Author Archives: Car-T

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

Top 15 private biotech companies of 2015

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Alector: “We are developing drug leads against unique, genetically-validated neuro-immune targets through a novel, hypothesis-driven approach to neurodegeneration. We thankFierceBiotech for recognizing the potential of our strategy,” said Arnon Rosenthal, co-founder, president and CEO of Alector. Release

Arvinas: “We are thrilled to be selected by FierceBiotech to be among the top private biotech firms globally,” Manuel Litchman, president and chief executive officer of Arvinas, said. “This award recognizes the dedication of our employees, our founder, Dr. Craig Crews, and our partners to bring our PROTAC technologies to patients with … Read more

Drug Development Process

About Drug Development Process

Clinical trials involve the administration of an investigational pharmaceutical product to individuals under the supervision of qualified medical investigators. Clinical studies are conducted in accordance with protocols that detail the objectives of a study, the parameters to be used to monitor safety, and the efficacy criteria to be evaluated. Each protocol is submitted to the appropriate regulatory body and to a relevant IRB/REB prior to the commencement of each clinical trial. Clinical studies are typically conducted in three sequential phases which may overlap in time-frame.

In … Read more

CRISPR – EMBRYO EDITING

https://flipboard.com/@naturenewsteam/embryo-editing%3A-the-ethics-of-crispr-27j1164kz

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.

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ABEONA THERAPEUTICS

Tim Miller, Ph.D., President & CEO
Dallas, TX
(NASDAQ: ABEO)
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

Global Blood Therapeutics – respiratory distress syndrome


Global Blood Therapeutics initially focusing on indications with significant unmet need, including idiopathic pulmonary fibrosis (IPF) and acute respiratory distress syndrome (ARDS), in which hypoxia is believed to play a key role in disease pathogenesis and adverse patient outcomes. These are conditions in which the lungs cannot supply adequate oxygen to the blood. While supplemental oxygen therapy is a well-established lifesaving treatment in acute and chronic hypoxemic conditions, it is associated with a number of risks, including injury or infection as a result of intubation. This highlights a significant unmet … Read more