Drugs and Therapies - Drug Discovery Digest

Sarepta scraps a Duchenne drug as gene therapy sales rise

BioPharma Drive: Drug Pricing

NOVEMBER 7, 2024

Elevidys sales have increased since the FDA made a controversial choice to expand the therapy's use. Now, Sarepta is abandoning a successor to its drug Exondys 51, citing an “evolving" treatment landscape.

Therapies

Therapies Drugs FDA Treatment

Combo Therapy May Be Advance Against Liver Cancer

Drugs.com

MAY 6, 2024

MONDAY, May 6, 2024 -- A new combination therapy appears to boost the response rate for liver cancer patients receiving immunotherapy, according to results from a phase 2 clinical trial.The combo therapy involves bavituximab, a drug that.

Therapies

Therapies Drugs

FDA Approves Landmark Sickle Cell Gene Therapies, Casgevy and Lyfgenia

Drugs.com

DECEMBER 8, 2023

Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology. FRIDAY, Dec. 8, 2023 -- The U.S. Casgevy, developed by Vertex.

FDA Approval

FDA Approval Therapies FDA Treatment

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Pfizer hemophilia gene therapy arrives in US to uncertain future

BioPharma Drive: Drug Pricing

APRIL 26, 2024

The Food and Drug Administration approval of Beqvez comes as other gene therapies for the bleeding condition that were approved earlier struggle to gain traction.

Therapies

Therapies Drugs

Secondary Tumors After CAR-T Cancer Therapies Are Rare: Study

Drugs.com

JUNE 13, 2024

THURSDAY, June 13, 2024 -- CAR-T cell therapy to treat blood cancers is safer than previously thought, with little risk that the immunotherapy will create secondary cancers, a new study finds.The U.S. Food and Drug Administration issued a warning.

Therapies

Therapies Drugs

FDA Warns of Rare Secondary Cancer Risk With CAR-T Therapies

Drugs.com

JANUARY 24, 2024

Food and Drug Administration has told drugmakers to add a boxed warning to a type of cancer treatment called CAR-T therapy, saying the treatment itself may sometimes cause a secondary. WEDNESDAY, Jan. 24, 2024 (Healthday News) -- The U.S.

Therapies

Therapies FDA Treatment Drugs

Synthesis and Investigation of Peptide–Drug Conjugates Comprising Camptothecin and a Human Protein‐Derived Cell‐Penetrating Peptide

Chemical Biology and Drug Design

JANUARY 20, 2025

ABSTRACT Drug targeting strategies, such as peptidedrug conjugates (PDCs), have arisen to combat the issue of off-target toxicity that is commonly associated with chemotherapeutic small molecule drugs. The PDCs crossed membranes and cleavable PDCs killed melanoma cells with nanomolar potency.

Small Molecule

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FDA Approves First Gene Therapy, Elevidys, to Treat Duchenne Muscular Dystrophy

Drugs.com

JUNE 23, 2023

Food and Drug Administration on Thursday approved the drug Elevidys, the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD). FRIDAY, June 23, 2023 -- The U.S. The groundbreaking treatment will not be cheap:

FDA Approval

FDA Approval Therapies FDA Treatment

Retinal therapy may restore lost vision

Science Daily: Pharmacology News

APRIL 2, 2025

Researchers have successfully developed a novel drug to restore vision. The treatment method restores vision through retinal nerve regeneration.

Therapies

Therapies Treatment Research Drugs

Precision sells lymphoma drug to Imugene in retreat from cell therapy research

BioPharma Drive: Drug Pricing

AUGUST 16, 2023

The struggling biotech also plans to offload other cell therapy assets, including a multiple myeloma therapy, and focus exclusively on gene editing medicines.

Therapies

Therapies Research Drugs

FDA Approves Landmark Sickle Cell Gene Therapies

Drugs.com

DECEMBER 8, 2023

Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology.Casgevy, developed by Vertex. FRIDAY, Dec. 8, 2023 -- The U.S.

FDA Approval

FDA Approval Therapies FDA Treatment

FDA sets advisory meeting date for Brainstorm’s ALS cell therapy

BioPharma Drive: Drug Pricing

JUNE 6, 2023

Having taken the rare step of filing for approval over protest, Brainstorm will get another chance to make a case for its drug NurOwn during a Sept. 27 meeting of cell, tissue and gene therapy experts.

Therapies

Therapies FDA Drugs

FDA Approves First Gene Therapy to Treat Duchenne Muscular Dystrophy

Drugs.com

JUNE 23, 2023

Food and Drug Administration on Thursday approved the drug Elevidys, the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD). FRIDAY, June 23, 2023 -- The U.S. The groundbreaking treatment will not be cheap:

FDA Approval

FDA Approval Therapies FDA Treatment

FDA Advisors Say New Gene Therapy for Sickle Cell Disease, Exa-cel, is Safe

Drugs.com

NOVEMBER 2, 2023

1, 2023 -- A new gene therapy for sickle cell disease was deemed safe by a U.S. Food and Drug Administration advisory panel on Tuesday, paving the way for full approval by early December. WEDNESDAY, Nov. The FDA had already decided that the.

Therapies

Therapies FDA Disease Drugs

Royalty Pharma buys into Ferring’s cancer treatment in first gene therapy deal

BioPharma Drive: Drug Pricing

AUGUST 24, 2023

The deal, worth up to $500 million, is a bet by the drug rights dealmaker on the uncertain commercial prospects of Ferring's gene therapy Adstiladrin.

Therapies

Therapies Treatment Drugs

Combo Therapy Boosts Survival for Advanced Colon Cancer

Drugs.com

MAY 29, 2024

combination of two experimental immunotherapy drugs plus standard chemotherapy led to a median. WEDNESDAY, May 29, 2024 -- People battling advanced colon cancers might have a new treatment option that could extend their survival, a new trial finds.A

Therapies

Therapies Trials Treatment Drugs

CG follows big IPO with new results for bladder cancer drug

BioPharma Drive: Drug Pricing

MAY 3, 2024

Newly disclosed Phase 3 results presented Friday match findings the biotech disclosed last year, while offering a more comprehensive look at how its drug stacks up to rival therapies.

Drugs

Drugs Therapies

Scientists develop grain-sized soft robots controlled by magnetic fields for targeted drug delivery

Science Daily: Pharmacology News

OCTOBER 24, 2024

A team of scientists has developed grain-sized soft robots that can be controlled using magnetic fields for targeted drug delivery, paving the way to possible improved therapies in future.

Therapies

Therapies Drugs

FDA Says No to MDMA as Therapy for PTSD

Drugs.com

AUGUST 12, 2024

12, 2024 -- Advocates for the use of the psychedelic drug MDMA (midomafetamine) to treat mental health conditions were delivered a setback on Friday after the U.S. Food and Drug Administration declined to approve it as treatment for. MONDAY, Aug.

Therapies

Therapies FDA Treatment Drugs

Judo Bio debuts with $100M and a plan to take RNA drugs to the kidney

BioPharma Drive: Drug Pricing

OCTOBER 7, 2024

The Cambridge, Massachusetts-based biotech is using what it calls ligand-siRNA conjugates to reach drug targets in the kidney, which historically have been difficult to reach with RNA therapies.

RNA

RNA Therapies Drugs

Statin therapy may prevent cancer by blocking inflammatory protein

Science Daily: Pharmacology News

MAY 30, 2024

Scientists have found that statins -- commonly used cholesterol-lowering drugs -- may block a pathway that leads to the development of cancer in the context of chronic inflammation.

Therapies

Therapies Drugs

New cell therapy model accelerates cancer treatment development

Drug Target Review

FEBRUARY 10, 2025

In the rapidly advancing field of cell therapies, Dr Jason Bock has emerged as a leader, known for his innovative approach to optimising the development process. With over 25 years of experience in therapeutics, Bock has played a pivotal role in shaping the future of cell therapies, particularly through his work at CTMC.

Therapies

Therapies Treatment Engineering Clinical Trials

Bayer partners with RNA drugmaker to develop new cancer therapies

BioPharma Drive: Drug Pricing

AUGUST 28, 2024

Under a collaboration with NextRNA Therapeutics, Bayer will access the biotech’s platform to target long, non-coding RNA interactions with small molecule drugs.

RNA

RNA Small Molecule Therapies Drugs

IGM’s autoimmune pivot backfires as top drug disappoints in testing

BioPharma Drive: Drug Pricing

JANUARY 9, 2025

The biotech will stop developing two drugs and lay off nearly three-quarters of its staff after its lead therapy wasn’t as potent in early testing as the company expected.

Drugs

Drugs Therapies

Cancer cell therapy from Arcellx, Gilead shows promise in early data

BioPharma Drive: Drug Pricing

NOVEMBER 5, 2024

The companies’ CAR-T seemed to cause fewer movement disorders in a multiple myeloma trial than J&J's Carvykti has in other tests, although Wall Street analysts were divided on how the two drugs compare.

Therapies

Therapies Trials Drugs

FDA Approves First Gene Therapy for Severe Hemophilia A, Roctavian

Drugs.com

JUNE 30, 2023

Food and Drug Administration on Thursday approved a costly single-dose gene therapy for patients with severe hemophilia A. FRIDAY, June 30, 2023 -- The U.S. The treatment, Roctavian (valoctocogene roxaparvovec), will cost $2.9 million for a single.

FDA Approval

FDA Approval Therapies FDA Treatment

CRISPR therapy from Intellia may ameliorate rare heart disorder, data suggest

BioPharma Drive: Drug Pricing

NOVEMBER 18, 2024

But rival drugs are further ahead. Phase 1 data indicate Intellia’s medicine could be a powerful treatment for a cardiac form of ATTR amyloidosis.

Therapies

Therapies Treatment Drugs

ALS drug from Denali, Sanofi falls short in mid-stage study

BioPharma Drive: Drug Pricing

FEBRUARY 16, 2024

The disclosure the experimental therapy missed its trial goal comes after Sanofi had earmarked the drug for advancement into Phase 3.

Drugs

Drugs Therapies Trials

Atalanta banks $97M to send RNAi drugs into the brain

BioPharma Drive: Drug Pricing

JANUARY 28, 2025

The startup says its specially constructed RNAi therapies can get deep into brain tissue, yielding new ways to treat neurological conditions like Huntington’s.

Therapies

Therapies Drugs

Lilly gene therapy finding puts focus on hearing loss treatment pipeline

BioPharma Drive: Drug Pricing

JANUARY 24, 2024

are taking aim at the same drug target. An 11-year-old boy born deaf can hear after receiving Lilly's genetic medicine. Others, including biotechs in China and Regeneron in the U.S.,

Therapies

Therapies Treatment Drugs

Gene therapy startup emerges with green light for first-of-its-kind trial

BioPharma Drive: Drug Pricing

SEPTEMBER 12, 2024

Spun out of research at Nationwide Children’s Hospital, Vironexis claims it will be the first company to start a study testing a cancer drug delivered via the adeno-associated viruses commonly used in genetic medicines.

Therapies

Therapies Virus Trials Hospitals

Gilead to lay off staff at cell therapy unit Kite

BioPharma Drive: Drug Pricing

NOVEMBER 29, 2023

The cuts will impact 7% of the cancer drug division’s employees, although about 90 new roles will be created around “areas of growth.”

Therapies

Therapies Drugs

Ajax, aiming for a better JAK drug, raises $95M to begin first tests

BioPharma Drive: Drug Pricing

MAY 13, 2024

Goldman Sachs and Eli Lilly are among those backing the startup, which claims its prospect could be more potent than the myelofibrosis therapies that have come to market in recent years.

Therapies

Therapies Marketing Drugs

European regulators want to know more about the risks of GLP-1 drugs

BioPharma Drive: Drug Pricing

DECEMBER 1, 2023

The EMA's safety committee has more questions for makers of the in-demand therapies as it reviews whether the drugs are linked to the risk of suicidal thoughts.

Regulations

Regulations Drugs Therapies

Logistics in Focus: Orchestrating the Challenges of Cell Therapy

Conversations in Drug Development Trends

NOVEMBER 12, 2024

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. Below, we discuss some of these challenges in cell therapy trials.

Therapies

Therapies Clinical Supply Clinical Trials Trials

Sarepta sells FDA fast pass earned from Duchenne gene therapy approval

BioPharma Drive: Drug Pricing

JULY 5, 2023

The deal enables Sarepta to quickly cash in on the clearance of Elevidys, with $102 million in new funds to help bankroll its drug research.

Therapies

Therapies FDA Drug Research Research

Scholar Rock scores with ‘surprise’ success in SMA drug study

BioPharma Drive: Drug Pricing

OCTOBER 7, 2024

Phase 3 findings could position the biotech’s therapy to become part of a “new standard” for SMA, its CEO said, while boosting research into whether it can preserve muscle in people with obesity.

Therapies

Therapies Drugs Research

Bayer says menopause drug succeeds in breast cancer study

BioPharma Drive: Drug Pricing

JANUARY 9, 2025

The results suggest Bayer’s elinzanetant may help a broader group of women with vasomotor symptoms, which are associated with menopause as well as a common breast cancer therapy.

Therapies

Therapies Drugs

BioMarin drops drug programs in pipeline cull

BioPharma Drive: Drug Pricing

APRIL 25, 2024

The cuts of four pipeline prospects come as BioMarin resets its priorities following the slow launch of its hemophilia gene therapy Roctavian.

Therapies

Therapies Drugs

Scientists discover new T cells and genes related to immune disorders

Science Daily: Pharmacology News

JULY 4, 2024

The new T cell atlas is publicly available and should help in the development of new drug therapies for immune-mediated diseases. The discoveries were made possible by a newly developed technology they call ReapTEC.

Therapies

Therapies Disease Research Drugs

5 FDA decisions to watch in the third quarter

BioPharma Drive: Drug Pricing

JULY 3, 2024

Multibillion-dollar buyouts from Bristol Myers Squibb and Gilead could yield new drugs for brain and liver diseases, while a new cell therapy may reach market.

FDA

FDA Therapies Disease Marketing

New Experimental Drug, Vorasidenib, Could Be Advance Against Glioma Brain Tumors

Drugs.com

JUNE 7, 2023

MONDAY, June 5, 2023 -- An experimental targeted therapy can dramatically slow the progress of common slow-growing brain cancers, a new clinical trial finds. The oral drug vorasidenib nearly tripled progression-free survival in patients with grade.

Clinical Trials

Clinical Trials Drugs Therapies Trials

Redefining Acceleration of the Drug Development Journey

PPD

NOVEMBER 11, 2024

It is hard to ignore some of the most pressing, long-term trends driving the push to accelerate innovation and progress in drug development. As a result, biopharma and biotech companies working to bring their drug pipelines to the market require deep expertise from trusted partners to help deliver critical therapies for their patients.

Drug Development

Drug Development Clinical Trials Drugs Small Molecule

FDA Announces Plan to Phase Out Some Animal Testing

Drugs.com

APRIL 14, 2025

Food and Drug Administration announced it is shifting away from animal testing in the development of monoclonal antibody therapies and other drugs, favoring more effective, human-relevant methods.The new strategy. MONDAY, April 14, 2025 -- The U.S.

Animal Testing

Animal Testing FDA Therapies Drugs

Sarepta scraps a Duchenne drug as gene therapy sales rise

Combo Therapy May Be Advance Against Liver Cancer

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FDA Approves Landmark Sickle Cell Gene Therapies, Casgevy and Lyfgenia

Webinars

Pfizer hemophilia gene therapy arrives in US to uncertain future

Secondary Tumors After CAR-T Cancer Therapies Are Rare: Study

FDA Warns of Rare Secondary Cancer Risk With CAR-T Therapies

Synthesis and Investigation of Peptide–Drug Conjugates Comprising Camptothecin and a Human Protein‐Derived Cell‐Penetrating Peptide

FDA Approves First Gene Therapy, Elevidys, to Treat Duchenne Muscular Dystrophy

Retinal therapy may restore lost vision

Precision sells lymphoma drug to Imugene in retreat from cell therapy research

FDA Approves Landmark Sickle Cell Gene Therapies

FDA sets advisory meeting date for Brainstorm’s ALS cell therapy

FDA Approves First Gene Therapy to Treat Duchenne Muscular Dystrophy

FDA Advisors Say New Gene Therapy for Sickle Cell Disease, Exa-cel, is Safe

Royalty Pharma buys into Ferring’s cancer treatment in first gene therapy deal

Combo Therapy Boosts Survival for Advanced Colon Cancer

CG follows big IPO with new results for bladder cancer drug

Scientists develop grain-sized soft robots controlled by magnetic fields for targeted drug delivery

FDA Says No to MDMA as Therapy for PTSD

Judo Bio debuts with $100M and a plan to take RNA drugs to the kidney

Statin therapy may prevent cancer by blocking inflammatory protein

New cell therapy model accelerates cancer treatment development

Bayer partners with RNA drugmaker to develop new cancer therapies

IGM’s autoimmune pivot backfires as top drug disappoints in testing

Cancer cell therapy from Arcellx, Gilead shows promise in early data

FDA Approves First Gene Therapy for Severe Hemophilia A, Roctavian

CRISPR therapy from Intellia may ameliorate rare heart disorder, data suggest

ALS drug from Denali, Sanofi falls short in mid-stage study

Atalanta banks $97M to send RNAi drugs into the brain

Lilly gene therapy finding puts focus on hearing loss treatment pipeline

Gene therapy startup emerges with green light for first-of-its-kind trial

Gilead to lay off staff at cell therapy unit Kite

Ajax, aiming for a better JAK drug, raises $95M to begin first tests

European regulators want to know more about the risks of GLP-1 drugs

Logistics in Focus: Orchestrating the Challenges of Cell Therapy

Sarepta sells FDA fast pass earned from Duchenne gene therapy approval

Scholar Rock scores with ‘surprise’ success in SMA drug study

Bayer says menopause drug succeeds in breast cancer study

BioMarin drops drug programs in pipeline cull

Scientists discover new T cells and genes related to immune disorders

5 FDA decisions to watch in the third quarter

New Experimental Drug, Vorasidenib, Could Be Advance Against Glioma Brain Tumors

Redefining Acceleration of the Drug Development Journey

FDA Announces Plan to Phase Out Some Animal Testing

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