Drug Target Review

JUNE 9, 2025

One company leading the charge in this revolution is Alltrna , whose pioneering work in engineered transfer RNA (tRNA) therapeutics is offering new hope for patients with rare genetic diseases.

Disease

Drug Target Review

JUNE 30, 2025

“I was increasingly drawn to understanding the underlying biology of disease at a molecular level – the ‘why’ – and how we might harness science to create better medicines for patients,” Lepore says. From theory to therapy This approach is already delivering promising insights across key therapeutic areas. .

Disease

Alta Sciences

JULY 9, 2025

Altasciences at ASGCT 2025: An Open Forum for All Things Gene and Cell Therapy | By Kaylyn Koenig aasimakopoulos Thu, 07/10/2025 - 08:00 I recently returned from attending the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.

Therapies

Drug Target Review

JULY 11, 2025

From maths and physics to molecular biology Schnall-Levin did not begin in the life sciences. “I I took a couple of other software startup opportunities,” he adds, “but I realised I missed the genomics and science. I did physics and math as an undergraduate,” he explains. I started looking around and getting interested in biology.

Molecular Biology

The Pharma Data

JUNE 18, 2025

Pushkal Garg to Lead Unified Research and Development Organization, Signaling Strategic Expansion into Next Phase of RNAi Therapeutics Innovation Alnylam Pharmaceuticals , a global leader in RNA interference (RNAi) therapeutics, has announced the promotion of Pushkal Garg, M.D.,

RNA

Pharma Manufacturing

JUNE 17, 2025

According to the results of CRB’s Horizons: Life Sciences survey released in October 2024, 75% of respondents are using or plan to use continuous technologies almost exclusively within the next five years, based on a survey of 500 industry leaders including therapeutic developers and CDMO executives.

RNA

LifeSciVC

JUNE 12, 2025

Founding Vision: Harnessing RNA Editing for Transformative Medicine Korro Bio’s story began in 2018, co-founded by a remarkable group: Jean-François Formela, M.D. Academic specializing in RNA editing). Their collective expertise—spanning gene editing, venture creation, and foundational science—set the stage for building Korro.

RNA

Drug Target Review

JUNE 16, 2025

7 In immune therapies, MSI helps monitor immune-cell distribution and off-target effects, optimising therapeutic strategies and improving patient outcomes. Stefan has held pivotal roles at leading life science companies including Roche and Siemens Healthineers.

Drugs

Elrig

MARCH 25, 2025

This second ELRIG meeting on Therapeutic Oligonucleotides brings together esteemed scientists from academia, industry, and other members of the drug discovery community to explore the discovery, validation, and targeting of oligonucleotide-based drug candidates, including antisense oligonucleotides (ASOs) and small interfering RNA (siRNA).

RNA

Drug Target Review

DECEMBER 16, 2024

Since their first approvals in 2017, autologous CAR T-cell therapies have revolutionised the treatment of blood cancers, offering hope where previous options had failed. Consequently, these therapies come with significant burdens and are often underutilised despite their transformative patient outcomes.

Therapies

LifeSciVC

JULY 8, 2025

Over the past two decades, life sciences venture capital has developed a substantial body of data, pattern recognition, and tacit knowledge around what works in early-stage biotech. The structures that support them, how we align, coordinate, and decide, may need to evolve as much as the science they advance.

Hospitals

The Pharma Data

JUNE 9, 2025

Alnylam Secures European Commission Approval for AMVUTTRA® to Treat Cardiomyopathy in ATTR Amyloidosis Alnylam Pharmaceuticals , a global leader in RNA interference (RNAi) therapeutics, has received European Commission (EC) approval for an expanded indication of its RNAi drug AMVUTTRA® (vutrisiran).

Treatment

Codon

JUNE 5, 2025

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. The core of the CRISPR immune response is a guide RNA (gRNA) that binds to a CRISPR-associated (Cas) protein. Collectively, these repeat-protospacer regions are known as CRISPR arrays. Visit our website to get a copy.

DNA

Codon

APRIL 14, 2025

Some, like predicting microbial growth rate from genetics , predicting CRISPR editing efficiency at a locus given a guide RNA , and protein stability prediction are in progress but would benefit from more support, especially to make solutions widely applicable and available to potential users.

Protein Expression

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. Nolan Townsend said will help “realize the therapeutic and economic value of promising preclinical science.”

RNA

BioPharma Drive: Drug Pricing

JULY 16, 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. The Food and Drug Administration’s clearance was controversial.

Therapies

Codon

NOVEMBER 1, 2024

Scientists are already building a model that can, for example, look at which RNA molecules are expressed in a cell at t=0 and predict how those molecules will change at t=1. There are more than 500,000 life sciences researchers in the U.S., Live-seq similarly enables scientists to study RNA expression, without killing cells, over time.

DNA

BioPharma Drive: Drug Pricing

JULY 10, 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. This panel, known as ACIP for short, recommends who should receive FDA-approved vaccines.

Vaccine

Broad Institute

JUNE 25, 2025

The scientists suggest that new therapies that target these genes could directly address fibrosis and potentially be more effective for this complication than existing drugs, which are primarily focused on reducing inflammation. But now we can potentially think about additive therapies that more directly target the fibrotic process.”

Disease

Codon

JULY 21, 2025

The alchemy of van Helmont’s day was not a mature science. At some point between the 17th and 18th centuries, the study of matter and substance went from being an immature science, a bunch of people hanging around and wearing funny hats, to a mature one, a bunch of scientists hanging around and wearing funny hats.

Science

Science Daily: Pharmacology News

APRIL 9, 2025

The system can be formulated completely as RNA, dramatically simplifying delivery logistics compared to traditional systems that use both RNA and DNA.

RNA

Science Daily: Pharmacology News

MAY 29, 2024

Functional analysis based on these structures also revealed how a 'prime editor' could achieve reverse transcription, synthesizing DNA from RNA, without 'cutting' both strands of the double helix. Clarifying these molecular mechanisms contributes greatly to designing gene-editing tools accurate enough for gene therapy treatments.

RNA

Science Daily: Pharmacology News

JULY 5, 2023

An antisense therapy restores production of the protein FMRP in cell samples taken from patients with fragile X syndrome.

Protein Production

BioPharma Drive: Drug Pricing

JUNE 3, 2024

With new modalities such as gene and cell therapies, RNA therapeutics, complex biologics and more, today’s science brings unprecedented opportunities to address diseases that have long remained out of reach.

Science

New Drug Approvals

JULY 18, 2025

It acts as an RNA-dependent RNA polymerase (RdRp) inhibitor, the same mechanism as the previously approved molnupiravir and remdesivir. It acts as an RNA-dependent RNA polymerase (RdRp) inhibitor, the same mechanism as the previously approved molnupiravir and remdesivir. 105 : 150–158. doi : 10.1016/j.ejps.2017.05.009.

RNA

Science Daily: Pharmacology News

JULY 27, 2023

If translated into the clinic, this approach could expand access and reduce the cost of gene therapies for blood disorders, many of which currently require patients receive chemotherapy and a stem cell transplant.

RNA

Drug Target Review

JUNE 15, 2023

In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop small molecule therapeutics to target RNA processing. The field is faced with a number of obstacles that require specifically assay technologies to surmount.

RNA

Broad Institute

MAY 16, 2024

By Allessandra DiCorato May 16, 2024 Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor.

Therapies

Broad Institute

JANUARY 8, 2024

Now the researchers describe how they re-engineered both eVLPs and parts of the prime editing protein and RNA machinery to boost editing efficiency up to 170 times in human cells compared to the previous eVLPs that deliver base editors. DOI: 10.1038/s41587-023-02078-y Tags: Liu Lab Gene editing Rare Disease Gene-based therapy delivery

Engineering

Broad Institute

OCTOBER 23, 2024

By Leah Eisenstadt, Broad Communications October 23, 2024 Credit: Courtesy of the Broadbent family Brian and Julia Broadbent are raising their daughters Claire, top left, and Emma, seated, who is the first person to be diagnosed with a rare genetic disorder caused by the long noncoding RNA CHASERR.

DNA

Broad Institute

AUGUST 19, 2024

I’m so proud of this team and the partnership we formed to follow the science and set up some major positive impacts on women’s health,” Blainey said. Using RNA sequencing and working with the Broad’s Metabolomics Platform and collaborators at St. Paper cited Zhu M et al.

Hospitals

The Pharma Data

SEPTEMBER 28, 2020

This included compiling RNA data, as well as other data points such as patient demographics, clinical treatment arm and patient diagnosis. However, for others, it did not work – or did so only marginally, or with serious side effects,” said Stuart Henderson, global Life Sciences lead for Accenture.

Science

The Pharma Data

MARCH 9, 2022

Novartis today announced a license option agreement with Voyager Therapeutics, a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies, for three capsids to use in potential gene therapies for neurological diseases, with options to access capsids for two other targets. About Novartis.

Licensing

Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

Small Molecule

KIF1A

JULY 29, 2023

KIF1A in New York: The 2023 KAND Family & Scientific Engagement Conference We won’t be posting next Saturday, but not for a lack of science! Genetic and RNA-based therapies seek to correct mutant KIF1A – improving its force generation, velocity, and distance traveled by increasing the ratio of healthy to mutant KIF1A.

RNA

PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. But even if gene therapy could reach sufficient muscle cells to improve quality of life, another hurdle looms: the dystrophin gene that’s deleted in DMD is huge, the largest in a human genome at 2.2

FDA Approval

Alta Sciences

MARCH 1, 2024

Norbert Makori and Pierre Jolicoeur: The Benefits of Combining Preclinical and Bioanalytical for Your Gene Therapy Studies pmjackson Fri, 03/01/2024 - 22:20 Recent advances in gene therapy have allowed for diseases to be approached differently. q: How can Altasciences support sponsors’ Gene Therapy Programs?

Therapies