Drug Discovery World

JANUARY 5, 2023

CANbridge Pharmaceuticals has exercised its option to secure the exclusive global rights to develop, manufacture and commercialise a novel gene therapy to treat spinal muscular atrophy (SMA) from UMass Chan Medical School. . The benchmark therapy uses a vector similar to that used in the only gene therapy approved for SMA.

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pharmaphorum

JUNE 27, 2022

The safety of Astellas’ gene therapy portfolio has been thrust into the spotlight once again, after the FDA placed a clinical hold on a trial of its Pompe disease candidate AT845. The post Another Astellas gene therapy hits a safety hurdle appeared first on.

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Drug Discovery World

DECEMBER 20, 2022

The first gene therapy for haemophilia B could soon be available to patients in Europe following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP). . The post First gene therapy for haemophilia B gets go ahead in Europe appeared first on Drug Discovery World (DDW).

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pharmaphorum

FEBRUARY 8, 2022

With the safety of gene therapies thrown into the spotlight by a series of FDA clinical holds, Astellas has said that an interim readout in a phase 1/2 trial of its Pompe disease candidate AT845 hasn’t found any cause for concern. The post Astellas says Pompe gene therapy clears safety hurdle appeared first on.

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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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Pharmaceutical Technology

JUNE 15, 2023

The deal gives Astellas the option to exclusively license its rights for clinical development and commercialisation for any protein degrader compounds developed within the lead programme. Astellas could pay Cullgen up to $85m upon using its license option regarding the deal.

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The Pharma Data

JANUARY 12, 2021

Marianne De Backer, Head of Business Development & Licensing in Bayer’s Pharmaceuticals Division, pictured above. Also in December, the company partnered with Atara Biotherapeutics to develop off-the-shelf T-cell immunotherapies for high mesothelin-expressing tumors. Photo courtesy of Bayer. We have set the bar high.

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Pharmaceutical Technology

AUGUST 24, 2022

Lacerta Therapeutics has entered a new licensing and research partnership agreement with Eli Lilly and Company subsidiary, Prevail Therapeutics, to discover and develop adeno-associated virus (AAV) capsids for treating central nervous system (CNS) diseases. . Topic sponsors are not involved in the creation of editorial content.

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Delveinsight

DECEMBER 8, 2020

KaliVir, Astellas Pharma forms a licensing deal for VET2-L2 oncolytic virus. KaliVir Immunotherapeutics and Astellas Pharma entered into a worldwide exclusive licensing agreement for the development, research, and commercialization of VET2-L2 to widen the horizon of therapeutic approaches available in the Immuno-Oncology market.

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Pharmaceutical Technology

AUGUST 4, 2022

Poseida will carry out the Phase I clinical trials and produce clinical materials for a subset of both the company’s portfolio programmes licensed or optioned to Roche and the companies’ future partnership programmes, before transferring the programmes to the latter for additional development and marketing.

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pharmaphorum

JANUARY 13, 2022

Sanofi has joined a growing list of drugmakers going after alpha-synuclein targeting drugs for Parkinson’s disease, licensing a bispecific antibody from South Korea’s ABL Bio in a deal that could be worth more than $1 billion.

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Drug Discovery World

SEPTEMBER 16, 2022

W here globally is innovation occurring and what factors – industry, regulatory, and academia – are helping developers get their products to market? Here, DDW Editor Reece Armstrong explores the global hubs of innovation for cell and gene therapies. The UK stands as one of the key markets for cell and gene therapies.

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The Pharma Data

OCTOBER 26, 2020

(Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. Fast Track designation allows for early and frequent communication with the FDA throughout the entire drug development and review process.

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Drug Discovery World

SEPTEMBER 14, 2023

joined Pfizer’s Executive Leadership Team as Chief Oncology Research and Development Officer and Executive Vice President reporting to Chairman and Chief Executive Officer, Albert Bourla.

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The Pharma Data

APRIL 27, 2022

We plan to discuss these data with health authorities, and we will continue to expand our tislelizumab clinical development program in pursuit of novel, synergistic combinations with the ultimate goal of extending survival for more patients.”. About Novartis Novartis is reimagining medicine to improve and extend people’s lives.

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FDA Law Blog

JANUARY 4, 2023

The purpose of these town halls are to discuss topics related to OTAT-regulated products, engage with product development stakeholders, and to provide information to help stakeholders to help advance drug development. The next town hall will focus on the clinical development of gene therapy products for rare diseases in February 2023.

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Delveinsight

AUGUST 5, 2021

Marinus will pursue clinical development of the therapy and move it via the regulatory process in Europe. Fibroblasts are cells, which develop connective tissue and lead processes such as wound healing. A year-old Mestag has the funds to build a pipeline of antibodies and bring its single-cell approach toward the clinic.

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Drug Discovery World

OCTOBER 16, 2023

Dr Salako says there are some clear fields of work associated with different areas, which include: Hertfordshire as a cell and gene therapy hub, with a number of companies in this space clustered around Stevenage. This includes support for export and expansion into new territories as well as for innovation and development.

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pharmaphorum

NOVEMBER 12, 2020

UCB has made a new foray into the gene therapy space, buying fellow Belgium-based company Handl to get control of its adeno-associated virus (AAV) capsid delivery platform and two research programmes in neurodegenerative diseases. The post UCB revs up its gene therapy drive with Handl acquisition appeared first on.

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The Pharma Data

OCTOBER 27, 2020

Orphan Drug designation affords Passage Bio the potential for certain benefits, including up to seven years of market exclusivity, assistance in the drug development process, tax credits for clinical development, and exemptions from certain FDA fees. About PBKR03. More information is available at www.passagebio.com.

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Drug Discovery World

FEBRUARY 22, 2023

The multi-year clinical development of Hemgenix was led by uniQure and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialise the treatment. The post EU-approved gene therapy reduced haemophilia bleeds by 64% appeared first on Drug Discovery World (DDW).

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Camargo

AUGUST 11, 2021

The FDA oversees all drug approvals in the US via New Drug Applications (NDAs), with approvals for biologic products being approved via Biologics License Applications (BLAs). Sponsors whose products qualify for the centralized procedure can also seek scientific advice from the EMA anytime during development.

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Drug Discovery World

MARCH 31, 2023

CSL’s Hemgenix (etranacogene dezaparvovec) has been authorised as the first gene therapy for adults with severe and moderately severe haemophilia B (congenital Factor IX (FIX) deficiency) in Great Britain. The post First gene therapy for haemophilia B approved in Great Britain appeared first on Drug Discovery World (DDW).

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pharmaphorum

SEPTEMBER 29, 2020

As of 2018, there were over 1,100 cancer therapies in development, and as of 2020, 362 of them were cell and gene therapies. Many novel therapies have been successfully developed through such partnerships.

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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. It may also allow for priority or rolling review of a company’s Biologics License Application (BLA).

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The Pharma Data

MAY 15, 2023

Food and Drug Administration (FDA) has accepted the company’s supplemental Biologics License Application (sBLA) for Vabysmo® (faricimab) for the treatment of macular edema following retinal vein occlusion (RVO). 1,2 Vabysmo’s safety profile was consistent with previous trials.

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The Pharma Data

JANUARY 27, 2021

Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for PBFT02, an adeno-associated virus (AAV)-delivery gene therapy that is being studied for the treatment of patients with Frontotemporal Dementia (FTD) with granulin (GRN) mutations. chief executive officer of Passage Bio. “FTD

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Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Peter Marks, head of FDA’s CBER – the organization responsible for regulating gene therapies.

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The Pharma Data

AUGUST 5, 2021

Bayer recently formed its own cell and gene therapy platform as part of its transformation strategy for Pharmaceuticals. The platform already has potentially ground-breaking medical innovations in clinical development, such as a therapy for the treatment of Parkinson’s. Pharmaceuticals raises sales and earnings.

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The Pharma Data

NOVEMBER 5, 2020

Velders is presently chairman of HollandBIO, the association of Dutch biotech companies, and provides years of experience, specifically in the cell and gene therapy areas. Taveras will lead all research and non-clinical development functions supporting the company’s pipeline of investigational therapies.

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The Pharma Data

JUNE 26, 2021

Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA), under Priority Review, for Port Delivery System with ranibizumab (PDS) for the treatment of neovascular or “wet” age-related macular degeneration (nAMD). Roche (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S.

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FDA Law Blog

SEPTEMBER 20, 2021

We have seen first-hand the difficult task faced by CBER review staff to keep up with the flood of innovative cell and gene therapy products, as well as the taxing influx of COVID-19 pandemic related applications requiring review resources in both Centers. Notable Dates and Timelines – Applications open, Q4 of FY 2023.

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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.

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Pharmaceutical Technology

AUGUST 26, 2008

” Today most big pharmaceutical companies are pursuing biotechnology either in house or by using an in-licensing strategy. The dynamic has now changed as many big companies have now acquired biotechnology companies or have developed ‘biotechnology divisions’ of their own.

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The Pharma Data

MARCH 20, 2023

2,3 These data are among a Zolgensma data set being presented during the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, which also include, in part, real-world evidence data from the RESTORE registry. 7 Severity varies across a spectrum of types corresponding to the number of copies of the back-up SMN2 gene.

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Drug Discovery World

JULY 13, 2023

The rules and regulations Unlicensed drugs are drugs that have not yet been assessed by a regulator, while off-label use is when an approved medication is prescribed outside of the terms of its license, e.g., for a different disease or in a different patient group. What are the risks?

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Drug Discovery World

MARCH 29, 2023

Peter O’Donnell, Applied Clinical Trials’ Brussels Correspondent, summarises this as the EMA being tasked with creating a European network of patient representatives, academics, drug developers, investigators, and centres with expertise in the performance of studies in the paediatric population.

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