LifeSciVC

AUGUST 21, 2024

At this point of the year, the JPMorgan conference seems like ancient history and you are looking into flights for JPM 2025. Instead, it was based on the large unmet need, the advantages of a unique ADC-based delivery platform, and a strong preclinical data package that supported this strategy. Was that only six months ago?

Clinical Trials 81

Clinical Trials Trials Disease Treatment 81

BioPharma Drive: Drug Pricing

JUNE 24, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime.

RNA 229

RNA Drugs Therapies Licensing 229

Broad Institute

JUNE 24, 2025

By Broad Communications June 24, 2025 Credit: Casey Atkins David Liu is a core member at the Broad Institute where he is the Richard Merkin Professor and director of the Merkin Institute for Transformative Technologies in Healthcare. wasn’t the first patient to receive base editing therapy.

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Treatment Disease DNA Clinical Trials 133

Agency IQ

MAY 3, 2024

Two consultations on clinical trial considerations, one on advanced therapies and the other addressing non-inferiority and equivalence comparisons, are closing at the end of May. The pharma reform package remains in E.U. leaders’ eyesight as the Parliament winds down ahead of its June elections. On April 10, the E.U.

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Regulations Clinical Trials Production Biosimilars 40

Broad Institute

JANUARY 14, 2025

By Allessandra DiCorato January 14, 2025 Credit: Maria Nemchuk, Broad Communications Eric Minikel and Sonia Vallabh run a lab with a singular focus: preventing and treating prion disease within their lifetime. Theres still a long way to go to make this a therapy, Minikel said. Online January 14, 2025. Nature Medicine.

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Disease DNA Protein Production Treatment 144

Broad Institute

MAY 27, 2025

Gene editing disrupts Huntingtons mutation in mice By Allessandra DiCorato May 27, 2025 Breadcrumb Home Gene editing disrupts Huntingtons mutation in mice Making single-letter edits in stretches of repeated DNA stopped or reversed the genetic change that causes Huntingtons disease and Friedreichs ataxia. Online May 26, 2025.

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DNA Disease Packaging Research 133

Agency IQ

JULY 8, 2024

Date What’s Happening Explanation Source June 29 GGP Report FDA expected to finalize report on Good Guidance Practices AgencyIQ June 30 User Fee Payments Industry required to confirm NDA prescription drug products in Orange Book for purposes of PDUFA payments in FY 2025 FDA July 1 Anniversary Anniversary of the signing of the Public Health Service (..)

FDA 40

FDA Science Regulations Production 40

Codon

JUNE 5, 2025

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. pyogenes protein — whose compactness makes them far easier to package into viral vectors and deliver into the human body. Researchers have also discovered a slew of small Cas9s — some less than half the size of the S.

DNA 79

DNA RNA Clinical Trials Engineering 79

Agency IQ

JULY 22, 2024

As such, it doesn’t review things like vaccines, blood products or gene therapies – those products are instead reviewed by CBER. AgencyIQ compiles these data using information in approval letters, labeling and review packages posted to the Drugs@FDA database.

Clinical Trials 52

Clinical Trials Trials Science FDA 52

The Pharma Data

JANUARY 6, 2021

End-to-End Single-Site Solution from Drug Substances to Fill-Finish & Packaging. In addition to drug substance manufacture, the facility will also provide commercial scale, automated fill-finish and assembly, packaging and labeling services. Packaging line?. Spring 2025. TOKYO , Jan.

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Packaging Small Molecule Production FDA Approval 52

Agency IQ

OCTOBER 27, 2023

WUSF / AgencyIQ November 1 Initial deadline for NDSRIs Under a 2023 guidance document, the FDA has recommended that pharmaceutical companies assess Nitrosamine Drug-Related Substance Impurities for their products by November 1, 2023, with confirmatory testing due by August 1, 2025.

FDA 40

FDA Science Regulations Production 40

Agency IQ

OCTOBER 27, 2023

11/9/2023: Enters into force 11/9/2025: Date of application To contact the authors of this resource, please email Sierra Milam ( smilam@agencyiq.com ). policy, such as public registers, drafts, reflection papers, concept papers, and guidance documents expected through Q1 2024.

Regulations 40

Regulations Pharmaceuticals Production Packaging 40

BioPharma Drive: Drug Pricing

JUNE 23, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. Influenza A virus" [Micrograph]. Retrieved from Flickr. His research, writing offer clues.

Drugs 264

Drugs Virus Therapies FDA 264

The Pharma Data

JUNE 18, 2021

The objective of this Phase 3 study is to evaluate the efficacy and safety of risankizumab 180 mg and 360 mg as maintenance therapy versus withdrawal from risankizumab treatment (control) in patients with moderate to severe Crohn’s disease who responded to risankizumab IV induction treatment in the ADVANCE and MOTIVATE studies.

Disease 40

Disease Treatment Therapies Trials 40

The Pharma Data

JUNE 18, 2021

The ADVANCE study included patients with past intolerance or inadequate response to conventional therapy (non-bio-IR) and/or biologic therapy (bio-IR). The ADVANCE study included a mixed population of patients who had responded inadequately or are intolerant to conventional and/or biologic therapy. NORTH CHICAGO, Ill. ,

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Disease Therapies Treatment Clinical Trials 40

Alta Sciences

JANUARY 21, 2025

Highlights From the 2024 American College of Toxicology 45th Annual Meeting pmjackson Tue, 01/21/2025 - 21:59 The ACT 2024 meeting took place at JW Marriott in Austin, TX My fellow Altasciences colleagues and I recently attended the 2024 annual American College of Toxicology (ACT) meeting in Austin, Texas.

Science 52

Science Therapies Packaging FDA 52

New Drug Approvals

MAY 14, 2025

Albanell-Fernndez M (January 2025). Grasselli Kmet N, Luzzati R, Monticelli J, Babich S, Conti J, Bella SD (March 2025). “Salvage therapy of complicated Candida albicans spondylodiscitis with Rezafungin” European Journal of Clinical Microbiology & Infectious Diseases. New Drug Therapy Approvals 2023 (PDF).

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Pharmacokinetics FDA FDA Approval Treatment 62

New Drug Approvals

APRIL 27, 2025

This article incorporates text from this source, which is in the public domain. ^ “Drug Approval Package: Flyrcado Injection” U.S. Retrieved 21 January 2025. ^ “Novel Drug Approvals for 2024” U.S. New Drug Therapy Approvals 2024 (PDF). January 2025. Archived from the original on 21 January 2025.

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FDA Clinical Trials FDA Approval Disease 57

LifeSciVC

MAY 28, 2025

As illustrated in LEKs May 2025 report Is Biopharma Doing Enough to Advance Novel Targets ? Think about how many CART CD-19 companies got started or gene editing and gene therapy plays literally, dozens and dozens of them emerged, all chasing similar indications. the scale of the current crowding challenge is staggering.

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Production Marketing DNA Drugs 80

The Open Targets Blog

MARCH 19, 2025

All of our data is up to date as of Q1 2025. The pipelines underpinning our genetic analyses have been completely rewritten as a Python package for post-GWAS analysis: Gentropy. Studies with and without summary statistics are processed and flagged accordingly. Colocalisation is now based on credible set overlaps.

Pharmacogenetics 65

Pharmacogenetics Disease Clinical Trials Trials 65

Drug Target Review

MAY 8, 2025

This combination of single-dose treatment with allele selectivity represents a unique approach among investigational HD therapies in development. This fundamentally different approach combining the durability of a single-dose therapy with the precision of allele-selective editing addresses a critical unmet need in HD treatment.

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Disease Treatment DNA Therapies 111

SugarCone Biotech

APRIL 8, 2025

Astra Zenecas purchase of Esobiotec with a $425M upfront has rekindled interest in CAR-T cell therapy technologies, and in particular, the in vivo cell therapy companies. In vivo CAR-T cell therapy requires that genetic material be delivered to the desired immune cells while those cells are still inside the patient.

Therapies 56

Therapies RNA Packaging Virus 56

Codon

JANUARY 2, 2025

That DNA was then shipped to France, where Imagene , a company specializing in robust and room-temperature storage solutions, packaged the molecules into laser-sealed, stainless steel capsules. Xander Balwit & Niko McCarty The Editors Join Asimov Press in 2025!

DNA 132

DNA Laboratories Packaging Vaccine 132

BioPharma Drive: Drug Pricing

JUNE 23, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. You can unsubscribe at anytime. Track funding rounds here.

FDA 153

FDA Drugs Therapies Licensing 153

Codon

FEBRUARY 9, 2025

To achieve this, E11 Bio synthesizes protein barcodes (short peptides with unique amino acid sequences), packages them into AAV vectors (engineered, non-harmful viruses for drug delivery), and delivers these into the brain tissue. ” Asimov Press (2025). ” Xander Balwit is editor-in-chief of Asimov Press. Cite: Balwit X.

Research 114

Research Disease Engineering Regulations 114

BioPharma Drive: Drug Pricing

JUNE 12, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. PBMs would no longer be able to pocket the difference as profit if the bill passes.

Small Molecule 130

Small Molecule Packaging Therapies Pharmacy 130

Codon

FEBRUARY 23, 2025

After decades of research into therapies and preventive measures, though, global AIDS deaths have now fallen to about 600,000 annually. In January 2025, the incoming Trump administration issued a 90-day freeze on PEPFAR while it conducts a “programmatic efficiencies” review. ” Asimov Press (2025).

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Clinical Trials Virus Vaccine DNA 77