Therapies - Drug Discovery Digest

Gene therapy faces fresh uncertainty as two more top FDA officials depart

BioPharma Drive: Drug Pricing

JUNE 20, 2025

The reported dismissal of high-ranking CBER officials Nicole Verdun and Rachael Anatol resurfaced lingering concerns about how gene therapies will be regulated under new FDA leadership.

Therapies

Therapies FDA Regulations

FDA turns back Capricor’s Duchenne cell therapy

BioPharma Drive: Drug Pricing

JULY 11, 2025

The company said it was “surprised” by the decision, which followed the ouster of cell and gene therapy officials from the agency during deramiocel’s review.

Therapies

Therapies FDA

Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment

Science Daily: Pharmacology News

MAY 15, 2025

A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the rare condition carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after birth, has responded positively to the treatment.

Treatment

Treatment Therapies Disease Research

FDA meeting gives window into gene therapy field’s angst

BioPharma Drive: Drug Pricing

JUNE 6, 2025

Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a regulatory forum Thursday.

Therapies

Therapies FDA

FDA to use new review tool on Sarepta’s gene therapy work

BioPharma Drive: Drug Pricing

JUNE 4, 2025

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene therapy applications it later brings to the regulator.

Therapies

Therapies FDA Regulations

Type Of Hormone Therapy Can Alter Breast Cancer Risk, Study Says

Drugs.com

JULY 7, 2025

MONDAY, July 7, 2025 — Some women have expressed concerns about the risk of breast cancer associated with using hormone therapy to treat symptoms of menopause like hot flashes and night sweats, and now, new research suggests that one type of.

Therapies

Therapies Research

Deafness reversed: Single injection brings hearing back within weeks

Science Daily: Pharmacology News

JULY 2, 2025

A cutting-edge gene therapy has significantly restored hearing in children and adults with congenital deafness, showing dramatic results just one month after a single injection. The therapy worked best in young children but still benefited adults, with one 7-year-old girl regaining almost full hearing.

Virus

Virus Therapies Research

Novel stem cell therapy repairs 'irreversible' corneal damage in clinical trial

Science Daily: Pharmacology News

MARCH 4, 2025

Results from a phase 1/2 clinical trial of a novel stem cell treatment for cornea injuries found 14 patients treated and tracked for 18 months had a more than 90% success rate at restoring the cornea's surface and improvements in vision.

Clinical Trials

Clinical Trials Trials Therapies Treatment

Is Radiation Therapy For Cancer a Double-Edged Sword?

Drugs.com

JULY 7, 2025

MONDAY, July 7, 2025 — Radiation therapy to treat cancer might be a double-edged sword that increases the risk posed by other malignant tumors, a new study argues. While radiation is often used alone or in combination with other treatments to.

Therapies

Therapies Treatment

Cell and gene therapy investment, once booming, is now in a slump

BioPharma Drive: Drug Pricing

AUGUST 29, 2024

Far fewer venture funding rounds were closed by cell and gene therapy developers over the first six months of 2024 than in prior years. Experts say there are several factors.

Therapies

FDA endorses speedy approval path for Regenxbio Duchenne gene therapy

BioPharma Drive: Drug Pricing

NOVEMBER 18, 2024

The agency's openness to a targeted pivotal study shows it’s still willing to consider accelerated clearance for Duchenne gene therapies despite questions about their effectiveness.

Therapies

Therapies FDA

100x improvement in sight seen after gene therapy trial

Science Daily: Pharmacology News

SEPTEMBER 6, 2024

The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it.

Therapies

Therapies Trials Research

Artiva prices $167M IPO, riding optimism for autoimmune cell therapy

BioPharma Drive: Drug Pricing

JULY 18, 2024

The offering comes days after the publication of a paper showing what analysts said was early proof that “off-the-shelf” cell therapies can treat inflammatory diseases.

Therapies

Therapies Disease

SpliceBio lands $135M for a new kind of eye gene therapy

BioPharma Drive: Drug Pricing

JUNE 11, 2025

The startup, which is backed by the venture arms of Sanofi, Roche and Novartis, is using dual adeno-associated viruses to help overcome the packaging constraints of current genetic medicines.

Packaging

Packaging Therapies Virus

New chronic pain therapy retrains the brain to process emotions

Science Daily: Pharmacology News

MAY 6, 2025

Researchers have created an effective therapy for chronic pain that reduces pain intensity by focusing on emotional regulation.

Therapies

Therapies Regulations Research

Duchenne approval exposes FDA rift over Sarepta gene therapy

BioPharma Drive: Drug Pricing

JUNE 21, 2024

Peter Marks’ decision to override the objections of agency staff and broaden use of Elevidys could have a “lasting impact” on gene therapy as well as the FDA, one analyst wrote.

Therapies

Therapies FDA

Hormone Therapy for Breast Cancer May Lower Dementia Risk

Drugs.com

JULY 16, 2024

TUESDAY, July 16, 2024 -- Hormone therapy for breast cancer might reduce a woman’s later risk of dementia and Alzheimer’s disease, a new study finds.Overall, hormone therapy is associated with a 7% lower risk of developing Alzheimer’s or a related d.

Therapies

Therapies Disease

Lexeo gene therapy shows signs of heart benefit in small study

BioPharma Drive: Drug Pricing

JULY 15, 2024

The company claims the results support exploring the possibility of an accelerated approval, citing flexibility by FDA officials in reviewing rare disease gene therapies.

Therapies

Therapies FDA Disease

Bright Light Therapy May Ease Multiple Forms of Depression

Drugs.com

OCTOBER 7, 2024

7, 2024 -- Light therapy: It's long been a go-to therapy for seasonal affective disorder (SAD), a kind of depression that can beset some people when winter looms and days shorten.But new research is suggesting that time spent in front. MONDAY, Oct.

Therapies

Therapies Research

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

New gene therapy for muscular dystrophy offers hope

Science Daily: Pharmacology News

JULY 17, 2024

A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.

Therapies

Therapies Treatment Disease Research

Early Hints at a Gene Therapy Cure for Herpes

Drugs.com

MAY 15, 2024

WEDNESDAY, May 15, 2024 -- An experimental gene therapy could one day provide a first-ever cure for genital and oral herpes, researchers report.The gene therapy removed 90% or more of oral herpes infection in lab mice, and it also suppressed how.

Therapies

Therapies Research

CAR-T for lupus: the ‘tip of the iceberg’ for cell therapy in autoimmune disease

BioPharma Drive: Drug Pricing

JANUARY 30, 2024

Since a landmark paper in 2022, drugmakers have begun nearly a dozen trials of cell therapies for lupus, with more set to start. Here’s why their efforts are worth watching.

Therapies

Therapies Disease Trials

Early Physical Therapy Key for Concussion Recovery, Study Shows

Drugs.com

JANUARY 6, 2025

MONDAY, Jan 6, 2025 -- Turns out, starting physical therapy earlier after a concussion may significantly improve recovery outcomes.A new study published recently in the Physical Therapy & Rehabilitation Journal followed just over 200 adults.

Therapies

Light Therapy Might Help Ease 'Dry' Form of Macular Degeneration

Drugs.com

OCTOBER 22, 2024

22, 2024 -- Light therapy could be a useful treatment for the most common form of age-related macular degeneration, a new study says.The therapy, called photobiomodulation or “red light” therapy, can reduce the risk of vision loss and. TUESDAY, Oct.

Therapies

Therapies Treatment

First-of-its-kind cell therapy approved by FDA for rare soft tissue cancer

BioPharma Drive: Drug Pricing

AUGUST 2, 2024

Adaptimmune’s Tecelra is the first TCR cell therapy to reach market and, at $727,000, is also the priciest cellular medicine for cancer in the U.S.

Therapies

Therapies FDA Marketing

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

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

Can robots break the cell therapy bottleneck?

BioPharma Drive: Drug Pricing

MAY 29, 2024

A partnership between cell therapy delivery specialist Portal Biotechnologies and precision robotics maker Multiply Labs could address manufacturing hurdles, the CEOs said.

Therapies

First therapy chatbot trial shows AI can provide 'gold-standard' care

Science Daily: Pharmacology News

MARCH 27, 2025

Researchers conducted the first clinical trial of an AI-powered therapy chatbot and found that, on average, people with diagnosed mental disorders experienced clinically significant improvements in their symptoms over eight weeks, according to new results.

Therapies

Therapies Trials Clinical Trials Research

Trial Confirms 'Life-Changing' Impact of Gene Therapy, Beqvez, for Hemophilia B

Drugs.com

SEPTEMBER 26, 2024

25, 2024 -- A new gene therapy approved earlier this year can serve as a sustainable single-dose treatment for people with hemophilia B, newly published clinical trial data shows. THURSDAY, Sept. People with hemophilia B saw their bleeding episodes.

Therapies

Therapies Trials Clinical Trials Treatment

Iovance, with approval of ‘TIL’ cell therapy, readies for complex launch

BioPharma Drive: Drug Pricing

FEBRUARY 20, 2024

The biotech set a price of roughly $515,000 per patient for its therapy Amtagvi, the first to be approved based on a decades-old technique of using tumor-infiltrating lymphocytes.

Therapies

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

Gene Therapy Reverses a Rare Cause of Vision Loss

Drugs.com

SEPTEMBER 6, 2024

6, 2024 -- Gene therapy may restore vision to children and adults robbed of their sight by a rare inherited condition called Leber congenital amaurosis, researchers report.The illness is caused by mutations in the GUCY2D gene, which. FRIDAY, Sept.

Therapies

Therapies Research

Gene Therapy Improves Vision in People With Inherited Blindness

Drugs.com

MAY 6, 2024

MONDAY, May 6, 2024 -- An injectable gene therapy caused measurable improvements in vision among a small group of people with inherited blindness, an early-stage clinical trial says.Researchers recruited 14 people with Leber Congenital Amaurosis.

Therapies

Therapies Clinical Trials Trials

With Duchenne decision ahead, FDA’s Marks pushes for speedy gene therapy approvals

BioPharma Drive: Drug Pricing

MAY 24, 2024

But the head of the FDA’s CBER office didn’t tip where the agency stands on potentially broadening use of Sarepta’s Duchenne gene therapy Elevidys.

Therapies

Therapies FDA

CAR-T Therapy Won't Raise Odds for a Second Cancer, Study Finds

Drugs.com

SEPTEMBER 11, 2024

11, 2024 -- Contrary to a warning placed on labels for CART-T cancer therapies, use of these treatments does not appear to boost the odds for a secondary cancer later, a new study shows.Researchers at Memorial Sloan Kettering. WEDNESDAY, Sept.

Therapies

Therapies Treatment

Zebrafish protein unlocks dormant genes for heart repair

Science Daily: Pharmacology News

JANUARY 2, 2025

This study marks an important step toward regenerative therapies to prevent heart failure. In mice, this protein was able to restore the heart by activating dormant repair genes without causing side effects, such as heart enlargement.

Therapies

Therapies Research

Major Women's Health Study Supports Hormone Replacement Therapy in Early Menopause

Drugs.com

MAY 1, 2024

WEDNESDAY, May 1, 2024 -- Hormone replacement therapy can safely ease middle-aged women’s symptoms during early menopause, data from a major women’s health study show. Women younger than 60 can use hormone replacement to treat symptoms.

Therapies

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

Scientists reverse Parkinson’s symptoms in mice — Could humans be next?

Science Daily: Pharmacology News

JULY 5, 2025

In mouse models, restoring the protein’s function with a targeted copper supplement dramatically rescued movement, hinting at a future therapy that could slow or halt the disease in people.

Therapies

Therapies Disease

Single-dose gene therapy is potentially life-changing for adults with hemophilia B

Science Daily: Pharmacology News

SEPTEMBER 25, 2024

Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the new results of an international Phase III clinical trial.

Therapies

Therapies Clinical Trials International Trials

Gene Therapy Might Tweak Fats, Help Prevent Arthritis in Overweight Kids

Drugs.com

OCTOBER 15, 2024

14, 2024 -- Gene therapy could help obese kids avoid health problems associated with excess weight, including arthritis, a new, preliminary study in mice suggests.The gene therapy prompts cells to convert inflammation-causing omega-6. TUESDAY, Oct.

Therapies

Single psilocybin trip delivers two years of depression relief for cancer patients

Science Daily: Pharmacology News

JUNE 16, 2025

In a groundbreaking trial, a single dose combined with therapy significantly reduced emotional suffering, and these effects often lasted over two years. Psilocybin, the active ingredient in magic mushrooms, might just revolutionize how depression and anxiety are treated in cancer patients.

Therapies

Therapies Trials Research

Gene therapy faces fresh uncertainty as two more top FDA officials depart

FDA turns back Capricor’s Duchenne cell therapy

Trending Sources

Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment

FDA meeting gives window into gene therapy field’s angst

FDA to use new review tool on Sarepta’s gene therapy work

Type Of Hormone Therapy Can Alter Breast Cancer Risk, Study Says

Deafness reversed: Single injection brings hearing back within weeks

Novel stem cell therapy repairs 'irreversible' corneal damage in clinical trial

Is Radiation Therapy For Cancer a Double-Edged Sword?

Cell and gene therapy investment, once booming, is now in a slump

FDA endorses speedy approval path for Regenxbio Duchenne gene therapy

100x improvement in sight seen after gene therapy trial

Artiva prices $167M IPO, riding optimism for autoimmune cell therapy

SpliceBio lands $135M for a new kind of eye gene therapy

New chronic pain therapy retrains the brain to process emotions

Duchenne approval exposes FDA rift over Sarepta gene therapy

Hormone Therapy for Breast Cancer May Lower Dementia Risk

Lexeo gene therapy shows signs of heart benefit in small study

Bright Light Therapy May Ease Multiple Forms of Depression

Sarepta scraps a Duchenne drug as gene therapy sales rise

New gene therapy for muscular dystrophy offers hope

Early Hints at a Gene Therapy Cure for Herpes

CAR-T for lupus: the ‘tip of the iceberg’ for cell therapy in autoimmune disease

Early Physical Therapy Key for Concussion Recovery, Study Shows

Light Therapy Might Help Ease 'Dry' Form of Macular Degeneration

First-of-its-kind cell therapy approved by FDA for rare soft tissue cancer

Pfizer hemophilia gene therapy arrives in US to uncertain future

FDA Approves Landmark Sickle Cell Gene Therapies, Casgevy and Lyfgenia

Can robots break the cell therapy bottleneck?

First therapy chatbot trial shows AI can provide 'gold-standard' care

Trial Confirms 'Life-Changing' Impact of Gene Therapy, Beqvez, for Hemophilia B

Iovance, with approval of ‘TIL’ cell therapy, readies for complex launch

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

Gene Therapy Reverses a Rare Cause of Vision Loss

Gene Therapy Improves Vision in People With Inherited Blindness

With Duchenne decision ahead, FDA’s Marks pushes for speedy gene therapy approvals

CAR-T Therapy Won't Raise Odds for a Second Cancer, Study Finds

Zebrafish protein unlocks dormant genes for heart repair

Major Women's Health Study Supports Hormone Replacement Therapy in Early Menopause

Combo Therapy May Be Advance Against Liver Cancer

Scientists reverse Parkinson’s symptoms in mice — Could humans be next?

Single-dose gene therapy is potentially life-changing for adults with hemophilia B

Gene Therapy Might Tweak Fats, Help Prevent Arthritis in Overweight Kids

Single psilocybin trip delivers two years of depression relief for cancer patients

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