genetic disorders Archives

Best in Biotech 24 Jul 2025

Which biotech companies are gaining traction in genetic diseases?

Discover twelve companies focused on genetic diseases that managed to close funding rounds in the last two years.

July 24, 2025 - 12 minutesmins - By Jules Adam

Podcast 11 Jul 2025

How OMass Therapeutics is tackling congenital adrenal hyperplasia, a rare genetic disorder

This week we talk about Congenital Adrenal Hyperplasia and how OMass Therapeutics is approaching it with the company CEO, Ros Deegan.

July 11, 2025

News and Trends 12 May 2025

Pharma giants pull back on AAV research: what’s next for the gene therapy space?

Biopharmas have been scaling back on AAV research for sometime now. Find out why and what’s on the horizon for drug delivery methods.

May 12, 2025 - 8 minutesmins - By Roohi Mariam Peter

Best in Biotech 29 Apr 2025

Six biotech companies leading the charge in hemophilia treatment

Discover six hemophilia companies developing new treatments for the genetic bleeding disorder, from gene therapies to RNAi therapies.

April 29, 2025 - 10 minutesmins - By Willow Shah-Neville

In Depth 7 Apr 2025

Will tRNA therapy be the next big thing in genetic disease treatment?

Discover how an emerging technology called tRNA therapy could become the next big thing in genetic disease treatment.

April 7, 2025 - 12 minutesmins - By Willow Shah-Neville

Best in Biotech 20 Feb 2025

3 promising biotech approaches to treat Hunter syndrome, a rare genetic disorder

Discover three promising approaches being investigated to treat Hunter syndrome, a rare genetic disorder affecting children.

February 20, 2025 - 10 minutesmins - By Willow Shah-Neville

In Depth 21 Nov 2024

Huntington’s disease: a therapeutic field on a bumpy ride

For years, biotechs have been trying to develop medicines to cure the disease but much to no avail. Now, as clinical studies advance, there is hope.

November 21, 2024 - 9 minutesmins - By Roohi Mariam Peter

Best in Biotech 21 Nov 2024

Eight areas where gene therapy shines

Delve into the numerous applications of gene therapy, from cancer to hematological disorders and respiratory diseases.

November 21, 2024 - 12 minutesmins - By Jules Adam

News and Trends 23 Oct 2024

RNA editing in humans: the race begins with first successful trial

Wave Life Sciences achieves RNA editing in the clinic, crossing a major milestone in the space.

October 23, 2024 - 6 minutesmins - By Roohi Mariam Peter

Best in Biotech 3 Oct 2024

Six biotechs driving progress in Duchenne muscular dystrophy

In this article, we take a look at six clinical-stage biotechs that are fostering R&D in the Duchenne muscular dystrophy therapeutic space.

October 3, 2024 - 8 minutesmins - By Roohi Mariam Peter

News and Trends 27 Sep 2024

Aptadir Therapeutics launches to develop new class of RNA inhibitors against cancer and genetic disorders

Italian startup Aptadir Therapeutics has launched with $1.6 million to bring a new class of drugs to the clinic and a technology to be reckoned with.

September 27, 2024 - 6 minutesmins - By Roohi Mariam Peter

In Depth 10 Jul 2024

Biotech’s battle against skeletal dysplasia: Ray of hope for children with rare genetic disorder

As treatments for skeletal dysplasia are intended to alleviate painful symptoms, two drugs are battling out in the clinic to prove their effectiveness.

July 10, 2024 - 7 minutesmins - By Roohi Mariam Peter

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Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

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genetic disorders

Which biotech companies are gaining traction in genetic diseases?

How OMass Therapeutics is tackling congenital adrenal hyperplasia, a rare genetic disorder

Pharma giants pull back on AAV research: what’s next for the gene therapy space?

Six biotech companies leading the charge in hemophilia treatment

Will tRNA therapy be the next big thing in genetic disease treatment?

3 promising biotech approaches to treat Hunter syndrome, a rare genetic disorder

Huntington’s disease: a therapeutic field on a bumpy ride

Eight areas where gene therapy shines

RNA editing in humans: the race begins with first successful trial

Six biotechs driving progress in Duchenne muscular dystrophy

Aptadir Therapeutics launches to develop new class of RNA inhibitors against cancer and genetic disorders

Biotech’s battle against skeletal dysplasia: Ray of hope for children with rare genetic disorder

Subscribe to our newsletter to get the latest biotech news!

Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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