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. Topic sponsors are not involved in the creation of editorial content.

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Pharmaceutical Technology

FEBRUARY 8, 2023

Genenta Science and AGC Biologics have signed a development and manufacturing service agreement (MSA). Under the deal, AGC Biologics will be responsible for manufacturing the cell therapy lentivirus-based product for the ongoing clinical programmes of Genenta Science.

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Pharmaceutical Technology

JANUARY 11, 2023

Anima’s mRNA Lightning platform uses automated high-scale phenotypic screening in live mRNA biology and MOAi technology to identify small molecule mRNA drugs and their mechanisms of action. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper. Optimise your cell therapy process: a guide to cell thawing.

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Pharmaceutical Technology

JUNE 9, 2023

Rocket Pharmaceuticals has received fast track and orphan drug designations for RP-A601 from the US Food and Drug Administration (FDA) to treat plakophilin-2-related arrhythmogenic cardiomyopathy (PKP2-ACM). In April 2023, the FDA approved the investigational new drug (IND) application for RP-A601 to begin a Phase I clinical trial.

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Pharmaceutical Technology

SEPTEMBER 22, 2022

Avrobio has received rare pediatric disease designation from the US Food and Drug Administration (FDA) for its investigational gene therapy, AVR-RD-04, designed to treat cystinosis. Additionally, no adverse events (AEs) linked to the drug have been reported so far. . Cystinosis patients have a high deficiency of this protein.

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Pharmaceutical Technology

JUNE 1, 2023

A new intracellular drug delivery centre will be established in the UK to support potential ribonucleic acid (RNA) vaccines and therapeutics , as well as the development of innovative drug delivery technologies. Topic sponsors are not involved in the creation of editorial content.

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European Pharmaceutical Review

MARCH 10, 2023

A review by researchers on ocular drug delivery discussed strategies that could enhance ocular bioavailability. Currently, effective topical administration is challenging as the industry aims to prolong drug residence time and guarantee appropriate ocular permeation.

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Pharmaceutical Technology

MAY 23, 2023

In 2022, Avrobio’s investigational gene therapy, AVR-RD-04, received a rare paediatric disease designation from the US Food and Drug Administration. Topic sponsors are not involved in the creation of editorial content. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

APRIL 4, 2023

AGC Biologics has entered into a service agreement with the Jikei University in Japan for an idiopathic pulmonary fibrosis (IPF) project. The company will undertake a technology transfer and feasibility study for a drug product focused on the treatment of IPF at its centre of cell and gene expertise in Milan, Italy.

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Pharmaceutical Technology

FEBRUARY 9, 2023

ReviR combines computational and high throughput drug discovery technologies to deliver advanced medicines to patients. The company’s VoyageR AI platform integrates computational methods to drug traditionally undruggable RNA targets. Topic sponsors are not involved in the creation of editorial content.

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World of DTC Marketing

FEBRUARY 7, 2021

Drug Discovery and Manufacturing: It helps in the initial screening of drug compounds to the predicted success rate based on biological factors. Precision medicine or next-generation sequencing helps in the faster discovery of drugs and tailored medication for individual patients. . Measuring RNA, DNA quickly.

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European Pharmaceutical Review

MAY 2, 2023

The US Food and Drug Administration (FDA) has released a new draft guidance to further support the use of decentralised clinical trials (DCTs) for drugs, biologics and devices. Ultimately, the regulatory body hopes the approach will result in more treatment options and improved patient outcomes.

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Pharmaceutical Technology

FEBRUARY 16, 2023

Under the deal, MD Anderson and Xilis aim to advance the development of drug and discovery projects using the MOS technology. It may offer opportunities for third-party collaborations to guide the new cell therapies and drugs development, if successful. Topic sponsors are not involved in the creation of editorial content.

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Pharmaceutical Technology

APRIL 14, 2023

Twist CAR and TCR Libraries allow researchers and drug developers to fine-tune these receptors to address the challenges of cell therapies, including off-target toxicity, enabling rapid advancement toward the clinic.” Topic sponsors are not involved in the creation of editorial content.

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Pharmaceutical Technology

MAY 24, 2023

The therapy has also received orphan drug and fast-track designations. The RMAT designation programme is intended to accelerate the drug’s development and review processes for products, including gene therapies. Topic sponsors are not involved in the creation of editorial content.

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Pharmaceutical Technology

APRIL 28, 2023

The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Editas Medicine’s gene therapy EDIT-301 in sickle cell disease, based on an April 27 announcement. The US agency previously granted the Orphan Drug Designation to EDIT-301 for its study in beta thalassemia, in May 2022.

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Pharmaceutical Technology

AUGUST 25, 2022

The recommendation of the European Medicines Agency (EMA) for Roctavian to maintain orphan drug designation permitting a ten-year market exclusivity window was endorsed by the EC. The company plans to resubmit a Biologics License Application (BLA) for Roctavian by the end of next month.

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Pharmaceutical Technology

APRIL 25, 2023

Forge Biologics has received a qualified person (QP) declaration to manufacture adeno-associated virus (AAV) gene therapies to support European clinical programmes. It received orphan drug, fast track and rare paediatric disease designations from the US Food and Drug Administration.

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Pharmaceutical Technology

APRIL 18, 2023

The US Food and Drug Administration (FDA) has accepted Bristol Myers Squibb and 2seventy bio’s supplemental biologics licence application for Abecma. The regulator has set 16 December 2023 as the prescription drug user fee act date, which is the deadline for the review of new drugs.

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Pharmaceutical Technology

AUGUST 17, 2022

Biopharmaceutical contract development and manufacturing organisation (CDMO) AGC Biologics has entered a strategic collaboration with RoosterBio to expedite the manufacturing of cell and exosome therapies. Topic sponsors are not involved in the creation of editorial content.

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Pharmaceutical Technology

MAY 22, 2023

The US Food and Drug Administration (FDA) has granted approval for Krystal Biotech’s Vyjuvek (beremagene geperpavec-svdt) to treat dystrophic epidermolysis bullosa (DEB) in patients aged six months and above. Vyjuvek is a non-invasive, topical, re-dosable gene therapy that delivers functional human COL7A1 gene copies to offer wound healing.

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Pharmaceutical Technology

MARCH 31, 2023

The new facility is expected to enable the manufacturing of drug products for Kenya and Africa, and will be able to quickly scale up its production and respond to public health emergencies on the continent and worldwide. Topic sponsors are not involved in the creation of editorial content.

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Pharmaceutical Technology

MAY 5, 2023

The move represents Siegfried’s strategy to expand its presence in biologics, and provides important growth opportunities in the mid to long term. Topic sponsors are not involved in the creation of editorial content. DiNAMIQS will initially continue to run under its own name and as a mostly independent venture within Siegfried.

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Pharmaceutical Technology

MAY 12, 2023

Last month, the US Food and Drug Administration (FDA) granted the treatment an orphan drug designation for sickle cell disease. Topic sponsors are not involved in the creation of editorial content. The EHA Hybrid Congress will take place on June 8-11 in Frankfurt, Germany.

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pharmaphorum

NOVEMBER 16, 2022

But combined, OAKS and DERBY were two phase 3 trials involving 1,258 patients […] randomised equally to one of three arms essentially: pegcetacoplan 15mg monthly, same drug same dose every other month, or sham treatment through two complete years,” Dr Wykoff said. Biological teachings and outcomes of the AAO meeting.

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Pharmaceutical Technology

MAY 1, 2023

In March this year, the US Food and Drug Administration (FDA) accepted Ossium Health’s Investigational New Drug (IND) application for OSSM-001 to treat refractory perianal fistulas in Crohn’s disease patients. Topic sponsors are not involved in the creation of editorial content.

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Pharmaceutical Technology

APRIL 21, 2023

Samyang Holdings Biopharm Division CEO Young-Joon Lee stated: “LG Chem has exceptional research and development expertise in innovative drug development and a number of promising new drug pipelines. This is expected to shorten the development time of new drugs and to support personalised treatments.

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Pharmaceutical Technology

JUNE 8, 2023

These solutions will help drug developers address the challenges of regulatory compliance, production scalability, quality assurance and cost-effectiveness. Topic sponsors are not involved in the creation of editorial content. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

MAY 15, 2023

The US Food and Drug Administration (FDA) has given orphan drug designation to SiSaf’s siRNA [a double-stranded RNA molecule that is non-coding] therapeutic, SIS-101-ADO, for the treatment of autosomal dominant osteopetrosis type 2 (ADO2), a rare and serious skeletal disorder in children.

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pharmaphorum

SEPTEMBER 11, 2022

The US FDA has cleared the first-in-class tyrosine kinase 2 (Tyk2) inhibitor as Sotyktu , and BMS says it now plans to launch the new drug later this month, aiming to mount a challenge to Amgen’s $2.3 billion oral psoriasis therapy Otezla (apremilast). Otezla was acquired by Amgen for $13.4 Otezla was acquired by Amgen for $13.4

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Pharmaceutical Technology

FEBRUARY 23, 2023

The company provides end-to-end contract development and manufacturing organisation (CDMO) services from the discovery to the commercialisation of drugs. Topic sponsors are not involved in the creation of editorial content. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

MAY 3, 2023

An anti-CD19 and CD20 bi-specific (CAR-T) therapy, C-CAR039 received regenerative medicine advanced therapy and fast-track designations from the US Food and Drug Administration to treat relapsed/refractory diffuse large B-cell lymphoma (DLBCL). Topic sponsors are not involved in the creation of editorial content.

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Pharmaceutical Technology

JANUARY 12, 2023

The collaboration will use the vector biology, manufacturing capabilities and the drug development experience of Solid Biosciences and Phlox Therapeutics’ knowledge in genetic cardiomyopathies and RNA therapies. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper.

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Legacy MEDSearch

MAY 22, 2023

Food and Drug Administration (FDA) has granted Breakthrough Device Designation for the company’s Orlucent® Skin Fluorescence Imaging System. Orlucent, Inc., a company focused on clinical assessment of suspicious moles, today announced the U.S. “It’s estimated that anywhere from 2% to 53% of U.S.

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Pharmaceutical Technology

APRIL 21, 2023

China’s National Medical Products Administration (NMPA) has cleared CARsgen Therapeutics’ CT041 investigational new drug (IND) for postoperative adjuvant therapy to treat CLDN18.2 Topic sponsors are not involved in the creation of editorial content. positive pancreatic cancer (PC). It targets CLDN18.2

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pharmaphorum

SEPTEMBER 28, 2022

The move is an attempt by the US regulator to encourage sponsors to run paediatric studies – where it is appropriate – and solve the problem of new drugs, biologics, and medical devices coming to market with very little information on how they should be used in young people.

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Pharmaceutical Technology

APRIL 27, 2023

The UK’s National Institute for Health and Care Excellence (NICE) has recommended two new personalised immunotherapy therapies from Kite Pharma to treat aggressive forms of blood cancer for the Cancer Drugs Fund (CDF). Established in 2011, the CDF is a source of funding to increase patient access to cancer drugs in the UK.

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