Pharma Marketing Network

DECEMBER 21, 2020

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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Outsourcing Pharma

JANUARY 18, 2024

Inceptua Group and Cycle Pharmaceuticals partnership will give access to Free Goods Partnership that gets medicine to patients with extremely rare genetic conditions, which they would be unable to access otherwise.

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pharmaphorum

APRIL 23, 2021

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. . For example, adding a small-molecule might stabilise the medicine, allowing levels of the target protein to increase.

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Pharmaceutical Technology

MAY 24, 2023

Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases.

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Pharmaceutical Technology

AUGUST 24, 2022

Prevail will also handle the complete production, clinical development and marketing activities. Lacerta is entitled to receive an upfront payment as well as development and commercial milestone payments from Prevail. In January last year, Lilly acquired biotechnology company Prevail for nearly $1.04bn.

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Development Gene Therapy Genetics Gene 130

Drug Discovery World

FEBRUARY 12, 2024

Why has there been so little progress in medicine? 7 Human biology-based research At Safer Medicines Trust, we believe that using human biology-based research is the best way to develop safe and effective treatments for patients. Rat Trap: The Capture of Medicine by Animal Research – and How to Break Free.

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Medicine Genome Genomics Drugs 59

Scienmag

JUNE 8, 2021

Data supports QurAlis’ approach to treat hyperexcitability induced neurodegeneration in ALS patients; QurAlis gears up for clinical development of therapeutic candidate, QRL-101 CAMBRIDGE, Mass.–(BUSINESS

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The Pharma Data

DECEMBER 13, 2020

As part of the collaboration, Hummingbird will be leveraging Tempus’ AI-enabled platform and proprietary data, as well as joining its TIME Trial® Network, for rapid identification, site activation and efficient enrollment of cancer patients who have NRG1 fusions and meet eligibility criteria for HMBD-001 clinical trials. About Tempus.

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Pharmaceutical Technology

DECEMBER 15, 2022

These targets will be selected with genetic validation and mechanistic hypotheses, preclinical validation and subject stratification biomarkers to streamline the assets’ clinical development. Additionally, the drug discovery capabilities of PrecisionLife will be used to offer a clear rationale to Ono for choosing targets. .

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Drug Discovery World

MAY 17, 2023

Leveraging historical trial databases has become commonplace in assessing the feasibility of trials in therapeutic areas with a large breadth of clinical development history. EHR data also has a key role to play in designing future clinical trials.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Genetics 52

Pharmaceutical Technology

AUGUST 17, 2022

The rights to the oRNA-LNP technology platform of Orna will be retained by the company, which will also progress various other fully owned programmes in oncology and genetic disease areas. In addition, Merck will make a $100m equity investment in Orna’s Series B funding round concluded recently.

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The Pharma Data

OCTOBER 21, 2020

and Shionogi Limited as shareholders, today announced the positive findings of a pooled analysis of COVID-19-related impacts across the investigational long-acting cabotegravir and rilpivirine clinical development programme. About the long-acting regimen of cabotegravir and rilpivirine. Shionogi joined in October 2012.

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Pharmaceutical Technology

MARCH 1, 2023

These therapies validated antisense technology as a new platform for drug discovery and for targeting rare genetic disorders. RNA-based therapeutics are a rapidly growing category of drugs that may improve the standard of care (SOC) for many diseases and could soon become the mainstay of personalised medicine.

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Drug Discovery World

APRIL 24, 2024

Evotec will identify best-in-class small molecules targeting a key fibrotic pathway with strong genetic support identified by Variant Bio and progress the programme towards the selection of a clinical development candidate(s) using its R&D platform.

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Development In-Vivo In-Vitro Genome 52

pharmaphorum

JULY 5, 2022

Cell therapy specialist Mogrify has struck a deal with Japanese drugmaker Astellas to look at ways to deploy regenerative medicine to treat hearing loss caused by factors such as chronic exposure to loud noises. SNHL is the most common form of hearing loss, account for more than 90% of cases and affecting millions of people worldwide.

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Drug Discovery World

FEBRUARY 10, 2023

The research will support the clinical development of CTx001, CTx’s lead gene therapy asset for the treatment of GA, by enabling the identification of patients with complement-driven AMD for future interventional studies. The post Non-interventional geographic atrophy study launches appeared first on Drug Discovery World (DDW).

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Gene Therapy Gene Genetics Clinical Development 52

Drug Discovery World

FEBRUARY 1, 2024

Vivet is a clinical stage biotech company developing gene therapies for rare inherited metabolic disorders. CTX is a rare autosomal recessive genetic disorder that affects the body’s ability to metabolise fats known as cholesterols.

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Gene Therapy Gene Genetics Manufacturing 52

XTalks

OCTOBER 20, 2023

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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The Pharma Data

DECEMBER 17, 2020

NASDAQ: REGN ) today announced that the New England Journal of Medicine (NEJM) has published initial clinical data from an ongoing seamless Phase 1/2/3 trial of the antibody cocktail casirivimab and imdevimab in non-hospitalized patients with COVID-19. TARRYTOWN, N.Y. , 17, 2020 /PRNewswire/ — Regeneron Pharmaceuticals, Inc.

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Pharmaceutical Technology

DECEMBER 22, 2022

Blueprint Medicines wins the Financing award this year after securing significant financing partnerships worth up to $1.25bn. The funding will offer capital to extend Blueprint’s varied pipeline toward marketing as well as to continue seeking strategic and synergistic business development prospects. Innovation.

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XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. Gene-altering and other advanced medicines are radically rewriting the future for people with some of the rarest, and most devastating, diseases on earth. “They called him ‘The Boy.’ Adjusting for COVID-19.

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The Pharma Data

MAY 1, 2023

“The fluctuating symptoms and unpredictable nature of atopic dermatitis, along with limited medicines that can adequately manage long-term uncontrolled symptoms, represent major challenges in the treatment of this chronic disease,” said Jenny Murase, M.D., Lilly and partner Almirall S.A. expect regulatory decisions in the U.S.

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Drug Discovery World

JANUARY 15, 2024

The NVIDIA DGX SuperPOD will be installed at Amgen’s deCODE genetics’ headquarters in Reykjavik, Iceland and will be used to build a human diversity atlas for drug target and disease-specific biomarker discovery.

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Drug Discovery World

AUGUST 28, 2023

DS: What role does genetics play in autophagy? Could therapies like these treat rare genetic conditions? PH : Genetics plays an enormous role. In fact, some variants of Parkinson’s and ALS are genetic in origin. Could it be possible to prevent genetic diseases? DS: What’s next for the company’s pipeline?

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Genetics Protein Genetic Disease Clinical Trials 52

pharmaphorum

SEPTEMBER 14, 2020

AstraZeneca has resumed UK trials for its coronavirus vaccine, after the country’s medicines regulator gave the all-clear following a safety scare. A UK safety committee has concluded its investigations and recommended to the country’s Medicines and Healthcare products Regulatory Agency (MHRA) that trials are safe to resume.

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XTalks

FEBRUARY 24, 2023

Genentech’s ongoing clinical development program for Lunsumio is evaluating the drug as a monotherapy and in combination with other medicines for the treatment of B-cell NHLs, including FL and diffuse large B-cell lymphoma (DLBCL). When did Lunsumio launch in the US and how is the launch of Lunsumio progressing?

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The Pharma Data

MAY 31, 2022

Dupixent would be the first and only medicine specifically indicated to treat prurigo nodularis in the U.S., The potential use of Dupixent in prurigo nodularis is currently under clinical development, and the safety and efficacy have not been fully evaluated by any regulatory authority.

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Genetics Antibody Medicine Trials 52

The Pharma Data

SEPTEMBER 16, 2020

executive vice president of Research and Development at Amgen, along with members of Amgen’s clinical development team and clinical investigators, will discuss Phase 1 data being presented on the Company’s investigational KRAS G12C inhibitor sotorasib (AMG 510). Reese , M.D., On Monday, Sept. PDT , David M.

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Drug Discovery World

MARCH 5, 2024

As research efforts continue in these areas, there is also the linked hurdle that unlike anti-seizure medicine which typically acts quite instantly on the seizure, more holistic symptoms can be more challenging to measure and often take more time to analyse, meaning research efforts also take more time and increase in complexity.

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pharmaphorum

MARCH 14, 2022

Chief Executive Officer, Chief Scientific Officers, Chief Medical Officers, Managing Directors, CEO, Head of Department, Principal Research Scientists, Clinical lead, Clinical Operations in the following fields: • Pain and Migraine. Analgesic pre-clinical development. CNS clinical trials. • Drug development.

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Clinical Trials Clinical Trials Development Medicine 52

Drug Discovery World

SEPTEMBER 14, 2023

Excluding contributions from Comirnaty and Paxlovid, company revenues grew $537 million, or 5%, operationally. AstraZeneca saw total revenue reach $22,295m, up 4% despite a decline of $2,181m from Covid-19 medicines. Excluding Covid-19 medicines, total revenue increased 16% and product sales increased 15%. Specialty care grew 11.8%

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XTalks

APRIL 11, 2023

Most people typically develop Parkinson’s disease after the age of 60, and early onset forms of Parkinson’s disease are often inherited, though some forms may be linked to genetic mutations or exposure to environmental toxins like pesticides. The hashtag encourages people to take six minutes to raise awareness for Parkinson’s disease.

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The Pharma Data

OCTOBER 27, 2020

The company plans to tackle the existing challenges of precision medicine with a three-prong approach – developing better medicines for known cancer-driving mutations, developing drugs for targets previously thought to be undruggable, and finding new, untapped targets that could lead to even better drugs. Sirnaomics.

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Gene Gene Therapy Medicine Genetics 52

Camargo

MAY 2, 2021

Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product developers. Note: Some medicines are in more than one category.

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Drug Discovery World

JUNE 19, 2023

This collaboration between PrecisionLife and Nanopharmaceutics uses mechanistic patient stratification biomarkers to inform clinical trial design and transform the lives of patients.

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Drug Discovery World

SEPTEMBER 14, 2023

Erdafitinib has demonstrated promising results in advanced, FGFR-altered UC, making this submission a vital step towards improving outcomes for patients in the future.

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XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. The European Medicines Agency (EMA) initially recommended six novel advanced therapy medicinal products (ATMPs) with orphan designation in 2022, but the orphan designation of one of these ATMPs was later withdrawn.

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