Drug Target Review Cell & Gene Therapy ebook
15 June 2022
This ebook features articles from gene therapy specialists that look at current platforms as well as the future of promising modalities for gene therapies. It also tackles novel stem cell technology considerations and gives insight into the careful balancing act between efficacy and safety in gene therapy.
Dr Mary Spellman, Chief Medical Officer and Senior Vice President of R&D at Castle Creek Biosciences, explains how both ex vivo and in vivo platforms can be used to alter human genetic sequences for therapeutic benefit, highlighting the benefits of both approaches for rare skin and connective tissue disorders as well as rare metabolic diseases.
Dr Gaurav Shah, co-founder of Rocket Pharmaceuticals, shares both the progress and obstacles that his gene therapy company is experiencing in bringing life-changing therapies to patients with rare diseases.
Focusing on cell therapy, Dr Terri Gaskell, Chief Technology Officer at Rinri Therapeutics, explores some pre-clinical challenges and question associated with using a novel stem cell technology as a potential therapeutic strategy for hearing loss.
Viral vectors are the delivery agent of choice for some approved therapies, however Brent Warner, President of Gene Therapy at Poseida Therapeutics, shares how non-viral gene therapy might be a viable option when delivered via DNA and RNA nanoparticle systems.
In this ebook:
- Leveraging a dual technology platform to develop gene therapies for a range of rare diseases
Dr Mary Spellman, Chief Medical Officer and Senior Vice President of R&D at Castle Creek Biosciences
- Challenges and opportunities: lessons learned in developing potential gene therapy cures
Dr Gaurav Shah, Chief Executive Officer of Rocket Pharmaceuticals
- The promise of cell therapy: scaling up a new treatment paradigm for hearing loss
Dr Terri Gaskell, Chief Technology Officer at Rinri Therapeutics
- Providing new hope for rare disease using non-viral gene therapy
Brent Warner, President, Gene Therapy at Poseida Therapeutics