Category Archives: Gene therapy

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
Read more

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 therapy for spinal muscular atrophy (SMA) Type 1


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

Bioviva – Ending Ageing through Gene Therapy

BioViva‘s CEO Liz Parrish is a fire-starter and probably not everyone’s favorite person. But if your goal is as bold as defeating aging then you’ll have to break some eggs. And so feel as you might but I have to admit that Parrish is definitely one of my favorite people – not only for the scientific work that she does but also because she is a fun, passionate and fully committed individual that inspires me. Thus it was no surprise that, despite some technical difficulties with my set up, I … Read more


A new genetic engineering technology called CRISPR/Cas9 has opened the door to unprecedented tinkering with the human genome. Nature takes a look at the scientific possibilities and the ethical dilemmas they raise.… Read more

Gene Therapy and regenerative medicine companies.



Tim Miller, Ph.D., President & CEO
Dallas, TX
PlasmaTech Biopharmaceuticals/Abeona Therapeutics is focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases. PlasmaTech’s lead program is a gene therapy for Sanfilippo syndrome (MPS IIIA and IIIB) in collaboration with patient advocate groups, researchers and clinicians. In addition, the company is pursuing two additional proprietary platforms, Salt Diafiltration (SDF™) Process and Polymer Hydrogel Technology (PHT™), and is active in the development and commercialization of human plasma-derived therapeutics, including its proprietary

Read more

RNAi Technology companies

Acuity Pharmaceuticals (now OPKO Health)
Aegera Therapeutics
Agilent Technologies
Alcon (subsidiary of Novartis)
Allele Biotechnology
Altogen Labs
AmpliPhi Biosciences
Antisense Therapeutics
Aptose Biosciences
Arcturus Therapeutics
ArmaGen Technologies
Arrowhead Research
Ascletis Pharmaceuticals
Atugen AG (acquired by Silence Therapeutics)
Avecia OligoMedicines
Benitec Biopharma
Biogen Idec
Biomatik Corporation
Biomics Biotechnologies
Biomics Biotechnology
BioSpring GmbH
Blueberry Therapeutics
Bristol-Myers Squibb
Calando Pharmaceuticals (subsidiary of Arrowhead)
CRISPR Therapeutics
Cubist Pharmaceuticals (acquired by Merck)
Dainippon Sumitomo
Dharmacon RNAi Technologies (part … Read more