Roots Analysis

OCTOBER 30, 2023

In the past few years, Next Generation RNA therapeutics have emerged as one of the key therapeutic modalities in the modern healthcare industry. These RNA based therapeutics play a crucial role in protein production and regulation of gene functions.

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pharmaphorum

APRIL 19, 2022

The funding will be used to continue the clinical development of lead oligonucleotide PGN-EDO51 for DMD, as well as two other oligo drugs PGN-EDODM1 for myotonic dystrophy type 1 (DM1) and PGN-EDO53, another DMD candidate. Once there, they disrupt the expression of RNA coding for disease-associated proteins.

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Pharmaceutical Technology

FEBRUARY 22, 2023

CMN-005 is under clinical development by CoImmune and currently in Phase II for B-Cell Acute Lymphocytic Leukemia (B-Cell Acute Lymphoblastic Leukaemia). The cell therapy is developed based on C.I.K. It acts by targeting the cancer cells which express CD19 antigen. It is administered through intravenous route.

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Delveinsight

SEPTEMBER 14, 2021

ADARx bags USD 75 Million to advance its RNA tech pipeline. ADARx Pharmaceuticals, a biotechnology company developing RNA targeting therapeutics , announced the completion of a USD 75 million Series B financing to progress its drug development pipeline. SR One Capital Management and OrbiMed Advisors co-led Series B.

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XTalks

JUNE 14, 2023

More Drugs for Dry Eye Disease Are on the Way Several other drugs have recently gained FDA approval or are currently in advanced stages of clinical development for the treatment of dry eye disease. One such approved medication is Miebo , an ophthalmic solution developed by Bausch + Lomb and Novaliq.

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pharmaphorum

NOVEMBER 18, 2021

Danish drugmaker Novo Nordisk must like what it has seen in its two-year-old alliance with gene-silencing specialist Dicerna Pharma – it has just agreed to acquire the biotech for $3.3 billion in cash. If approved, it could become a $500 million-a-year product, according to the company.

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Pharma Marketing Network

DECEMBER 21, 2020

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. greater safety, transient and reversible effect on gene expression).

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Genetics Medicine Gene Therapy Marketing 52

pharmaphorum

MARCH 29, 2022

“C9orf72-associated ALS is a complex genetic form of ALS and there are multiple mechanisms by which the scientific community believes the C9orf72 gene causes disease. Based on the results from this study, Biogen and Ionis have confirmed that the BIIB078 clinical development programme will be discontinued.

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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 Prior to that, he held various oncology research and development positions at Merrimack Pharmaceuticals and Archemix.

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Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. 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. Gene Therapy Definition.

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The Pharma Data

OCTOBER 19, 2020

Caris Precision Oncology Alliance members also have access to the Caris Pharmatech oncology trial network, which can help reduce the time it takes to identify and connect appropriate patients with novel targeted cancer therapies in clinical development.

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The Pharma Data

MARCH 28, 2022

Based on these results, the BIIB078 clinical development program will be discontinued, including its ongoing open-label extension study. “We Vice President and Head of the Neuromuscular Development Unit at Biogen. About Ionis Pharmaceuticals, Inc.

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The Pharma Data

MARCH 11, 2021

Sanofi Pasteur, the vaccines global business unit of Sanofi, and Translate Bio (NASDAQ: TBIO), a clinical-stage messenger RNA (mRNA) therapeutics company, today announced the start of the Phase 1/2 clinical trial for MRT5500, an mRNA vaccine candidate against SARS-CoV-2, the virus that causes COVID-19. About Translate Bio.

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The Pharma Data

JANUARY 17, 2021

About STP705 Sirnaomics’ leading product candidate, STP705, is a siRNA (small interfering RNA) therapeutic that takes advantage of a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to directly knock down both TGF-?1 1 and COX-2 gene expression.

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Gene Expression Trials Development Production 52

pharmaphorum

APRIL 28, 2022

The new hub will focus on centralising things like clinical development strategy, clinical operations, and regulatory approval strategy – not necessarily drug development itself. How big pharma tackles rare diseases.

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The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

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pharmaphorum

DECEMBER 23, 2020

2020 also saw some of the first “tumour agnostic” cancer drugs get to market, with Bayer’s Vitravki (larotrectinib) getting funding in the UK for tumours with confirmed neurotrophic tyrosine receptor kinase (NTRK) gene fusions.”. It was the third approval from Alnylam’s pipeline of RNA interference therapeutics to make it to market.

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The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics.

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Roots Analysis

AUGUST 13, 2024

Messenger ribonucleic acid (mRNA) is a type of single-stranded ribonucleic acid (RNA), which helps in transferring genetic information in order to produce proteins. The success of mRNA-based therapeutics and vaccines has increased the research activity in novel / next generation RNA therapeutics market.

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The Pharma Data

NOVEMBER 24, 2020

The safety and efficacy of Oxlumo are also being evaluated in the ongoing ILLUMINATE-C Phase 3 clinical trial in patients of all ages with advanced PH1, including patients on dialysis. Visit OXLUMO.com for more information, including full Prescribing Information. Until today, there were no approved pharmaceutical therapies for PH1.

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Worldwide Clinical Trials

MAY 30, 2024

Differences in regulatory sentiments and industry sponsors’ subsequent clinical development strategies historically restricted access based on geography. An area of interest has been deciphering ALS’s genetic underpinnings and delivering functional copies of dysfunctional genes to the patient.

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Delveinsight

AUGUST 5, 2021

Sanofi is betting the genetic technology behind the fast development of two highly effective coronavirus shots last year will result in vaccines for other viruses as well as drugs for diseases of the lung and liver, announcing a deal to acquire a research partner Translate Bio for USD 3.2 Mestag is led by CEO Susan Hill, Ph.D.,

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XTalks

AUGUST 2, 2023

Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. 2022 was the first year on market for cell-based gene therapies Abecma (Idecabtagene vicleucel) and Breyanzi (lisocabtagene maraleucel) , which generated $388 million and $182 million, respectively. Both are anti-CD20 agents.

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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.

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XTalks

JANUARY 6, 2025

From leveraging artificial intelligence (AI) to streamline diagnostics and treatments to exploring the untapped potential of RNA-based therapeutics, biotechnology is shaping the future of healthcare and beyond. As of January 31, 2024, approximately 131 unique RNA-based therapies are in clinical development across various therapeutic areas.

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XTalks

SEPTEMBER 14, 2020

The company’s candidate vaccine, mRNA-1273, is a synthetic messenger RNA that encodes the stabilized SARS-CoV-2 spike protein. Below are some free webinars you can register for that have delved into the topic: Continuing Clinical Research Innovations After COVID-19. Addressing the Rapidly Evolving Vaccine Development Landscape.

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