pharmaphorum

OCTOBER 8, 2020

Scribe Therapeutics, a start-up focusing on gene-editing using CRISPR/Cas9, has burst onto the biotech scene with a $415 million deal with Biogen. It will also use a variation of CRISPR/Cas9 known as CRISPR/Casx which is smaller, making it easier to deliver using viral vectors for example, and more efficient at modifying genes in the body.

Gene Editing 55

Gene Editing Gene In-Vivo Clinical Trials 55

Delveinsight

JANUARY 12, 2021

Medivir, IGM Biosciences enters into an exclusive licensing agreement for Birinapant. Medivir AB has entered into an exclusive licensing agreement with IGM Biosciences to receive global, exclusive development rights for Birinapant. AvantGen Enters into a Licensing Agreement for its Anti-SARS-CoV-2 Antibodies with IGM Biosciences.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9 Cancer is king .

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Launched in 2019, the company raised $700 million in June 2020 in its initial financing.

In-Vivo 52

In-Vivo Marketing Engineer Genetics 52

Drug Discovery World

JULY 9, 2024

At Taconic Biosciences, we are dedicated to advancing drug discovery by providing researchers with straightforward access to the most sophisticated research models engineered to meet the highest genetic, health, and quality standards. Therefore, licensing agreements play a crucial role in determining the availability of these models.

In-Vivo 59

In-Vivo Genetic Engineering Genetics Drugs 59

Drug Discovery World

SEPTEMBER 14, 2023

Also in July 2023, Alexion, AstraZeneca Rare Disease (Alexion) and Pfizer entered into an agreement for Alexion to purchase and license the assets of Pfizer’s early-stage rare disease gene therapy portfolio for a total consideration of up to $1bn, plus tiered royalties on sales. billion, a decrease of $15.0 Vaccines were up 9.1%

Drugs 59

Drugs Vaccination Vaccine Medicine 59

The Pharma Data

DECEMBER 9, 2020

The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.

In-Vivo 52

In-Vivo In-Vitro Antibody Development 52

The Pharma Data

APRIL 8, 2022

Based on these studies, one of those drugs was licensed to Cantex Pharmaceuticals for the treatment of COVID-19 and other inflammatory lung diseases. Every breath stretches the lungs’ tissues with each inhale and relaxes them with each exhale. a Wyss Technology Development Fellow at the Institute. During his Ph.D.

Immune Response 52

Immune Response In-Vivo Genetic Analysis Research 52

The Pharma Data

AUGUST 3, 2021

SVP, Chief Medical Officer, Novartis Gene Therapies. “We SVP, Chief Medical Officer, Novartis Gene Therapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. 2 years and <5 years old with SMA Type 2.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

The Pharma Data

MARCH 20, 2023

Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post-dosing, children who were treated after presenting symptoms of SMA maintained all previously achieved motor milestones.

Gene Therapy 40

Gene Therapy Gene In-Vivo Licensing 40

Drug Discovery World

DECEMBER 12, 2023

KRRO-110 is designed to co-opt an endogenous enzyme Adenosine Deaminase Acting on RNA (ADAR) to repair a pathogenic single nucleotide variant (SNV) on RNA and restore production of normal A1AT. Korro’s RNA editing approach has the potential to be transformative for AATD patients and Korro is well-positioned to deliver a best-in-class therapeutic.

RNA 52

RNA In-Vivo Genotype Genetics 52

Drug Discovery World

NOVEMBER 9, 2023

Peter O’Callaghan, PhD, Head of Expression System Sciences, Biologics and Licensing, Lonza, on: ‘Solve bispecific heavy-light chain mispairing with bYlok technology’. Croasdale-Wood will then participate in a keynote chat, interviewed by Miho.

Engineer 52

Engineer Antibody Protein In-Vivo 52

The Pharma Data

OCTOBER 15, 2020

Axovant – Kristin Vuori was named to the board of directors at Axovant Gene Therapies Ltd. Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. BeyondSpring – BeyondSpring Inc. Additionally, Parag V.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52

Drug Discovery World

FEBRUARY 26, 2024

For example, Vertex Pharmaceuticals’ stem-cell treatment for sickle-cell disease and transfusion-dependent β-thalassemia was authorised by the Medicines and Healthcare products Regulatory Agency (MHRA) in November 2023, making it the first licensed therapy that uses the gene-editing tool CRISPR to help treat patients.

Manufacturing 52

Manufacturing In-Vivo Development Production 52

The Pharma Data

JULY 8, 2021

Food and Drug Administration (FDA) has accepted a Biologics License Application (BLA) and granted Priority Review for tezepelumab in the treatment of asthma. Amgen (NASDAQ:AMGN) announced that the U.S. Tezepelumab is being developed by Amgen in collaboration with AstraZeneca. Reese, M.D., About Severe Asthma.

Allergies 52

Allergies Trials In-Vivo Medicine 52

Drug Discovery World

FEBRUARY 22, 2024

According to Medicine Discovery Catapult, these include: “Navigating the complexity of the CNS; understanding the disease pathology, and accessing predictable in vitro and in vivo cell models 2 ”. Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In In 2019, dementia accounted for 1.8

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59