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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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 29, 2021

US drugmaker Eli Lilly – still waiting for an FDA decision on one non-opioid pain drug – has just added another to its pipeline via a licensing agreement with Japan’s Asahi Kasei worth up to $410 million. Other candidates include a PACAP38 antibody, SSTR4 agonist and TRPA1 antagonist, all in phase 1 development.

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pharmaphorum

JULY 5, 2022

The new partnership will apply Mogrify’s cellular reprogramming and bioinformatics expertise to try to identify transcription factors – proteins which can turn specific genes on or off – that can be used to generate new cochlear hair cells. The post Astellas teams up with UK biotech Mogrify on hearing loss project appeared first on.

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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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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. It enables the companies to work closely together, throughout the continuum of research, development and commercialisation.

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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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The Pharma Data

NOVEMBER 4, 2020

Ltd (“Juyou”), a biotechnology company that develops and sells medical and cosmetic skincare products, for the commercialization and development of Pliaglis ® in mainland China (the “License Agreement”). million and US$1.8 For additional information, please visit [link].

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pharmaphorum

OCTOBER 21, 2022

Swiss pharma giant Roche has moved to strengthen its position in the KRAS-mutated cancer space through a $25 million licensing and collaboration agreement with Austrian arenaviral technology firm Hookipa Pharma. Roche also has the option to license a second, undisclosed, novel arenaviral immunotherapy.

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The Pharma Data

NOVEMBER 30, 2020

1, 2020 /PRNewswire/ — Sosei Group Corporation (“the Company”) (TSE: 4565) announces it has entered into a global collaboration and license agreement with Biohaven Pharmaceutical Holding Company Ltd. (“Biohaven”, NYSE: BHVN). ” Abbreviations used: CGRP – calcitonin gene-related peptide.

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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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The Pharma Data

MARCH 16, 2021

The potential of centrally acting COMT inhibitors has not yet been evaluated in the clinic due to the unavailability of such compounds. In partnering with the Lieber Institute, Boehringer Ingelheim has in-licensed preclinical pipeline candidates, representing a first-in-class approach for targets in neuropsychiatric disorders.

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The Pharma Data

MARCH 11, 2021

Preclinical studies are ongoing and will continue over the next several months to evaluate whether MRT5500, as well as additional mRNA vaccine candidates, will induce neutralizing antibodies against the emerging SARS-CoV-2 variants, with the potential to inform current and future clinical development. About Translate Bio.

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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

JANUARY 3, 2021

The Phase 1/2 clinical trial will primarily investigate the safety and tolerability of INZ-701 and characterize its pharmacokinetic and pharmacodynamic profile, including plasma pyrophosphate (PPi) and other biomarker levels, to establish a recommended dosing regimen for further clinical development. Demetrios Braddock, M.D.,

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Delveinsight

MARCH 2, 2021

To sum it, Chinook will manage clinical development and commercialization of product candidates while Evotec will be eligible for an undisclosed upfront payment, research funding, progress-dependent milestone payments, and tiered royalties on net sales for targets identified through the collaboration.

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The Pharma Data

JANUARY 13, 2021

Fast Track designation facilitates the development and expedites the review of drugs that are intended to treat serious and life-threatening conditions and show the potential to fill an unmet medical need. Brilacidin for UP/UPS was licensed to Alfasigma S.p.A. More information is available on the Company website at www.IPharmInc.com.

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Pfizer

SEPTEMBER 28, 2022

Emergency uses of the original and bivalent vaccines have not been approved or licensed by FDA, but have been authorized by FDA, under an Emergency Use Authorization (EUA) to prevent Coronavirus Disease 2019 (COVID 19) in: . • individuals 6 months of age and older (original vaccine). • individuals 12 years of age and older (bivalent vaccine)

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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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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

OCTOBER 14, 2020

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing small molecules and biologics to treat and prevent human disease and alleviate suffering. 1 Noyce RS, et al. 2018) PLoS One. 13(1):e0188453. About Tonix Pharmaceuticals Holding Corp.

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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. Group sales in the second quarter increased by 12.9

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Pharmaceutical Technology

JUNE 13, 2023

The US Food and Drug Administration (FDA) approved Ferring Pharmaceuticals’ Prior-Approval Supplement (PAS) to the Biologics License Application (BLA) for the bladder cancer gene-therapy Adstiladrin (nadofaragene firadenovec). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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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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

NOVEMBER 11, 2020

Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives approval of a biologics license application (BLA) for a rare pediatric disease product application may be eligible for a voucher which can be redeemed to obtain priority review for a subsequent marketing application for a different product.

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XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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The Pharma Data

SEPTEMBER 20, 2020

Amgen’s clinical development team and clinical investigators, will discuss Phase 1 data being presented on the Company’s investigational KRAS G12C ?inhibitor Even when clinical trials are successful, regulatory authorities may question the sufficiency for approval of the trial endpoints we have selected.

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The Pharma Data

OCTOBER 28, 2020

Five patients between the ages of 5 and 15 with deletions in the maternal UBE3A gene region were enrolled in the first three cohorts and are included in the interim data analysis. Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. About Angelman Syndrome.

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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

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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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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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 delivery vector – an AAV8 capsid – is designed to allow gene expression directly in target tissues like skeletal muscle.

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Pharmaceutical Technology

NOVEMBER 21, 2022

Manufacturing woes have affected both cell and gene-modified cell therapies in oncology recently, disadvantaging patients with multiple myeloma (MM) and melanoma.

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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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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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Pharmaceutical Technology

MAY 26, 2023

Tripling its production capacity, Touchlight can now manufacture 8kg plasmid DNA, a key component for mRNA gene therapies and vaccines. As per the company, dbDNA is suitable for rapid, scalable manufacture of GMP DNA and can incorporate gene sequences of sizes ranging from 500bp to 20kb, which are typically unstable as pDNA.

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