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Vertex eyes controllable genetic drugs with $1.3bn Obsidian alliance

pharmaphorum

Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . Alternatively, a drug can be administered that switches off the genetic medicine and brings protein expression to a halt.

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Gene Therapy and Pharmacokinetics

Camargo

Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Additionally, gene editing allows us either to remove or to modify harmful genes. Formulation Considerations.

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Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug

XTalks

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

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QN-023a by Hangzhou Qihan Biotechnology for Relapsed Acute Myeloid Leukemia: Likelihood of Approval

Pharmaceutical Technology

QN-023a is under clinical development by Hangzhou Qihan Biotechnology and currently in Phase I for Relapsed Acute Myeloid Leukemia. QN-023a overview QN-023a is under development for the treatment of relapsed or refractory acute myeloid leukemia (AML). The company develops gene-editing organ transplantation technology.

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QN-019a by Hangzhou Qihan Biotechnology for B-Cell Acute Lymphocytic Leukemia (B-Cell Acute Lymphoblastic Leukaemia): Likelihood of Approval

Pharmaceutical Technology

QN-019a is under clinical development by Hangzhou Qihan Biotechnology and currently in Phase I for B-Cell Acute Lymphocytic Leukemia (B-Cell Acute Lymphoblastic Leukaemia). The company develops gene-editing organ transplantation technology.

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Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

Pharma Marketing Network

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

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Verve Therapeutics and Lilly partner to advance gene editing programme

Pharmaceutical Technology

Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo gene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). The company will receive a combined upfront payment and equity investment of $60m.