pharmaphorum

SEPTEMBER 18, 2020

Vertex is already the top player in CF with a quartet of exon-skipping medicines that tackle the mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that underlies the disease, but there are still a sizeable group of patients with no treatment options.

Gene Editing 105

Gene Editing Gene Vaccination Vaccine 105

Drug Discovery World

MAY 6, 2024

Professor Dr Carla van Gils, Director of KWF, comments: “Many of academia’s most innovative agents can be difficult to bring into clinical development. Partnering with the Centre for Drug Development enables us to jump into the bench-to-bedside gap and fast track academic findings into clinical trials.

Drugs 59

Drugs Life Science Gene Editing Gene 59

The Pharma Data

DECEMBER 13, 2020

“Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO Dr. Zhang completed his postdoctoral fellowship at Harvard Medical School/Boston Children’s Hospital.

Business Development 40

Business Development Development Gene Editing In-Vivo 40

Advarra

NOVEMBER 29, 2022

Recent Problems in Cell and Gene Therapy Development. Dealing with confusing regulatory guidelines is one of the top obstacles researchers face as they develop CGT products 1. In the last two years, there haven’t been any CGT product approvals, primarily because of the COVID-19 pandemic.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 52

Delveinsight

SEPTEMBER 14, 2021

The proceeds from the Series B financing will be utilized to progress ADARx’s proprietary RNA platform technologies for base editing, inhibition, degradation, and delivery. Vor said in a statement that it is actively enrolling in its Phase I/II clinical trial for AML patients who currently have restricted treatment options.

RNA 67

RNA Clinical Trials Clinical Trials Drugs 67

Delveinsight

MARCH 2, 2021

To sum it, Chinook will manage clinical development and commercialization of product candidates while Evotec will be eligible for an undisclosed upfront payment, research funding, progress-dependent milestone payments, and tiered royalties on net sales for targets identified through the collaboration.

Drug Delivery 40

Drug Delivery Research Drugs Gene Editing 40

The Pharma Data

NOVEMBER 3, 2020

According to the FDA, the purpose of Fast Track designation is to get important new drugs to patients earlier by facilitating the development, and expediting the review, of drugs to treat serious conditions and fill an unmet medical need. LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Editing Genome 52

The Pharma Data

JANUARY 17, 2021

To complement the internal capabilities, Aruvant is partnering with world-leading contract development manufacturing organizations. The strategic long-term manufacturing agreement with Lonza is a critical step to advancing the clinical development of ARU-1801 for sickle cell patients.

Manufacturing 52

Manufacturing Gene Therapy Trials Gene 52

The Pharma Data

DECEMBER 27, 2020

This year has been difficult, but it has reinforced the importance of our mission: to treat and potentially even reverse the effects of serious diseases and conditions by advancing our novel cell therapy product candidates through clinical trials and into the hands of physicians.

Manufacturing 52

Manufacturing Production Development Clinical Trials 52

Worldwide Clinical Trials

MAY 30, 2024

Differences in regulatory sentiments and industry sponsors’ subsequent clinical development strategies historically restricted access based on geography. was withdrawn, rendering it unapproved for patient use, based on negative review comments by the Committee for Medicinal Products for Human Use. The MAA file in the E.U.

Gene 204

Gene Clinical Trials Clinical Trials Gene Therapy 204

Drug Discovery World

OCTOBER 16, 2023

Dr Fiona Withey, CEO of TrakCel, says: “As a leading provider of software solutions to the advanced therapy sector, TrakCel has facilitated product development and global export from their headquarters in Cardiff, UK. This includes support for export and expansion into new territories as well as for innovation and development.

Drugs 75

Drugs Life Science Gene Therapy Genome 75

XTalks

FEBRUARY 10, 2023

Jeff Vassallo, Senior Director of Clinical Trial Management, at the global clinical contract research organization (CRO) Medpace. These details must be thoughtfully considered early in clinical development to ensure studies are completed within the projected timeline and budget,” added Dr. Vassallo.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Gene Therapy 52

The Pharma Data

DECEMBER 2, 2020

ERS Genomics – Ireland’s ERS Genomics Limited, and Germany’s Vivlion GmbH, announced a non-exclusive license agreement granting Vivlion access to ERS Genomics’ CRISPR/Cas9 patent portfolio, to enhance Vivlion’s gene editing reagents and screening services. Financial details of the agreement were not disclosed. in Mainland China.

Drugs 52

Drugs Gene Editing Genome Genomics 52

The Pharma Data

JANUARY 10, 2021

EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. Previously, the Company received Rare Pediatric Disease designation from the FDA for EDIT-301.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

XTalks

AUGUST 2, 2023

Most of the company’s revenue comes from the manufacture and sale of biopharmaceutical products. They are committed to capitalizing on growth opportunities primarily through the advancement of their own product pipeline and constantly improving their existing products, as well as through business development activities.

Sales 98

Sales Manufacturing FDA Approval Life Science 98

Drug Discovery World

NOVEMBER 30, 2023

Field also covered the capabilities and strategies that enable acceleration of Fc-fusion, Fab fragment and bispecific antibodies through pre-clinical development. It was presented by Lei Shi, PhD, Senior Vice President, R&D, Biointron Biological, titled: ‘Accelerating early discovery through HTP and high-speed antibody production’.

Antibody 59

Antibody Protein Engineer In-Vivo 59

Drug Discovery World

OCTOBER 7, 2022

To achieve this, we have brought our drug discovery sites together under a single leadership team, able to move at speed to partner with academia and industry partners to deliver novel therapies into clinical development. . TH : Cancer Research UK annually invests £40 million in research.

HR 52

HR Research Drugs Development 52

XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize.

Life Science 111

Life Science Vaccination Vaccine Gene 111

Pharmaceutical Technology

MAY 26, 2023

Enzymatic DNA production company Touchlight have augmented its DNA production capabilities with a newly announced expansion to its London facilities. Tripling its production capacity, Touchlight can now manufacture 8kg plasmid DNA, a key component for mRNA gene therapies and vaccines.

DNA 130

DNA Manufacturing Gene Therapy Gene 130