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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FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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The Pharma Data

JANUARY 25, 2021

a biotechnology company developing cell and gene therapy treatments for patients suffering from vascular disease, today announced that it raised up to $5.5M that will lead clinical testing of a novel gene therapy for a serious vascular disease in Europe. MIAMI, Jan. 25, 2021 (GLOBE NEWSWIRE) — Ambulero, Inc. ,

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The Pharma Data

JANUARY 4, 2021

Collaboration with Biogen to develop gene therapy for an undisclosed target to treat inherited eye disease, plus option for additional target. 05, 2021 (GLOBE NEWSWIRE) — ViGeneron GmbH , a gene therapy company, today announced a global collaboration and licensing agreement with Biogen Inc. .

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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.

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The Pharma Data

DECEMBER 3, 2020

TWO: 4162) announced today that it has entered into a Collaboration and License Agreement with UK-based Sentinel Oncology Limited for advancing the new drug development of SOL-578 , a Checkpoint Kinase 1 (Chk1) inhibitor, under which PharmaEngine will fund the IND enabling studies for SOL-578. TAIPEI , Dec.

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FDA Law Blog: Drug Discovery

JANUARY 4, 2023

Brevig, Senior Regulatory Device and Biologics Expert — On December 7, 2022, FDA’s Center for Biologics Evaluation and Research (CBER) and the Office of Tissues and Advanced Therapies (OTAT) held a town hall to answer questions related to cell therapy and tissue-engineered products chemistry, manufacturing, and contr­­ols (CMC).

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The Pharma Data

NOVEMBER 1, 2020

Corlieve is focused on developing novel therapeutics for severe neurological disorders. Corlieve’s lead program employs a novel AAV gene therapy approach for the treatment of refractory temporal lobe epilepsy (TLE), the most commonly diagnosed focal epilepsy in humans. Maryland , US), a leading gene therapy company.

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The Premier Consulting Blog

NOVEMBER 20, 2024

This process can be initiated during the review period of the biologics license application (BLA) or new drug application (NDA) prior to approval. 490.100 titled, “Process Validation Requirements for Drug Products and Active Pharmaceutical Ingredients Subject to Pre-Market Approval.”

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DrugBank

OCTOBER 4, 2024

This involves assessing the strength and breadth of patents, evaluating the potential for future patent challenges, and analyzing the value derived from licensing agreements and royalty streams. The high costs associated with bringing a new drug to market, estimated to be over $2.6

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The Pharma Data

DECEMBER 29, 2020

a biotechnology company developing cell therapies that enable organ regeneration, announced today that the U.S Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application. Its drug development pipeline includes thymus, pancreas, and kidney regeneration.

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Conversations in Drug Development Trends

OCTOBER 26, 2022

That’s because psychedelic trials are logistically heavy, requiring special licensing for drug handling, training for staff in Schedule I drug requirements, and standardized facilities. Certain licensing must be obtained. Raters and therapists are not the same. More guidance on safety considerations can be found, here.

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Drug Target Review

AUGUST 9, 2024

The science behind drug formulation The formulation of a drug is both an art and a science, requiring a deep understanding of the drug’s chemical and physical properties. 1 Challenges in drug formulation Developing a successful drug formulation is fraught with challenges.

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FDA Law Blog: Drug Discovery

OCTOBER 28, 2024

This Guidance explicitly excludes gene therapies (a change from the draft guidance), but instead makes reference to a separate guidance that has been in place since 2020 titled Considerations for Long-Term Clinical Neurodevelopmental Safety Studies in Neonatal Product Development.

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Drug Target Review

NOVEMBER 22, 2023

Whilst TGF-β is an attractive target for cancer therapy because of its critical role in promoting tumor aggressiveness, immune escape and resistance to therapy, activation of alternative resistance pathways has hindered drug development attempts in the past.

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The Pharma Data

DECEMBER 22, 2020

Primary endpoints of this study include safety, tolerability, and tumor response of the ASP-1929 photoimmunotherapy in combination with anti-PD1 therapy. Outside of Japan , Illuminox therapies have not yet been approved as safe or effective by any regulatory authority. SAN MATEO, Calif. , ” About Rakuten Medical, Inc.

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The Pharma Data

JUNE 28, 2021

Broadens company’s oncology platform of Targeted Alpha Therapies / Acquisition includes actinium-225 labeled differentiated PSMA small molecule for the treatment of prostate cancer. Lisa Placanica, Senior Managing Director Center for Technology Licensing at Weill Cornell Medicine. Noria) and PSMA Therapeutics Inc.

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Crown Bioscience

MAY 4, 2021

Crown Bioscience Director Mike Batey takes a closer look at pediatric cancer and how recent regulatory changes and additional research focus may deliver new therapies for kids. Unfortunately, children have limited access to novel therapies, and new drugs for adults are often only tested in children many years following approval.

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The Pharma Data

NOVEMBER 20, 2020

NASDAQ: REGN) today announced that the antibody cocktail casirivimab and imdevimab administered together (formerly known as REGN-COV2 or REGEN-COV2), a therapy currently being investigated for use in COVID-19 , has received Emergency Use Authorization (EUA) from the U.S. Food and Drug Administration (FDA). TARRYTOWN, N.Y.,

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Conversations in Drug Development Trends

NOVEMBER 27, 2023

Food and Drug Administration (FDA) announced its acceptance of the Biologics License Application (BLA) for exa-cel. In recognition of the unmet need and medical urgency for innovative therapies in the sickle cell space, the FDA granted exa-cel Priority Review, with a formal decision expected by December 8, 2023.

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Drug Target Review

MAY 17, 2024

Lerociclib may provide MYC-driven HCC patients with a safe and more effective alternative to current therapies, which are far from curative. This underscores our strategy to focus on a specific patient population with the right drugs – a key aspect of our pipeline strategy and platform capabilities.

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Drug Target Review

APRIL 9, 2024

Initial works from the lab concentrated on exploring the role of replicative stress in cancer and ageing, for which the group combined cell biology, mouse models and drug development projects. Among other discoveries, the group developed inhibitors of the ATR kinase and showed their potential for cancer therapy.

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The Pharma Data

DECEMBER 14, 2020

Harbour BioMed (HBM) announced with Utrecht University on Monday that they have licensed their fully human SARS-CoV-2 neutralizing antibody, 47D11, and its program to AbbVie. ” Under the license agreement, the development of ABBV-47D11 will be greatly advanced. Kate Krav-Rude/Shutterstock.

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The Pharma Data

JANUARY 13, 2021

14, 2021 /PRNewswire/ — MindMed (NEO: MMED), (OTCQB: MMEDF), (DE: MMQ), a leading psychedelic medicine biotech company today announced the addition of Robert Barrow , an accomplished pharmaceutical executive, as Chief Development Officer. We are excited to attract such top tier talent from the psychedelic drug development community.

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Codon

SEPTEMBER 29, 2024

It suggested that, in the future, certain classes of drugs, specifically genetic therapies, could be screened entirely virtually via models of life. It wasn’t a silver bullet to the hard problem of drug development, but it wasn’t too far off either. A flurry of new, promising therapeutic targets arose.

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The Pharma Data

DECEMBER 9, 2020

Under its ongoing collaboration and license agreement, Crescendo’s proprietary transgenic platform and engineering expertise is being used to build Humabody ® -based therapeutics against certain targets selected by Takeda. This is the sixth technical milestone achieved by Crescendo in its collaboration with Takeda. – Ends-.

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The Pharma Data

AUGUST 21, 2020

Eli Lilly, on the heels of detailing a clinical win in China for PD-1 inhibitor Tyvyt, has decided to take a bigger stake in the drug developed by partner Innovent Biologics in an expanded deal that could vault it into the lucrative—yet ultra-competitive—U.S. Lilly first licensed Tyvyt from Innovent in 2015. lung cancer market.

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LifeSciVC

JULY 17, 2024

By Mike Cloonan, Chief Executive Officer of Sionna Therapeutics, as part of the From The Trenches feature of LifeSciVC The drug development process in rare diseases is rife with challenges especially when companies target significant differentiation or first-in-class targets.

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LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. recent announcements from Merck and Sanofi for obesity and broader metabolic disease). Join the club.

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The Pharma Data

APRIL 12, 2023

Nasdaq: DNLI) today announced that Biogen has exercised the option to license Denali’s Antibody Transport Vehicle (ATV): Amyloid beta program (ATV:Aβ). We are pleased with Biogen’s decision to license ATV: Aβ and we are hopeful this will foster the development of next-generation anti- Aβ therapeutics.”

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The Pharma Data

OCTOBER 27, 2020

Novartis has been granted an option to in-license global rights of MP0420 and MP0423 – multi-targeted direct-acting antiviral therapeutic candidates demonstrating potential efficacy against COVID-19.

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Agency IQ

MAY 31, 2024

The legislation further explained that for a certain method to qualify as a “platform technology,” it must demonstrate “reasonable likelihood to bring significant efficiencies to drug development and review processes.” based on a letter of authorization from the application holder).” which would never be acceptable.

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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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The Pharma Data

JANUARY 27, 2021

. A total of 15 patients have been recruited to date in the Phase I dose escalation part of the study, all of whom were late stage and have failed conventional treatments, including several lines of rituximab-containing therapies. About BioInvent. The Company’s validated, proprietary F.I.R.S.T

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The Pharma Data

DECEMBER 20, 2020

Food and Drug Administration (FDA) is plenty busy with COVID-19 vaccine Emergency Use Authorizations (EUAs) this month, but they’re also wrapping up the year with a few PDUFA dates for other therapies. Athenex and Almirall have a license deal with Almirall having the license to research, develop and commercialize the drug in the U.S.

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The Pharma Data

OCTOBER 18, 2020

DUBLIN–( BUSINESS WIRE )– Inflection Biosciences Ltd , a company developing innovative therapeutics for the treatment of cancer, today announced the appointment of industry veteran Gregory I. Greg possesses a wealth of experience in developing new cancer therapeutics across multiple U.S. Berk, MD, to its Board of Directors.

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The Pharma Data

JANUARY 12, 2021

Biond Biologics announced today that it has entered into a potentially $1 billion global licensing agreement with Sanofi to take a joint stab at defeating advanced forms of cancer with a more comprehensive and expedited approach to immuno-oncology. . Keitma/Shutterstock. Co-Founder and Chief Executive Officer at Biond.

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