top of page
PharmaTell Banner Med Finance.jpeg

Eyes On Pharma Blog 

Eyes on Genetic Medicines & Pipelines

As mentioned in our previous post, Eyes on Gene Therapy The Background, we thought it would also be helpful to share information on development projects across the Genetic Medicines area. It's impressive to see the numbers of indications, types of therapies being tested, and different approaches the companies are taking to find cures for rare diseases.

For much of the data we refer to the ASGCT 3Q23 Update on Gene Cell and RNA Therapies report. We've also included PharmaTell Studio reports highlighting some of the recent approvals and data readouts in the Fourth Quarter (See links below). Let's take a look at what's approved and what's in development!

Globally Approved Gene, Cell, and RNA therapies for clinical use (end of 3Q23 Data)
  • 27 gene therapies (including genetically modified cell therapies) - in Q3 2023, Innovvent and IASO Bio's Fucaso, an anti-BCMA-targeting CAR-T therapy, was approved in China for multiple myeloma.

  • 26 RNA therapies- in Q3 2023, Daiichi Sankyo’s Daichirona COVID-19 RNA vaccine was approved in Japan.

  • 65 non-genetically modified cell therapies.

In 4Q23 the following therapies were approved:

  • Casgevy - Vertx/CRISPR for Sickle Cell Disease - 16 Nov in EU and 8 Dec USA

  • Lyfgenia - bluebird bio for Sickle Cell Disease - 8 Dec USA

  • Wainua - Ionis - AZN Pharmaceuticals for Transthyretin-mediated Amyloidoisis 21 Dec

Current Genetic Medicines with US FDA and/or EMA Approval | Positive Opinion

Genetic Medicines Pipeline

  • 2,082 gene therapies (including genetically modified cell therapies such as CAR T-cell therapies) are in development, accounting for 53% of gene, cell, and RNA therapies .

  • 862 non-genetically modified cell therapies are in development, accounting for 22% of gene, cell, and RNA therapies.

A total of 3,866 therapies are in development, ranging from preclinical through pre-registration.

3Q23 ASGCT Report - Gene therapy pipeline: Quarterly comparison

For the first time in the past year, the number of gene therapies in Phase I development has grown since the previous quarter, increasing by 7%. For the fourth quarter in a row, the number of Phase III gene therapies has remained unchanged at 30 therapies. Rocket Pharmaceutical filed for approval in the US for its lentiviral gene therapy, RP-L201, in leukocyte adhesion deficiency. Abeona Therapetics filed for approval in the US for its engineered cell therapy, EB-101, in epidermolysis bullosa.

Therapies currently in pre-registration:

In China:

  • Zevor-cel (CARsgen Therapeutics)

  • Inaticabtagene autoleucel (CASI Pharmaceuticals, Juventas Cell Therapy)

In the EU, UK, and US:

·      Exagamglogene autotemcel (CRISPR Therapeutics, Vertex Pharmaceuticals) - Approved 8 Dec 2023

In the EU and US:

·      Fidanacogene elaparvovec (Pfizer)

In the US:

  • Lovotibeglogene autotemcel (bluebird bio) - Approved 8 Dec 2023

  • RP-L201 (Rocket Pharmaceuticals)

  • EB-101 (Abeona Therapeutics)


Genetic modification: in vivo vs. ex vivo

Ex vivo genetic modification is more widely used for gene therapies in pipeline development. In Q3 2023, in vivo delivery techniques were used in 31% of gene therapies, one percentage point higher than the previous quarter.

Gene Therapy breakdown: CAR-Ts continue to dominate the pipeline

CAR-T cell therapies remain the most common technology used in the pipeline of genetically modified cell therapies (preclinical through to pre-registration), representing 47%, followed by the “other” category at 36%, which includes a list of less commonly used technologies including TCR-NK, CAR-M, and TAC-T. 97% of CAR-T cell therapies are in development for cancer indications. The remaining non-oncology indications include scleroderma, HIV/AIDS, and autoimmune disease (unspecified).

On a side note – the potential risk of secondary cancers associated with CAR-T therapies is currently being investigated by the FDA. The FDA’s investigation stems from reports of T-cell malignancies, including CAR-positive lymphoma, in patients treated with BCMA- or CD19-directed CAR-T immunotherapies. The FDA’s review applies to all approved CAR-Ts, six in total so far. Several article in PharmaTell Studio talk more about the impact of this review here and here.

Gene therapy pipeline: Most commonly targeted therapeutic areas

Oncology and rare diseases (as mentioned previously) remain the top targets for gene therapy development in both the overall pipeline (preclinical to pre-registration) and in the clinic (Phase I to pre-registration). Development for rare diseases most comonly occurs in oncology, representing a majority of 53% compared to non-oncology rare disease gene therapy pipeline development.

Gene therapy pipeline: Most common rare diseases targeted

Of the 1,023 pipeline (preclinical to pre-registration) gene therapies being developed, 8 out of the top 10 are oncological, as seen throughout 2022 and H1 2023.

In the same order as the previous eight quarters, the top oncology targets for gene therapy were:

  1. Myeloma (96 therapies)

  2. Non-Hodgkin’s lymphoma (86 therapies)

  3. Acute myelogenous leukemia (85 therapies)

  4. B-cell lymphoma (70 therapies)

  5. Ovarian cancer (63 therapies).

Gene therapy pipeline: Most common targets of the gene therapies in preclinical trials through pre-registration for which targets are disclosed.

Oncology Targets

1.     CD19 (153 therapies)

2.     B-cell maturation antigen (BCMA), also known as TNF receptor superfamily member 17 (55 therapies)

3.     CD22 molecule (33 therapies)

Non-Oncology Targets

1.     CD19 (13 therapies)

2.     Coagulation factor VIII (12 therapies) drops to second in 3Q23.

Gene therapy clinical trial activity

58 trials were initiated in Q3 2023 for gene therapies. The proportion of gene therapy trials for non-oncology indications has increased by six percentage points since the previous quarter, to 38%, continuing the trend of an increasing proportion of non-oncology gene therapy trials initiated each quarter since Q4 2022.

Non-genetically modified cell therapy pipeline: Most common therapeutic areas targeted:

(of the cell therapies in development (preclinical through pre-registration)

  • Oncology (295 therapies) and rare diseases (260 therapies) remain the top areas of non- genetically modified cell therapy development.

  • Of the non-genetically modified cell therapies in preclinical to pre-registration stages for rare diseases, 64% are in development for non-oncology rare diseases, as found in the previous quarter.

Non-genetically modified cell therapy pipeline: Most common diseases targeted:

Of the therapies for which indications are specified, for the first time in over a year, COVID- 19 complications is not within the top 3:

  1. Osteoarthritis (38 therapies)

  2. Acute respiratory distress syndrome (36 therapies)

  3. Parkinson’s disease (34 therapies)

Non-genetically modified cell therapy pipeline: Most common rare diseases targeted:

Of the therapies in development (preclinical through pre-registration) for rare diseases, the top 3 oncology indications are:

  1. liver cancer (20 therapies)

  2. acute myelogenous leukemia (18 therapies)

  3. ovarian cancer (16 therapies).

The top 3 non-oncology indications are:

  1. acute respiratory distress syndrome (36 therapies)

  2. graft-versus-host disease (18 therapies)

  3. spinal cord injury (17 therapies).

Non-genetically modified cell therapy trial activity

21 trials were initiated for non-genetically modified cell therapies in Q3 2023, 10 less than the previous quarter. Of these 21, 57% are for non-oncology indications, a decrease of 1 percentage point from Q2 2023.

RNA therapy pipeline: Most common modalities

Of RNA therapies in the pipeline, messenger RNA (mRNA) and RNA interference (RNAI) continue to be the preferred RNA modalities for research.

RNAI, mRNA, and antisense oligonucleotides: Preclinical vs. clinical

The majority of RNAI, mRNA, and anitsense therapeutics in development are in preclinical development, representing 75%, 68%, and 67% of their respective pipelines.

RNA therapies: Most commonly targeted therapeutic areas:

(Of the 976 RNA therapies currently in the pipeline from preclinical trough pre-registration)

  1. Non-oncology indications continue to be the most targeted rare diseases by RNA therapies, representing a majority of 81%.

  2. Rare diseases (307 therapies)

  3. anti-infective indications (237 therapies)

  4. oncology indications (213 therapies).

RNA therapies: Most common rare diseases targeted:

(Of the RNA therapies currently in the pipeline from preclinical through pre-registration)

Top specified rare oncology indications are:

  1. pancreatic (21 therapies)

  2. liver(15 therapies)

  3. ovarian cancer (10 therapies).

For non-oncology rare diseases:

  1. Duchenne muscular dystrophy (28 therapies)

  2. amyotrophic lateral sclerosis (22 therapies)

  3. Huntington’s disease (13 therapies)

RNA therapy pipeline: Clinical trial activity

25RNA trials were initiated in Q3 2023, compared to 26 in Q2 2023, 80% of which were non- oncology indications.

Alliance, Acquisitions, Financing in Genetic Medicines

Total financings, alliances, and acquisitions amounted to 116 transactions in Q3 2023, remaining virtually flat from the previous quarter’s 117 aggregate. Q3 2023’s deal activity represented a 5% increase over the 110 deals signed in the same quarter a year ago. In the last year, financing volume has seen the biggest jump, with 54 completed in Q3 2022 vs. 67 in Q3 2023 (+24%)

Q3 2023 Acquisitions

Acquisition volume remained steady in Q3 2023 with 9 deals:

Novartis paid $500 million upfront plus up to $500 million in earn-outs for DTx Pharma in the largest deal of the quarter. While Quince made an equity exchange deal to acquire EryDel and its ex vivo red blood cell platform.

Start-up Financing

Start-up financing transactions in 3Q23 were lower than the previous quarter (17 vs 21 transactions), but significantly lower in value - $348 M vs $1.3M. Details are in the tables below.

Start-Up Genetic Medicine Firms in 3Q23

ASGCT highlights 3 new startups in the area for 3Q23:

  • Tenpoint Therapeutics - Targeting degenerative ocular diseases with vision-restoring engineered cell-based therapeutics and in vivo reprogramming. Series A / $ 70M

  • Kincell Bio - Early-stage immune cell therapy CDMO. Initial Funding / $36M

  • Epigenic Therapeutics - Next-gen gene modulation therapies based on epigenome regulation and editing for chronic and prevalent diseases. Series A /$32M

Upcoming Catalysts

We barely skimmed the surface of all the work that is ongoing in the area - please dig deeper in the articles below and your other favorite resources. Feel free to share your thoughts and resources with us too! It's great to see the different targets and I'm anxious to see the results for so many patients who have been unable to get relief for their conditions for so long.

If you are interested in following all the happenings in the Genetic Medicines area - you might like our Syndicated Alert which provides current research and insights on the area in a bi-weekly email alert. Check it out here.

Please refer to the following articles for more details on the information shared in this article, plus more on Cell and RNA Therapies, and select companies covered by our network.


ASGCT - Citeline Report:  Gene, Cell, + RNA Therapy Landscape Report, Q3 2023 Quarterly Data Report

Not included in post but a great Deep Dive Report in PharmaTell Studio :Genomic Medicine Deep Dive - Redrawing Frontiers

Editor’s Note: Fair Use: We do not own the content of the sources we reference for this post. We have compiled insights and data from multiple sources and are conveying the information in one blog post to report and educate our clients on the latest insights and opinions in the area. Please contact the original authors for questions.

If you are a PharmaTell client and can’t access the PharmaTell-specific links, please contact us at  

If you are not a client and would like to learn more about PharmaTell and obtain access for a free trial, please send us an email at, and we will be happy to assist you.


bottom of page