age with B-cell precursor acute
lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.